On November 1, 2023 United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, reported its financial results for the quarter ended September 30, 2023 (Press release, United Therapeutics, NOV 1, 2023, View Source [SID1234636659]). Total revenues in the third quarter of 2023 grew 18% year-over-year to $609.4 million, compared to $516.0 million in the third quarter of 2022.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"It’s a testament to our innovative research and commercial execution that we are again reporting record quarterly revenue and double-digit revenue growth," said Martine Rothblatt, Ph.D., Chairperson and Chief Executive Officer. "On top of our record results, we continue to progress our near-term pipeline with the ongoing TETON 1 and TETON 2 studies of nebulized Tyvaso in idiopathic pulmonary fibrosis and the ADVANCE OUTCOMES study of ralinepag in pulmonary arterial hypertension, both of which could generate data in 2025."

"With over $600 million in quarterly revenue, we’re well on our way to achieving our expected $4 billion annual run rate by mid-decade," said Michael Benkowitz, President and Chief Operating Officer. "Our commercial teams achieved meaningful growth in all of our key products, and our recent field force expansion in PH-ILD will help us build on this momentum."

Third Quarter 2023 Financial Results

Key financial highlights include (dollars in millions, except per share data):

Three Months Ended
September 30,

Dollar
Change

Percentage
Change

2023

2022

Total revenues

$

609.4

$

516.0

$

93.4

18

%

Net income

$

267.6

$

239.3

$

28.3

12

%

Net income, per basic share

$

5.71

$

5.26

$

0.45

9

%

Net income, per diluted share

$

5.38

$

4.91

$

0.47

10

%

Revenues

The table below presents the components of total revenues (dollars in millions):

Three Months Ended
September 30,

Dollar
Change

Percentage
Change

2023

2022

Net product sales:

Tyvaso DPI(1)

$

205.1

$

63.1

$

142.0

225

%

Nebulized Tyvaso(1)

120.7

194.6

(73.9

)

(38

)%

Total Tyvaso

325.8

257.7

68.1

26

%

Remodulin(2)

131.1

114.0

17.1

15

%

Orenitram

92.0

87.5

4.5

5

%

Unituxin

51.3

46.1

5.2

11

%

Adcirca

7.3

10.7

(3.4

)

(32

)%

Other

1.9

—

1.9

NM

(3)

Total revenues

$

609.4

$

516.0

$

93.4

18

%

(1)

Net product sales include both the drug product and the respective inhalation device.

(2)

Net product sales include sales of infusion devices, such as the Remunity Pump.

(3)

Calculation is not meaningful.

Net product sales of our treprostinil-based products (Tyvaso DPI, nebulized Tyvaso, Remodulin, and Orenitram) grew by $89.7 million, or 20%, for the third quarter of 2023, as compared to the third quarter of 2022. The growth in total Tyvaso revenues resulted primarily from an increase in total quantities sold driven by the commercial launch of Tyvaso DPI in June 2022 and continued growth in utilization by patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD). The growth in Tyvaso DPI revenues resulted from an increase in quantities sold. The decrease in nebulized Tyvaso revenues resulted primarily from a decrease in quantities sold following the commercial launch of Tyvaso DPI. The increase in Remodulin revenues resulted from an increase in quantities sold.

Expenses

Cost of sales. The table below summarizes cost of sales by major category (dollars in millions):

Three Months Ended
September 30,

Dollar
Change

Percentage
Change

2023

2022

Category:

Cost of sales

$

69.1

$

37.1

$

32.0

86

%

Share-based compensation expense(1)

1.0

0.1

0.9

900

%

Total cost of sales

$

70.1

$

37.2

$

32.9

88

%

(1)

Refer to Share-based compensation below.

Cost of sales, excluding share-based compensation. Cost of sales for the three months ended September 30, 2023 increased as compared to the same period in 2022, primarily due to an increase in Tyvaso DPI royalty expense and product costs following its commercial launch in June 2022.

Research and development expense. The table below summarizes the nature of research and development expense by major expense category (dollars in millions):

Three Months Ended
September 30,

Dollar
Change

Percentage
Change

2023

2022

Category:

External research and development(1)

$

47.2

$

37.6

$

9.6

26

%

Internal research and development(2)

34.3

29.1

5.2

18

%

Share-based compensation expense(3)

3.6

2.0

1.6

80

%

Impairments(4)

—

—

—

—

%

Other(5)

(0.4

)

(2.6

)

2.2

85

%

Total research and development expense

$

84.7

$

66.1

$

18.6

28

%

(1)

External research and development primarily includes fees paid to third parties (such as clinical trial sites, contract research organizations, and contract laboratories) for preclinical and clinical studies and payments to third-party contract manufacturers before FDA approval of the relevant product.

(2)

Internal research and development primarily includes salary-related expenses for research and development functions, internal costs to manufacture product candidates before FDA approval, and internal facilities-related expenses, including depreciation, related to research and development activities.

(3)

Refer to Share-based compensation below.

(4)

Impairments primarily includes impairment charges to write down the carrying value of in-process research and development and of certain property, plant, and equipment as a result of research and development activities. There were no impairment charges during the three months ended September 30, 2023 and September 30, 2022.

(5)

Other primarily includes upfront fees and milestone payments to third parties under license agreements related to development-stage products and adjustments to the fair value of our contingent consideration obligations.

Research and development expense, excluding share-based compensation. Research and development expense for the three months ended September 30, 2023 increased as compared to the same period in 2022, primarily due to increased expenditures related to: (1) the TETON 1 and TETON 2 clinical studies of nebulized Tyvaso in patients with idiopathic pulmonary fibrosis; and (2) organ manufacturing projects.

Selling, general, and administrative expense. The table below summarizes selling, general, and administrative expense by major category (dollars in millions):

Three Months Ended
September 30,

Dollar
Change

Percentage
Change

2023

2022

Category:

General and administrative

$

90.4

$

85.7

$

4.7

5

%

Sales and marketing

20.7

17.2

3.5

20

%

Share-based compensation expense (benefit)(1)

16.5

(4.5

)

21.0

467

%

Total selling, general, and administrative expense

$

127.6

$

98.4

$

29.2

30

%

(1)

Refer to Share-based compensation below.

Share-based compensation. The table below summarizes share-based compensation expense (benefit) by major category (dollars in millions):

Three Months Ended
September 30,

Dollar
Change

Percentage
Change

2023

2022

Category:

Stock options

$

6.0

$

5.7

$

0.3

5

%

Restricted stock units

12.5

9.9

2.6

26

%

Share tracking awards plan (STAP)

2.1

(18.5

)

20.6

111

%

Employee stock purchase plan

0.5

0.5

—

—

%

Total share-based compensation expense (benefit)

$

21.1

$

(2.4

)

$

23.5

979

%

The increase in share-based compensation expense for the three months ended September 30, 2023, as compared to the same period in 2022, was primarily due to an increase in STAP expense driven by a two percent increase in our stock price for the three months ended September 30, 2023, as compared to an 11 percent decrease in our stock price for the same period in 2022.

Income tax expense. Income tax expense for the three months ended September 30, 2023 and 2022 was $84.2 million and $73.2 million, respectively. Our effective income tax rate for the three months ended September 30, 2023 and 2022 was 24 percent and 23 percent, respectively.

Inducement Restricted Stock Units

On October 30, 2023, we granted a total of 23,483 restricted stock units under our 2019 Inducement Stock Incentive Plan to 16 newly hired employees. One of the grants will vest in three equal installments on the third, fourth, and fifth anniversaries of the grant date on October 30, 2026, October 30, 2027, and October 30, 2028. All other grants cliff vest fully on the third anniversary of the grant date on October 30, 2026. These restricted stock unit grants assume continued employment on such dates, and are subject to the standard terms and conditions we filed with the SEC as Exhibit 10.2 to our Current Report on Form 8-K on March 1, 2019. We are providing this information in accordance with Nasdaq Listing Rule 5635(c)(4).

Webcast

We will host a webcast to discuss our third quarter 2023 financial results on Wednesday, November 1, 2023, at 9:00 a.m. Eastern Time. The webcast can be accessed live via our website at View Source A replay of the webcast will also be available at the same location on our website approximately 12 hours after the conclusion of the call.

On November 1, 2023 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, reported the presentation of additional data from the Phase 3 DeFi trial at the 2023 Connective Tissue Oncology Society (CTOS) Annual Meeting, being held November 1-4, 2023 (Press release, SpringWorks Therapeutics, NOV 1, 2023, View Source [SID1234636658]). These data demonstrate the impact of nirogacestat, an investigational gamma secretase inhibitor, on physical and role functioning in adults with desmoid tumors. Data from the DeFi trial demonstrated statistically significant and clinically meaningful improvements across all primary and key secondary endpoints and were previously presented at leading medical congresses and published in the March 9, 2023 edition of the New England Journal of Medicine.1-3

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Desmoid tumors are locally aggressive and invasive soft tissue tumors that can cause severe pain and functional impairment. These impairments can be physical, such as difficulty walking or carrying out daily tasks, or role-related, such as difficulty caring for children or working, and they severely impact the day-to-day lives of patients," said Jim Cassidy, M.D., Ph.D., Chief Medical Officer of SpringWorks. "We are pleased that the robust data from our Phase 3 DeFi trial demonstrated that nirogacestat provided clinically meaningful improvements in key patient-reported outcomes, including pain as well as both physical and role functioning. We look forward to the opportunity to bring this important new medicine to the desmoid tumor community following anticipated U.S. regulatory approval."

Poster Presentation at the 2023 CTOS Meeting

Impact of Nirogacestat on Functional Status in Patients with Desmoid Tumors: Results from the Phase 3 DeFi Study

Abstract #: 1571083
Poster #: P 188
Poster Session Date and Time: Thursday, November 2, 5:30-6:30 p.m. CEST

As previously reported in the DeFi trial (NCT03785964), nirogacestat met its primary endpoint of significantly improving progression-free survival compared to placebo in adult patients with progressing desmoid tumors (hazard ratio: 0.29 [95% CI, 0.15–0.55]; P<0.001). Nirogacestat also achieved a significant and clinically meaningful improvement in physical and role functioning status, a key secondary endpoint, compared with placebo at Cycle 10 (p<0.001). The most frequently reported treatment-emergent adverse events that occurred in participants receiving nirogacestat were diarrhea (84%), ovarian dysfunction (75% of women of childbearing potential), nausea (54%), fatigue (51%), hypophosphatemia (42%), and maculopapular rash (32%).

During the DeFi study, the impact of nirogacestat on functional status was evaluated. Changes from baseline in physical and role functioning were compared between nirogacestat and placebo at Cycle 10, the prespecified time point for key secondary endpoints. Statistically significant and clinically meaningful improvements in physical and role functioning were observed with nirogacestat compared with placebo at Cycle 10 across all three assessment tools used: the GOunder/Desmoid Tumor Research Foundation DEsmoid Impact Scale (GODDESS DTIS) Physical Functioning (PF) subscale, the European Organisation for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) PF and Role Functioning (RF) subscales, and the Patient-Reported Outcomes Measurement Information System Physical Function Short Form 10a (PROMIS PF10a) tool. Improvements in functional status were rapid, becoming evident as early as Cycle 2 (the first post-treatment time point evaluated) and these improvements were maintained through Cycle 24 across multiple tools measuring physical and role functioning.

The following results are being presented at CTOS:

Nirogacestat significantly improved mean physical functioning score from baseline per the GODDESS DTIS PF domain compared with placebo at the pre-specified time point. The GODDESS DTIS PF domain captures varying degrees of vigorous and moderate daily activity, including moving and reaching.
Nirogacestat significantly improved mean physical functioning score from baseline per the EORTC QLQ-C30 PF subscale compared with placebo at the pre-specified time point. The EORTC QLQ-C30 PF subscale captures the concepts of strenuous activities, taking a long walk, taking a short walk, need to stay in a bed or chair, and help with eating, dressing, washing, and using the toilet.
Nirogacestat significantly improved mean role functioning score from baseline per the EORTC QLQ-C30 RF subscale compared with placebo at the pre-specified time point. The EORTC QLQ-C30 RF subscale captures the concepts of work or other daily activities and hobbies or leisure activities.
At Cycle 10, patients receiving nirogacestat were five times more likely to have a clinically meaningful improvement in physical functioning (GODDESS DTIS Physical Functioning and EORTC QLQ-C30 Physical Functioning), and twice as likely to have a clinically meaningful improvement in role functioning (EORTC QLQ-C30 Role Functioning) than those receiving placebo. By Cycle 4, the mean PROMIS PF10a score in the nirogacestat arm reached the average physical function observed in the general U.S. population whereas the score of the placebo arm did not.
"Treatment goals for patients with desmoid tumors often focus on tumor growth endpoints, such as progression-free survival, but reducing pain and improving functioning are also very important since these have a significant impact on patients’ quality of life," said Bernd Kasper, M.D., Ph.D., University of Heidelberg, Mannheim Cancer Center, Mannheim, Germany and Principal Investigator of the DeFi trial. "It is very encouraging that patients experienced meaningful improvements in their functional status while on nirogacestat and that these improvements were sustained over the course of the study."

About the DeFi Trial

DeFi (NCT03785964) is a global, randomized (1:1), double-blind, placebo-controlled Phase 3 trial evaluating the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors. The double-blind phase of the study randomized 142 patients (nirogacestat, n=70; placebo n=72) to receive 150 mg of nirogacestat or placebo twice daily. Key eligibility criteria included tumor progression by ≥20% as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months prior to screening. The primary endpoint was progression-free survival, as assessed by blinded independent central review, or death by any cause. Secondary and exploratory endpoints include safety and tolerability measures, objective response rate (ORR), duration of response, changes in tumor volume assessed by magnetic resonance imaging (MRI), and changes in patient-reported outcomes (PROs). DeFi includes an open-label extension phase, which is ongoing.

About Desmoid Tumors

Desmoid tumors are rare, aggressive, locally invasive, potentially morbid tumors of the soft tissues.4,5 While they do not metastasize, desmoid tumors are associated with a high rate of recurrence.4-6 Sometimes referred to as aggressive fibromatosis, or desmoid fibromatosis, these soft tissue tumors can be serious, debilitating, and in rare cases when vital organs are impacted, they can be life-threatening.5,8

Desmoid tumors are most commonly diagnosed in patients between the ages of 20 to 44 years, with a two-to-three times higher prevalence in females.7-10 It is estimated that there are 1,000-1,650 new cases diagnosed per year in the United States.10-12

Historically, desmoid tumors were treated with surgical resection, but this approach has become less favored due to a high recurrence rate after surgery.4,7,13 There are currently no FDA-approved therapies for the treatment of desmoid tumors.

About Nirogacestat

Nirogacestat is an oral, selective, small molecule gamma secretase inhibitor in Phase 3 clinical development for desmoid tumors. SpringWorks is also evaluating nirogacestat as a potential treatment for patients with ovarian granulosa cell tumors and for patients with multiple myeloma as part of several B-cell maturation agent (BCMA) combination therapy regimens in collaboration with leaders in industry and academia. Nirogacestat is an investigational drug for which safety and efficacy have not been established.

The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for nirogacestat for the treatment of adults with desmoid tumors, which is being reviewed under the FDA’s Real-Time Oncology Review program. The NDA has been given a Prescription Drug User Fee Act (PDUFA) action date of November 27, 2023. The FDA also granted Fast Track and Breakthrough Therapy Designations to nirogacestat for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. In addition, nirogacestat has received Orphan Drug Designation from the FDA for the treatment of desmoid tumors and from the European Commission for the treatment of soft tissue sarcoma.

On November 1, 2023 Seagen Inc. (Nasdaq:SGEN) (Seagen or the Company) reported financial results today for the third quarter ended September 30, 2023 (Press release, Seagen, NOV 1, 2023, View Source [SID1234636657]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

David Epstein, Chief Executive Officer of Seagen said, "Seagen continues to build momentum in 2023 and delivered strong performance, marked by record quarterly net product sales, with significant year-over-year growth of 33%, contributing to total revenues of $649 million. Growth was primarily driven by PADCEV (enfortumab vedotin-ejfv), which grew 89% over the same quarter of last year following the successful launch in the U.S. in combination with Keytruda (pembrolizumab) as a front-line treatment for patients with advanced urothelial cancer who are not eligible to receive cisplatin-containing chemotherapy. We also announced positive trial readouts across our commercial portfolio in multiple patient settings." Highlights include:

PADCEV in combination with Keytruda demonstrated a 53% reduction in the risk of death and a near doubling in the median overall survival (OS) (31.5 months vs 16.1 months) versus chemotherapy in patients with previously untreated locally advanced or metastatic urothelial cancer (la/mUC) who were eligible for cisplatin- or carboplatin-containing chemotherapy regardless of PD-L1 status. Data from the global phase 3 study, EV-302 / KEYNOTE-A39, were presented during the Presidential Symposium at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in October.
TIVDAK (tisotumab vedotin-tftv) demonstrated superior overall survival with a 30% reduction in the risk of death in the innovaTV 301 pivotal trial for patients with recurrent or metastatic cervical cancer with disease progression on or after first-line therapy. Additionally, progression-free survival was statistically significant with a 33% reduction in the risk of disease worsening or death compared with chemotherapy and the confirmed objective response rate was also improved with TIVDAK (17.8%) compared with chemotherapy (5.2%). Results were presented during the Presidential Symposium at ESMO (Free ESMO Whitepaper).
The HER2CLIMB-02 pivotal trial of TUKYSA (tucatinib) in combination with antibody-drug conjugate (ADC) ado-trastuzumab emtansine met the primary endpoint of progression-free survival in patients with previously treated HER2-positive metastatic breast cancer.
"We remain very excited about the pending acquisition of Seagen by Pfizer as we look to build a world-class oncology organization which will broaden patient reach and accelerate development of our innovative pipeline," concluded Mr. Epstein.

Roger Dansey, President of Research and Development and Chief Medical Officer, added, "We have now demonstrated an overall survival benefit in targeted indications across all four of our approved products. The PADCEV EV-302 study has the potential to be practice changing and offer a new standard of care for first-line metastatic bladder cancer. Building on our expertise and innovative platform, we are advancing next-generation ADC and immuno-oncology targeted therapies. We recently initiated a phase 3 trial of disitamab vedotin in combination with pembrolizumab in patients with previously untreated locally advanced or metastatic HER2-positive urothelial cancer, along with a phase 1 trial for SGN-EGFRd2, a novel T cell engaging bispecific antibody. We also remain on track to initiate a phase 3 trial of SGN-B6A in patients with previously treated non-small cell lung cancer and to meet our goal of submitting four Investigational New Drug (IND) applications for novel drug candidates in 2023 before year end."

PRODUCTS HIGHLIGHTS

PADCEV

Positive Results for the Phase 3 EV-302 Trial of the Combination of PADCEV and Keytruda as First-Line Treatment for Advanced Urothelial Cancer Presented at the ESMO (Free ESMO Whitepaper) Congress: In October 2023,data from the phase 3 EV-302 trial, which was conducted in collaboration with Merck and our partner Astellas, were presented during the Presidential Symposium at ESMO (Free ESMO Whitepaper) in a late-breaking oral presentation demonstrating a 53% reduction in risk of death with PADCEV and Keytruda compared to chemotherapy in patients with previously untreated locally advanced or metastatic urothelial cancer (la/mUC). The combination improved median overall survival by more than 15 months versus chemotherapy. OS results were consistent across all pre-defined subgroups, including cisplatin eligibility and PD-L1 expression level. The safety results in EV-302 were consistent with those previously reported with this combination in EV-103 in cisplatin-ineligible patients with la/mUC. No new safety signals were identified. An extension study in China continues to enroll patients. The EV-302 trial is intended to serve as the basis for global submissions and as the confirmatory trial for the U.S. accelerated approval of this combination. We intend to submit a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) this year.
Data Presented for Muscle-Invasive Bladder Cancer (MIBC) at ESMO (Free ESMO Whitepaper): In October 2023, data from Cohort L of the EV-103 phase 1b/2 trial evaluating PADCEV monotherapy as perioperative treatment in cisplatin-ineligible patients with MIBC were presented demonstrating promising activity and a generally manageable safety profile.
ADCETRIS

European Commission Approves ADCETRIS in Combination with Chemotherapy in Previously Untreated CD30+ Stage III Hodgkin Lymphoma: In October 2023, the European Commission approved the ADCETRIS (brentuximab vedotin) combination based on updated positive overall survival results from the Phase 3 ECHELON-1 study. ADCETRIS has been previously approved as a therapy for adult patients in Europe in six distinct indications, including those with previously untreated CD30+ Stage IV Hodgkin lymphoma.
Presenting Combination Data in Metastatic Solid Tumors at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting: Initial data of ADCETRIS in combination with Keytruda in metastatic solid tumors will be presented at the SITC (Free SITC Whitepaper) Annual Meeting being held November 3-5, 2023.
TUKYSA

Phase 3 HER2CLIMB-02 Trial Combining TUKYSA and Kadcyla Meets Primary Endpoint of Progression-Free Survival: In August 2023, the Company announced that the phase 3 HER2CLIMB-02 clinical trial of TUKYSA in combination with the antibody-drug conjugate Kadcyla (ado-trastuzumab emtansine) met its primary endpoint of progression-free survival (PFS), including for patients with active brain metastases. Patients in the trial had unresectable locally advanced or metastatic HER2-positive breast cancer and had received previous treatment with a taxane and trastuzumab.​ Overall survival data, a secondary endpoint, are not yet mature. Discontinuations due to adverse events were more common in the combination arm of the trial, but no new safety signals emerged for the combination. Results will be presented at an upcoming meeting.
TIVDAK

Positive Results for Phase 3 innovaTV 301 Clinical Trial Presented at ESMO (Free ESMO Whitepaper): In October 2023, data from the innovaTV 301 trial, conducted in collaboration with our partner Genmab, were presented in an oral session during the Presidential Symposium at ESMO (Free ESMO Whitepaper) that demonstrated a 30% reduction in risk of death compared to chemotherapy and a manageable and tolerable safety profile consistent with the known safety profile as presented in the U.S. prescribing information. No new safety signals were observed. The results from innovaTV 301 are intended to serve as the confirmatory trial for the U.S. accelerated approval and support potential global regulatory applications.
PIPELINE PROGRAMS

Initiated a Phase 3 Trial for Disitamab Vedotin for Patients with HER2-Positive, Metastatic Urothelial Cancer: The Company initiated a phase 3 trial evaluating disitamab vedotin in combination with pembrolizumab versus chemotherapy in patients with previously untreated locally advanced or metastatic HER2-positive urothelial cancer in the third quarter of 2023.
Planning to Initiate Phase 3 Trial for SGN-B6A in Patients with Previously Treated Non-Small Cell Lung Cancer (NSCLC) Before Year End: The Company expects to initiate a phase 3 trial for SGN-B6A, a novel, vedotin ADC targeting integrin beta-6, as a monotherapy in previously treated patients with NSCLC before the end of 2023.
Reported Initial Results for SGN-B7H4V at ESMO (Free ESMO Whitepaper): In October 2023, initial first-in-human clinical data for SGN-B7H4V were presented at ESMO (Free ESMO Whitepaper) demonstrating antitumor activity across a variety of dose levels, dose schedules and tumor types with commonly observed adverse events including peripheral neuropathy, neutropenia, diarrhea, and nausea, consistent with other ADCs. Development remains ongoing in phase 1 dose expansion cohorts in breast, endometrial, ovarian and other B7H4-expressing solid tumors.
Initiated Clinical Trial for Novel Targeted Therapy and Unveiled Novel ADC Program at AACR (Free AACR Whitepaper)-NCI-EORTC Conference: The Company initiated a phase 1 trial for SGN-EGFRd2, a gamma delta bispecific T-cell engager for EGFR-expressing solid tumors. Year to date, the Company has submitted INDs for three novel targeted cancer therapies, including SGN-EGFRd2, SGN-35T and SGN-CEACAM5C, with the goal of submitting one additional IND before year end. Preclinical data for SGN-35T, a next generation CD30-directed ADC with a novel tripeptide drug linker designed to improve the tolerability profile compared with ADCETRIS, were presented at the annual AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October.
For additional information on Seagen’s pipeline, visit www.seagen.com/science/pipeline.

CORPORATE HIGHLIGHTS

Update on Pfizer Acquisition: The European Commission approved the acquisition of Seagen by Pfizer unconditionally pursuant to Article 6(1)b under the EU Merger Regulation on October 19, 2023. The completion of the transaction remains subject to other customary closing conditions, including the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, relating to the consummation of the transaction, and the satisfaction or waiver of the other closing conditions specified in the Merger Agreement. The Company continues to expect that the transaction will be completed in late 2023 or early 2024.
Annual Corporate Responsibility Report Published, Detailing ESG Commitments: Seagen continues to push the boundaries of innovation while upholding its commitment to sustainable practices, as highlighted in the Company’s latest corporate responsibility report, Linking Breakthroughs to Lives. Accomplishments detailed include multiple positive clinical trial results and label expansions across our commercial portfolio, new DEI strategy, publishing our Human Rights Policy and Supplier Code of Conduct, enhancing governance programs across numerous areas including compliance and information security, and new environmental initiatives. Seagen is proud to announce that Newsweek named the Company one of America’s Greenest Companies 2024 and awarded it the highest rating of 5 stars, based on its ESG achievements. The report is available on Seagen’s website at View Source
THIRD QUARTER AND NINE-MONTHS 2023 FINANCIAL RESULTS

Revenues: Total revenues for the third quarter and nine months ended September 30, 2023 were $649 million and $1,772 million, respectively, compared to $510 million and $1,434 million for the same periods in 2022, primarily driven by growth in net product sales.

Revenues included the following components:

Three months ended September 30,

Nine months ended September 30,

(dollars in millions)

2023

2022

% Change

2023

2022

% Change

Total Net Product Sales

$

571

$

428

33

%

$

1,583

$

1,243

27

%

ADCETRIS

$

246

$

219

13

%

$

751

$

601

25

%

PADCEV

$

200

$

105

89

%

$

479

$

329

46

%

TUKYSA

$

102

$

88

16

%

$

289

$

267

8

%

TIVDAK

$

23

$

16

40

%

$

64

$

45

42

%

Royalty Revenues

$

64

$

44

45

%

$

145

$

111

30

%

Collaboration and License
Agreement Revenues

$

14

$

38

(63

)%

$

44

$

80

(45

)%

Note: Sum of product sales may not equal total net product sales due to rounding. Percent change reflects actual (unrounded) values.

Net Product Sales: The increases in net product sales for the third quarter and year-to-date of 2023 compared to the same periods in 2022 were driven by continued commercial execution. ADCETRIS demonstrated year-over-year growth of 13%, primarily attributed to volume growth from greater use in frontline advanced Hodgkin lymphoma, despite recent increased competition in this setting. PADCEV growth was driven by use as first-line treatment for patients with locally advanced or metastatic urothelial cancer who are not eligible to receive cisplatin-containing chemotherapy following its approval for this indication in April 2023. Of note, PADCEV sales in the year-to-date of 2022 included $19 million in sales to another company for a clinical trial they are conducting, while no such sales were booked in the year-to-date of 2023. TUKYSA performance reflects volume growth driven by the important role it serves in the treatment of HER2-positive metastatic breast cancer as well as contributions from its colorectal cancer indication. TIVDAK growth reflects continued uptake in its current indication.
Royalty Revenues: Growth in royalty revenues were primarily driven by royalties from sales of Polivy (polatuzumab vedotin) by Roche, which is an ADC that uses Seagen technology, as well as by sales of ADCETRIS outside the U.S. and Canada by Takeda.
Collaboration and License Agreement Revenues: The decrease in collaboration and license agreement revenues for the third quarter of 2023 was primarily driven by an upfront payment in the prior period and the decrease for the year-to-date also due to a prior period milestone partially offset by collaboration revenue.
Cost of Sales: Cost of sales for the third quarter and year-to-date in 2023 were $165 million and $458 million, respectively, compared to $108 million and $302 million for the same periods in 2022. The increases reflect higher sales of our medicines and the related gross profit share amounts owed to collaboration partners, which were $103 million and $249 million in the third quarter and year-to-date in 2023, respectively, compared to $71 million and $189 million for the same periods in 2022. Cost of sales also reflects amortization of TUKYSA acquired in-process technology costs, third-party royalties owed for PADCEV and TUKYSA net product sales, and cost of products sold. The year-to-date in 2023 cost of sales included a $47 million inventory write-off related to in-process production of one of our products that did not meet a release specification that was updated in June 2023. This inventory adjustment and new release specification are not expected to impact availability of product supply required to meet current or future demand.

Research and Development (R&D) Expenses: R&D expenses for the third quarter and year-to-date in 2023 were $449 million and $1,205 million, respectively, compared to $385 million and $987 million for the same periods in 2022 reflecting continued investment in clinical development of the Company’s approved drugs and pipeline programs.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses for the third quarter and year-to-date in 2023 were $266 million and $746 million, respectively, compared to $210 million and $605 million for the same periods in 2022. The increases in 2023 were driven by ongoing commercialization efforts, as well as $40 million in expenses year-to-date associated with the pending acquisition by Pfizer and other corporate activities.

Non-cash, share-based compensation expense for the nine months ended September 30, 2023 was $288 million, compared to $157 million for the same period in 2022.

Net Loss: Net loss for the third quarter of 2023 was $216 million, or $1.15 per diluted share, and net loss for the year-to-date of 2023 was $602 million, or $3.21 per diluted share.

Net loss for the third quarter of 2022 was $191 million, or $1.03 per diluted share, and net loss for the year-to-date of 2022 was $462 million, or $2.51 per diluted share.

Cash and Investments: As of September 30, 2023, Seagen had $1.2 billion in cash and investments.

CONFERENCE CALL

Given the pending acquisition of Seagen by Pfizer, Seagen is no longer providing financial guidance for 2023 and will not be hosting its quarterly conference call and does not expect to do so for future quarters. Earnings materials are available publicly on the Investor Relations page of our website at investor.seagen.com. Please direct any questions to Seagen Investor Relations at the contact information below.

On November 1, 2023 Schrödinger, Inc. (Nasdaq: SDGR), whose physics-based computational platform is transforming the way therapeutics and materials are discovered, reported financial results for the quarter ended on September 30, 2023 (Press release, Schrodinger, NOV 1, 2023, View Source [SID1234636656]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Schrodinger had an excellent third quarter marked by strong revenue growth and significant pipeline progress. More of our software customers are increasing the scale of their use of our technology, and we remain very confident about the outlook for the full year," said Ramy Farid, Ph.D., chief executive officer of Schrödinger. "We initiated our Phase 1 clinical study of SGR-2921 and our Phase 1 study of SGR-1505 in healthy volunteers is nearing completion. We look forward to sharing more details about our proprietary programs later this year."

Today, Schrodinger announced that the rights to two related oncology discovery programs would revert to the company, after Bristol Myers Squibb elected not to proceed with further development of these programs for strategic reasons.

Third Quarter 2023 GAAP Financial Results
•Total revenue for the third quarter was $42.6 million compared to $37.0 million in the third quarter of 2022.
•Software revenue for the third quarter was $28.9 million compared to $24.7 million in the third quarter of 2022.
•Drug discovery revenue was $13.7 million for the third quarter compared to $12.3 million in the third quarter of 2022.
•Software gross margin was 76% for the third quarter compared to 72% in the third quarter of 2022.
•Operating expenses were $79.8 million for the third quarter compared to $63.4 million for the third quarter of 2022.
•Other expense for the third quarter was $8.7 million compared to other income of $6.5 million in the third quarter of 2022, driven by changes in the fair value of equity investments and interest income.
•Net loss for the third quarter was $62.0 million, compared to $39.9 million in the third quarter of 2022.
•At September 30, 2023, Schrödinger had cash, cash equivalents, restricted cash and marketable securities of approximately $503 million, compared to approximately $456 million at December 31, 2022.

For the three and nine months ended September 30, 2023, Schrödinger reported net losses of $62.0 million and net income of $71.4 million, respectively, compared to net losses of $39.9 million and $122 million for the three and nine months ended September 30, 2022, respectively.

For the three and nine months ended September 30, 2023, Schrödinger reported non-GAAP net losses of $50.4 million and $134.8 million, respectively, compared to non-GAAP net losses of $44.9 million and $117.0 million for the three and nine months ended September 30, 2022, respectively. See "Non-GAAP Information" below and the table at the end of this press release for a reconciliation of non-GAAP net income (loss) to GAAP net income (loss).

2023 Financial Outlook
•Schrödinger today updated its financial guidance for 2023. The company’s financial expectations for the fiscal year ending December 31, 2023 are as follows:
•Software revenue growth is expected to be in the range of 15 percent to 18 percent.
•Drug discovery revenue is expected to range from $50 million to $70 million.
•Software gross margin is expected to be similar to software gross margin for the full year 2022.
•Operating expense growth in 2023 is expected to be significantly lower than operating expense growth in 2022.
•Cash used for operating activities is now expected to be higher in 2023 than 2022, based on the mix of revenue, the timing and size of milestones and expectations for new business development activity this year.
Recent Company Highlights
Wholly-Owned Pipeline
•Schrödinger continues to advance SGR-1505, its investigational MALT1 inhibitor. The Phase 1 dose-escalation study in healthy volunteers is nearing completion, and the company expects to report data from the study in the fourth quarter of 2023. Enrollment in the Phase 1 dose-escalation study in relapsed or refractory B-cell malignancy patients is ongoing in the U.S. and EU. The U.S. Food and Drug Administration recently granted Orphan Drug Designation to SGR-1505 for potential treatment in mantle cell lymphoma.

•Today Schrödinger announced the initiation of the Phase 1 clinical study of SGR-2921, an investigational CDC7 inhibitor, in patients with acute myeloid leukemia or myelodysplastic syndrome. The study is designed to evaluate the safety, pharmacokinetics, pharmacodynamics, and determine the recommended dose. SGR-2921 has exhibited anti-tumor activity as a monotherapy and in combination with standard of care agents in multiple preclinical tumor models.

•Schrödinger continues to advance SGR-3515, an inhibitor of Wee1 and Myt1. Concurrent loss of function of Wee1 and Myt1 confers selective vulnerability in cancer cells, a mechanism referred to as synthetic lethality. IND-enabling activities are ongoing to support an IND submission for SGR-3515 in the first half of 2024.

•Today Schrödinger announced that one of its previously undisclosed discovery programs is PRMT5-MTA (protein arginine methyltransferase 5/methylthioadenosine). PRMT5 has been shown to be a synthetic lethal target for MTAP-deleted cancers with potential roles in the treatment of both hematologic and solid tumors. The company expects to provide more details about its PRMT5-MTA program and other early-stage programs at its Pipeline Day on December 14, 2023.

Schrödinger Collaborators
•In October, Schrödinger collaborator Morphic Holdings presented additional Phase 2a data from the EMERALD-1 trial of MORF-057, an oral ɑ4ꞵ7 inhibitor in development for ulcerative colitis and Crohn’s disease at United European Gastroenterology (UEG) week.

•In September, Structure Therapeutics presented positive results from the Phase Ib multiple ascending dose study of GSBR-1290, an oral GLP-1R, in healthy overweight or obese individuals.

•In September, Schrödinger and the Bill & Melinda Gates Foundation renewed their agreement to invest in the discovery and development of novel non-hormonal contraceptive agents for global health.

Platform

•During the third quarter, Schrödinger and Gates Ventures LLC extended their agreement to develop and apply simulation methods to improve battery performance for a second three-year period. The new research agreement includes total consideration of $6M and runs through August 2026.

•Schrödinger announced quarterly software release 2023-3, which incorporated a number of important updates, including a major enhancement to the company’s Induced Fit Docking (IFD) technology for optimization of certain key ADMET properties, the first full release of technology that can be used to optimize antibody affinity as a function of pH, and technology to more accurately predict small molecule pKa values, a key intrinsic molecular property.

•In August, Schrödinger scientists published the results of a novel automated workflow, FEP Protocol Builder (FEP-PB), which uses active-learning to automate development of accurate FEP+ protocols, increasing the number of targets amenable to the technology.

Third Quarter 2023 Webcast and Conference Call Information
Schrödinger will host a conference call to discuss its third quarter 2023 financial results on Wednesday, November 1, 2023, at 4:30 p.m. ET. The live webcast can be accessed under "News & Events" in the investors section of Schrödinger’s website, View Source The archived webcast will be available on Schrödinger’s website for approximately 90 days following the event.

Schrödinger Pipeline Day Webcast Information
Schrödinger will host its Pipeline Day in New York City on Thursday, December 14, 2023, beginning at 10:00 a.m. ET. Pipeline Day will be a hybrid event, with limited in-person attendance available to members of the investment community, and a simultaneous webcast will be available for individual investors and other interested parties who wish to join virtually. The live presentation can be accessed in the "Investors" section of Schrödinger’s website and will be archived for approximately 90 days. To participate in the live webcast, please register for the event here. It is recommended that participants register at least 15 minutes in advance of the event.

On November 1, 2023 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, reported recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results (Press release, Sangamo Therapeutics, NOV 1, 2023, View Source [SID1234636655]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In 2020, we shared our refreshed company strategy which aims to both maximize the potential of our proprietary genomic editing and delivery technology, and to focus on areas where we believe we can apply that technology to be either first-in-class or best-in-class. The process of streamlining Sangamo’s pipeline has been accelerated within today’s challenging economic environment and we have had to make difficult decisions to defer further investments and seek collaboration partners or direct investment in both our Fabry gene therapy and CAR-Treg cell therapy programs," said Sandy Macrae, Chief Executive Officer of Sangamo. "As we work to unlock value in our clinical programs that is not currently reflected as part of Sangamo, we plan to do everything in our power to get these important assets into the hands of parties with the means to advance them towards patients. At the same time, we will continue to progress our promising epigenetic regulation programs for neurological diseases and hope to soon share a breakthrough in our capsid delivery capabilities, which we believe could open the door for many other high-value and unmet diseases to be addressed with our editing capabilities. We continue to seek ways to raise additional capital to strengthen our financial foundation."

The restructuring announced today represents a further step towards simplifying the Sangamo organization and focusing on our epigenetic regulation therapies treating neurological diseases and our novel AAV capsid delivery technologies. Sangamo is deferring new investments in its Fabry and CAR-Treg programs beyond what is currently committed and is actively seeking collaboration partners or direct investors in both. In addition, Sangamo expects to close its Brisbane, California facility in early 2024 to conserve cash resources, and will transition its headquarters to its Richmond, California facility as of January 1, 2024. As a result of this restructuring, Sangamo is reducing its US workforce by approximately 40%, or approximately 162 roles.

In connection with the restructuring, D. Mark McClung, Executive Vice President, Chief Operating Officer and Jason Fontenot, Senior Vice President, Chief Scientific Officer will be leaving the company on January 2, 2024. In the context of a streamlined and more focused organization, we are eliminating their roles. Until his departure, Mark will continue to lead our search for partners and investors in our Fabry and CAR-Treg programs. Amy Pooler, currently serving as Vice President, Neuroscience and Gregory Davis, currently serving as Vice President, Genome Engineering Design and Technology, have been appointed as Head of Research and Head of Technology, respectively, effective November 17, 2023.

The restructuring and workforce reductions, in combination with other potential cost reductions, are anticipated to reduce our non-GAAP annual operating expenses from approximately $240 million-$260 million in 2023 to approximately $115 million-$135 million in 2024, a decrease of approximately 50%. Sangamo expects to incur approximately $8 million-$10 million in one-time restructuring costs in the fourth quarter of 2023. Sangamo believes its cash, cash equivalents, and marketable securities as of September 30, 2023, in combination with the cost savings expected from the restructuring, workforce reduction and other potential cost reductions, will be sufficient to fund its planned operations into the third quarter of 2024.
Dr. Macrae continued: "I am grateful to all our employees for their commitment to Sangamo’s mission and the patients we seek to serve, and have special appreciation to all those who are leaving for their important contributions. Additionally, I would like to personally thank Mark for the wisdom, candor and leadership he has brought to Sangamo. He has been a trusted colleague and will be greatly missed. I would also like to thank Jason for his dedication, passion and leadership. He leaves a strong scientific legacy for which we will always be grateful."

Recent Business Highlights

Program Highlights

Neurology Epigenetic Regulation Programs – Progressed IND-enabling activities for Nav1.7; presented updated preclinical data at Prion 2023; presented preclinical data on zinc finger activators at the European Society of Gene and Cell Therapy (ESGCT); made significant progress in identifying new, potentially transformative AAV delivery capsids.
•Progressed IND enabling activities for the Nav1.7 program to treat chronic neuropathic pain. Continue to expect an IND submission for this program in 2024.
•Presented data from the prion disease program at the Prion 2023 Conference in October 2023, showing in animal models that Sangamo’s zinc finger repressors significantly reduce expression of the prion protein in the brain, extend lifespan and limit the formation of toxic prion aggregates.
•Presented an oral presentation at ESGCT in October 2023, showing that our zinc finger activators can be designed to restore normal gene and protein expression of SCN2A in vitro and in vivo to potentially address neurodevelopmental disorders such as autism spectrum disorder and intellectual disability.
•Presented data on Shank3 gene activation mediated by zinc finger transcriptional activators as a potential therapeutic approach for Phelan-McDermid Syndrome at ESGCT.
•Continued to advance identification and selection of engineered AAV capsids for enhanced central nervous system delivery through both intrathecal and intravenous delivery. Anticipate sharing nonhuman primate data from our capsid development efforts in early 2024.
Fabry Disease – Dosed total of 25 patients in Phase 1/2 STAAR study; all patients dosed to date continue to demonstrate sustained, elevated α-Gal A levels for up to three years for the longest treated patient; received Regenerative Medicine Advanced Therapy (RMAT) Designation from U.S. FDA; enrolled sufficient patients in the Phase 1/2 study believed to provide a preliminary assessment of safety and efficacy; deferring Phase 3 planning investments and actively seeking partners and investment.
•Dosed three additional patients in the dose expansion phase of the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, our wholly owned gene therapy product for the treatment of Fabry disease, for a total of 25 patients dosed to date, including 14 at the planned Phase 3 dose of 5×1013 vg/kg.
•All patients dosed to date continue to demonstrate sustained, elevated α-Gal A levels, with 12 patients having achieved at least one year of follow-up and the longest treated patient having achieved three years of follow-up.
•All 11 patients who were withdrawn from enzyme replacement therapy (ERT) remain off ERT, for up to 24 months for the longest withdrawn patient.
•Treated patients continue to report improvements in their quality of life, some even over and above the benefits they were experiencing on ERT.
•Received U.S. FDA RMAT designation for isaralgagene civaparvovec, which aims to expedite the review of new therapeutics that are intended to address an unmet need in patients with serious conditions. The U.S. FDA has previously granted isaralgagene civaparvovec both Orphan Drug and Fast Track Designations.
•Stopping further screening and enrollment in the Phase 1/2 STAAR study, after successfully enrolling sufficient patients believed to provide a preliminary assessment of efficacy and safety in the Phase 1/2 study.

•Expect to complete dosing of the remaining enrolled patients in the first half of 2024.
•Anticipate presenting updated Phase 1/2 clinical data at a medical meeting in early 2024.
•Deferring additional investments in Phase 3 planning until collaboration partnership or Phase 3 trial financing is secured.
CAR-Tregs – Received approval for accelerated dosing protocol for Phase 1/2 STEADFAST study from European regulatory authorities; dosed the first patient in the second dose cohort; successfully manufactured product candidate for third and new fourth dose levels; presented updated preclinical data at ESGCT; seeking a collaboration partner or direct investment in CAR-Tregs.
•Dosed the first patient in the second cohort of the Phase 1/2 STEADFAST study evaluating TX200, our wholly owned autologous CAR-Treg cell therapy treating patients receiving an HLA-A2 mismatched kidney from a living donor.
•The product candidate continues to be generally well tolerated in all four patients dosed to date.
•Received all necessary regulatory and ethics approvals for an accelerated dose escalation protocol from European regulatory authorities that could allow dosing to advance more quickly through the cohorts and which allows for a new and highest fourth dose cohort, compared to the three cohorts in the previously approved study protocol. The new, fourth cohort dose will be 18-fold higher than the first cohort starting dose.
•Completed manufacturing of the dose for the patient in the third cohort, who recently received a kidney transplant. Dosing of this fifth patient is expected in the fourth quarter of 2023, pending approval from the Safety Monitoring Committee.
•Completed manufacturing of the dose for the first patient in the fourth and highest dose cohort, who recently received a kidney transplant. Dosing of this sixth patient is expected in January 2024, pending approval from the Safety Monitoring Committee – which would accelerate dosing plans by 18 months compared to the previously approved study protocol.
•Presented preclinical data at ESGCT demonstrating the potential of autologous MOG-CAR-Tregs to provide a long-lasting treatment option for multiple sclerosis and updated animal model data demonstrating the promise of IL23R-CAR-Tregs in the potential treatment of Crohn’s disease.
•Actively seeking a potential collaboration partner or direct external investment in the CAR-Treg cell therapy programs. Expect to provide an update on these efforts in the first quarter of 2024.
•Deferring new investments until a collaboration partner or external investment is secured.
Hemophilia A (Pfizer) – dosing complete in Phase 3 AFFINE trial; pivotal data read-out expected in mid-2024; BLA and MAA submissions anticipated in second half of 2024.
•Pfizer has advised us that dosing is complete in the Phase 3 AFFINE trial of giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A.
•A pivotal readout is expected in mid-2024, with Pfizer anticipating BLA and MAA submissions in the second half of 2024 if the pivotal readout is supportive.
•Expect to present updated data with Pfizer from the Phase 1/2 ALTA study of giroctocogene fitelparvovec in an oral presentation at the 65th American Society for Hematology Annual Meeting and Exposition on December 11, 2023.

Third Quarter 2023 Financial Results

Consolidated net loss for the third quarter ended September 30, 2023 was $104.2 million, or $0.59 per share, compared to a net loss of $53.2 million, or $0.34 per share, for the same period in 2022, primarily due to a non-cash charge relating to impairment of long-lived asset of $44.8 million, which was a result of continued decline in our stock price and related market capitalization, initiation of actions to seek external financing and reprioritize certain research and development programs, and continued decline in equity values in the biotechnology industry.
Revenues
Revenues for the third quarter ended September 30, 2023 were $9.4 million, compared to $26.5 million for the same period in 2022.
The decrease of $17.1 million in revenues was primarily attributed to a decrease of $9.6 million and $9.1 million in revenue relating to our collaboration agreements with Novartis and Biogen, respectively, due to the termination of these collaboration agreements in June 2023, and a decrease of $1.4 million in revenue relating to our collaboration agreement with Kite, reflecting a reduction in collaboration activities during the quarter. These decreases were partially offset by an increase of $3.0 million in revenue relating to our other license agreements.

Total operating expenses on a GAAP basis for the third quarter ended September 30, 2023 were $115.8 million, compared to $81.3 million for the same period in 2022. GAAP operating expenses for the third quarter ended September 30, 2023 included a non-cash charge relating to impairment of long-lived asset of $44.8 million, as described above. Non-GAAP operating expenses, which exclude impairment charges and stock-based compensation expense, for the third quarter ended September 30, 2023 were $64.8 million, compared to $73.5 million for the same period in 2022.
The decrease in total operating expenses on a non-GAAP basis was primarily attributable to lower compensation and other personnel costs mainly due to lower headcount as a result of restructuring of operations and corresponding reduction in workforce announced in April 2023, and decrease in manufacturing and lab supply expenses due to deferral and reprioritization of certain research and development programs. These decreases were partially offset by higher facilities and infrastructure related costs, and higher external expenses as we advance our clinical and preclinical pipeline.
Cash, Cash Equivalents and Marketable Securities
Cash, cash equivalents and marketable securities as of September 30, 2023 were $132.1 million, compared to $307.5 million as of December 31, 2022. As of September 30, 2023, we have raised approximately $15.1 million in net proceeds under our at-the-market offering program since January 1, 2023. We believe that our available cash, cash equivalents and marketable securities as of September 30, 2023, in combination with the cost savings expected from the restructuring, workforce reduction and other potential cost reductions, will be sufficient to fund our planned operations into the third quarter of 2024.
Updated Financial Guidance for 2023
•GAAP operating expenses, including impairment of goodwill, indefinite-lived intangible assets, and long-lived assets, and stock-based compensation expense, for the full year 2023 are now estimated to be in the range of approximately $422 million to $442 million, reflecting the additional non-cash impairment charges recorded in the third quarter. The previous GAAP operating expenses guidance provided on August 8, 2023 was in the range of approximately $378 million to $398 million.
•We continue to estimate non-GAAP operating expenses to be in the range of approximately $240 million to $260 million, which remains unchanged from the last update on April 26, 2023. Estimated non-GAAP operating expenses exclude impairment of goodwill of $38.1 million, impairment of indefinite-lived intangible assets of $51.3 million, impairment of long-lived assets of $65.2 million, and stock-based compensation expense of $28.0 million.
Upcoming Events
Sangamo plans to participate in the following events:
Investor Conferences
•Truist Securities BioPharma Symposium, November 8-9, 2023
•Jefferies London Healthcare Conference, November 14-16, 2023
•EvercoreISI HealthCONx, November 28-30, 2023
Access links for available webcasts for these investor conferences will be available on the Sangamo website in the Investors and Media section under Events. Available materials will be found on the Sangamo website after the event under Presentations.

Conference Call to Discuss Third Quarter 2023 Results

The Sangamo management team will discuss these results on a conference call tomorrow, Thursday, November 2, 2023, at 8:30 a.m. Eastern Time.

Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided or to use the dial-out option to connect their phone instantly.
An updated corporate presentation is available in the Investors and Media section under Presentations.
The link to access the live webcast can also be found on the Sangamo website in the Investors and Media section under Events. A replay will be available following the conference call, accessible at the same link.