On November 1, 2023 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrine, oncology, metabolism and neurology disorders by modulating the effects of the hormone cortisol, reported results for the quarter ended September 30, 2023 (Press release, Corcept Therapeutics, NOV 1, 2023, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-announces-third-quarter-financial-results-2 [SID1234636629]).

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!

Financial Results

Revenue of $123.6 million, a 22 percent increase from third quarter 2022
Increase in 2023 revenue guidance to $470 – $480 million, from $455 – $470 million
Net income per common share of $0.28 (diluted), compared to $0.30 in third quarter 2022
Cash and investments of $414.8 million as of September 30, 2023
"Our strong results in the third quarter reflect returns on our substantial investment in helping physicians to better recognize and treat hypercortisolism. As screening for hypercortisolism (Cushing’s syndrome) becomes more common, the number of patients receiving medical therapy grows. We are confident this trend will continue. Korlym is an excellent treatment for patients with Cushing’s syndrome and there are many eligible patients who have yet to receive it. We are raising our 2023 revenue guidance again, to $470 – $480 million," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer.

Corcept’s third quarter 2023 revenue was $123.6 million, compared to $101.7 million in the third quarter of 2022. Third quarter operating expenses were $92.4 million, compared to $69.8 million in the third quarter of 2022, due to increased clinical trial activity and expenses to support the expansion of our clinical development and commercial teams. Net income was $31.4 million in the third quarter of 2023 compared to $34.6 million in the same period last year.

Cash and investments were $414.8 million at September 30, 2023 compared to $363.3 million at the end of the prior quarter.

Clinical Development

"We are also very excited by the potential of our clinical development programs, with important milestones approaching. In 2024, we expect to report data from our trials in Cushing’s syndrome (the GRACE, GRADIENT and CATALYST studies), ovarian cancer (ROSELLA) and ALS (DAZALS). We also plan to submit an NDA for relacorilant in Cushing’s syndrome and to complete enrollment of our Phase 2b MONARCH study in patients with NASH," added Dr. Belanoff.

Cushing’s Syndrome

GRACE – Phase 3 trial of relacorilant as a treatment for patients with all etiologies of Cushing’s syndrome – enrollment completed; new drug application (NDA) submission expected in the second quarter of 2024
GRADIENT – Phase 3 trial of relacorilant as a treatment for patients with Cushing’s syndrome caused by adrenal adenomas – continues enrollment; results expected in mid-2024
CATALYST – Phase 4 trial examining the prevalence of hypercortisolism in patients with difficult-to-control type 2 diabetes; patients with hypercortisolism may enter a randomized, double-blind, placebo-controlled study of Korlym – continues enrollment; prevalence phase results expected in first quarter of 2024 and full results by year-end 2024
"Relacorilant has tremendous promise as a treatment for patients with Cushing’s syndrome and we are eager to make it available," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "Additionally, our CATALYST trial has the potential to serve as a landmark study to guide physician’s understanding of Cushing’s syndrome. CATALYST is the largest study ever conducted to establish the prevalence of hypercortisolism in patients with difficult-to-control diabetes. We expect CATALYST’s findings to greatly enhance physicians’ ability to diagnose and treat the many patients with Cushing’s syndrome whose condition now frequently goes undiagnosed. We expect data from the prevalence phase of the CATALYST study by early next year."

Oncology

ROSELLA – 360-patient pivotal Phase 3 trial of relacorilant plus nab-paclitaxel in patients with recurrent, platinum-resistant ovarian cancer – continues enrollment; results expected by year-end 2024
Open-label, Phase 1b trial of relacorilant plus pembrolizumab in patients with adrenal cancer with cortisol excess – continues enrollment; results expected in early 2024
Randomized, placebo-controlled, Phase 2 trial of relacorilant plus enzalutamide in patients with prostate cancer – initiated in collaboration with the University of Chicago
"Our Phase 2 trial demonstrated the potential of relacorilant combined with nab-paclitaxel to become a new standard of care for the treatment of patients with platinum-resistant ovarian cancer. The results were published in June in The Journal of Clinical Oncology. Our pivotal ROSELLA trial aims to replicate those results. We expect data by the end of next year," said Dr. Guyer.

Amyotrophic Lateral Sclerosis (ALS)

DAZALS – 198-patient, randomized, double-blind, placebo-controlled, Phase 2 trial of dazucorilant in patients with ALS – continues enrollment; results expected by year-end 2024
"ALS, also known as Lou Gehrig’s disease, is a devastating illness with an urgent need for better treatment. We are conducting our DAZALS study at sites in Europe and the United States to investigate dazucorilant’s potential to significantly improve the lives of patients with ALS. We expect data from this study by the end of next year," said Dr. Guyer.

Non-alcoholic Steatohepatitis (NASH)

MONARCH – 150-patient, randomized, double-blind, placebo-controlled, Phase 2b trial of miricorilant in patients with biopsy-confirmed NASH – initiated in October 2023
"We intend MONARCH to build on the promising results of our Phase 1b study, which demonstrated that miricorilant effectively reduces liver fat, improves liver health and key metabolic and lipid measures and is well-tolerated. Miricorilant has the potential to greatly benefit the millions of patients with NASH. We look forward to sharing our Phase 1b results and more details about MONARCH at a medical conference this fall," said Dr. Guyer.

Conference Call

We will hold a conference call on November 1, 2023, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). Participants must register in advance of the conference call by clicking here. Upon registering, each participant will receive a dial-in number and a unique access PIN. Each access PIN will accommodate one caller. Additionally, a listen-only webcast will be available by clicking here. A replay of the call will be available on the Investors / Events tab of www.corcept.com.

On November 1, 2023 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science to develop a new generation of small-molecule medicines and transform how disease is treated, reported financial results for the third quarter ended September 30, 2023, as well as recent business highlights (Press release, C4 Therapeutics, NOV 1, 2023, View Source [SID1234636627]).

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!

"Our team has progressed three clinical trials this year and generated the necessary information to enable data-based portfolio decisions, which include prioritizing the ongoing Phase 1/2 trials of CFT7455 and CFT1946," said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. "The CFT8634 Phase 1 dose escalation data demonstrated our ability to safely degrade a previously undruggable target, further validating our platform to design BiDAC degraders with desirable drug-like properties. Unfortunately, high levels of BRD9 degradation did not result in sufficient efficacy for highly refractory patients with synovial sarcoma and SMARCB1-null solid tumors treated with CFT8634 as a single agent; thus, the development strategy to seek registration of CFT8634 in these rare tumors is not viable for C4T. On behalf of our entire team, I would like to express my sincere thanks to all patients and their caregivers as well as clinicians involved in the CFT8634 trial."

THIRD QUARTER 2023 AND RECENT ACHIEVEMENTS

CFT7455: CFT7455 is an oral degrader of IKZF1/3 for the potential treatment of relapsed refractory multiple myeloma (R/R MM) and relapsed refractory non-Hodgkin’s lymphomas (R/R NHL).

Progressed the Phase 1/2 Clinical Trial. In October 2023, C4T announced completion of the Phase 1 dose escalation for CFT7455 as a monotherapy in R/R MM using a 14 days on/14 days off dosing schedule. Twenty-two patients were enrolled across five dose escalation cohorts for this portion of the study. The Phase 1 dose escalation evaluating CFT7455 in combination with dexamethasone in R/R MM and as a monotherapy in R/R NHL continues to progress.
CFT8634: CFT8634 is an oral degrader of BRD9 for the potential treatment of synovial sarcoma and SMARCB1-null solid tumors.

Presented Phase 1 Dose Escalation at Connective Tissue Oncology Society (CTOS) Annual Meeting. In the Phase 1 dose escalation trial, CFT8634 was dosed orally starting at 2 mg daily and escalating to 50 mg daily. At the time of the data cutoff on August 29, 2023, 32 patients were enrolled (23 synovial sarcoma and nine SMARCB1-null tumor) and 84% of these patients had more than three prior treatments. All patients were evaluated for safety, the primary endpoint.

The median duration of treatment across all cohorts was 1.8 months (range of 0-11 months). CFT8634 was generally well-tolerated. As of the cutoff date, the majority of adverse events (AEs) reported were considered mild to moderate in severity. The most common treatment-related AEs were (in decreasing frequency) fatigue, dry mouth, neutropenia, dysgeusia and anemia.

Dose proportional increases in plasma exposure were achieved and maintained after single and repeat oral administration, respectively. The calculated half-life is 10 to14 hours after oral administration. Additionally, high levels of BRD9 degradation in tumor tissue obtained at day 15 were noted across all dose levels studied.

At the time of data cutoff, eight patients had stable disease (RECIST 1.1) as best responses at eight weeks. One patient (SMARCB1-null tumor) treated at the 15mg dose had a partial response.
Portfolio Decision Not to Advance CFT8634 Beyond Phase 1 Dose Escalation. C4T has made the portfolio decision not to advance CFT8634 clinical development beyond the Phase 1 trial. In the dose escalation trial, high levels of BRD9 degradation did not result in sufficient efficacy in heavily pre-treated synovial sarcoma and SMARCB1-null solid tumor patients treated with CFT8634 as a single agent. No additional patients will be enrolled in the CFT8634 Phase 1 trial and wind down activities are expected to be complete by the end of Q1 2024.
CFT1946: CFT1946 is an oral degrader targeting BRAF V600 mutations for the potential treatment of solid tumors including non-small cell lung cancer (NSCLC), colorectal cancer (CRC) and melanoma.

Progressed the Phase 1/2 Clinical Trial. The dose escalation portion of the CFT1946 Phase 1/2 clinical trial continues in solid tumors with BRAF V600 mutations, including NSCLC, CRC and melanoma.
Advanced Translational Work with CFT1946 in Preclinical CRC Models. Ongoing translational work in preclinical CRC models encoding BRAF V600X mutations has shown promising single agent CFT1946 anti-tumor effects.
CFT8919: CFT8919 is an oral degrader designed to be potent and selective against EGFR L858R mutations for the potential treatment of non-small cell lung cancer (NSCLC).

Clinical Trial Application (CTA) Accepted for Review by China National Medical Products Administration (NMPA). Betta Pharmaceuticals announced that their CTA submission for CFT8919 has been accepted for review by China NMPA.
COLLABORATION AND RESEARCH UPDATES

Presented at the 6th Annual Targeted Protein Degradation (TPD) Summit. In October 2023, C4T delivered a presentation at the TPD Summit that highlighted the evolution of the TORPEDO platform to include chemoproteomic approaches to identify covalent ligands to both novel targets and E3 ligases.
Betta Pharmaceuticals Stock Purchase Agreement. While both C4T and Betta Pharmaceuticals have met all closing conditions under the Betta Pharmaceuticals Stock Purchase Agreement, including Overseas Direct Investment (ODI) approval, Betta Pharmaceuticals has not fulfilled their obligation to fund the $25 million equity purchase for reasons Betta Pharmaceuticals has attributed to their own business circumstances. C4T and Betta Pharmaceuticals continue to collaborate on the development of CFT8919 under the separate License and Collaboration Agreement.
CORPORATE UPDATES

In September 2023, C4T appointed Kendra Adams as chief financial officer. Ms. Adams has more than twenty-five years of experience in financial, operational and strategic planning.
UPCOMING DATA PRESENTATION

CFT7455: Present data from the Phase 1 dose escalation portion of the ongoing Phase 1/2 clinical trial focusing on CFT7455 as a monotherapy in R/R MM on December 12 at 4:30 PM ET at a virtual company-sponsored event.
UPCOMING INVESTOR EVENTS

November 8, 2023: Management will participate in the virtual BMO Biopharma Spotlight Series Oncology Day.
November 14, 2023 at 3:00 PM ET: Management will participate in a fireside chat at the Stifel 2023 Conference taking place in New York, NY.
THIRD QUARTER 2023 FINANCIAL RESULTS

Revenue: Total revenue for the third quarter of 2023 was $11.1 million, compared to $6.8 million for the third quarter of 2022. The increase in revenue was primarily due to the completion of research activities on a nominated target under the Roche Agreement. Total revenue for the third quarter of 2023 reflects revenue recognized under collaboration agreements with Roche and Biogen, and total revenue recognized in the third quarter of 2022 reflects revenue recognized under collaborations agreements with Roche, Biogen and Calico.

Research and Development (R&D) Expense: R&D expense for the third quarter of 2023 was $28.3 million, compared to $29.7 million for the third quarter of 2022. The reduction in R&D expense was due to a decrease in preclinical expenses as programs progressed through the clinic.

General and Administrative (G&A) Expense: G&A expense for the third quarter of 2023 was $10.5 million, compared to $9.6 million for the third quarter of 2022. The increase in G&A expense was attributable to stock compensation expense.

Net Loss and Net Loss per Share: Net loss for the third quarter of 2023 was $27.0 million, compared to $32.0 million for the third quarter of 2022. Net loss per share for the third quarter of 2023 was $0.55 compared to $0.65 for the third quarter of 2022.

Cash Position and Financial Guidance: Cash, cash equivalents and marketable securities as of September 30, 2023, were $246.4 million, compared to $337.1 million as of December 31, 2022. The decrease in cash was attributable to ongoing operating expenses as well as the early payment of the outstanding principal balance of the term loan held with Perceptive Advisors of $12.5 million. C4T expects that its cash, cash equivalents and marketable securities as of September 30, 2023, will be sufficient to fund planned operating expenses and capital expenditures into the second half of 2025.

On November 1, 2023 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the nine months and third quarter ended September 30, 2023 (Press release, BioMarin, NOV 1, 2023, View Source [SID1234636626]).

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!

"Results in the quarter drove double-digit revenue growth year-over-year and supports BioMarin’s full-year 2023 revenue and profitability objectives, set at the beginning of the year," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin.

"We were very pleased to have recently received FDA approval for VOXZOGO for children under 5 years of age with achondroplasia, making it available in the U.S. for children of all ages with open growth plates. We also recently received approval in Europe to expand VOXZOGO treatment to children aged 4 months and older. These important age expansions will ensure that the youngest children treated with VOXZOGO have the opportunity to experience greater clinical benefit due to a longer potential treatment window. In parallel with exceptional commercial and regulatory execution of VOXZOGO during the quarter, we concluded key reimbursement steps to facilitate ROCTAVIAN treatment in Europe and the U.S. All together, these developments drive our expectation that total BioMarin revenues will approach $3 billion in 2024," said Mr. Bienaimé.

"In Germany, we have tentative agreement with the German Health Insurance Fund on the final ROCTAVIAN price, which we expect to be formalized in the coming weeks. With the final German price expected to be published by year-end, and 60 people in Germany eligible for next steps based on CDx testing results for AAV5 antibodies, we are encouraged by progress made in the quarter. We are also pleased to share that price negotiations with the Italian Medicines Agency are going well and that we expect a final price by year-end," Mr. Bienaimé added, "In the U.S. since gaining FDA approval of ROCTAVIAN on June 29, 2023, the commercial team has been building the reimbursement network to facilitate patient access, setting the stage for meaningful uptake of ROCTAVIAN in 2024."

Third Quarter Financial Highlights:

•Total Revenues for the third quarter of 2023 were $581.3 million, an increase of 15% compared to the same period in 2022. The increase in Total Revenues was primarily attributed to the following:
◦higher VOXZOGO sales volume due to new patients initiating therapy across all regions;
◦higher PALYNZIQ product revenues primarily due to new patients initiating therapy, particularly in U.S.; partially offset by
◦lower ALDURAZYME product revenues primarily driven by the timing of order fulfillment to Sanofi; and
◦lower KUVAN product revenues attributed to increasing generic competition as a result of the loss of exclusivity in the U.S.
•GAAP and Non-GAAP Net Income of $40.4 million and $89.5 million resulted in increases of $47.1 million and $37.5 million for the third quarter of 2023 compared to the same period in 2022, respectively. The increased net income was primarily due to higher gross profit driven by increased revenues, as well as lower income tax expense, partially offset by higher spend in research and development programs to support both early-stage research and clinical activities and higher sales and marketing expenses to support the commercial launch of ROCTAVIAN.
2

Global Commercial Launches of ROCTAVIAN and VOXZOGO
•In the U.S., following the June 29, 2023 FDA approval of ROCTAVIAN, a one-time, gene therapy for the treatment of adults with severe hemophilia A, the company executed a number of critical steps to drive awareness among patients, physicians and patient advocates, as well as prepare Hemophilia Treatment Center (HTC) sites for patient uptake. At the end of the quarter, payers representing more than 205 million U.S. lives had published ROCTAVIAN coverage policies. Warranty policies had been secured by payers representing more than 95 million lives. As reimbursement and HTC readiness become more connected, U.S. patient testing and treatment will be more readily accessible.
•Today in Germany, the second commercial patient was treated with ROCTAVIAN. Over the last several weeks, the company and the German National Association of Statuary Health Insurance Funds (GKV) tentatively agreed on a final ROCTAVIAN price and expect to complete all formalities by year-end. In Germany, 60 people are eligible for next steps ahead of treatment with ROCTAVIAN based on CDx testing to determine seronegativity to AAV5. In Italy, final price negotiations with the Italian Medicines Agency are going well and are expected to be formalized by year-end 2023.
•As a result of global delays securing pricing and reimbursement, and other market preparations for ROCTAVIAN treatment, and proximity to the holiday season, full-year 2023 guidance has been lowered to less than $10 million.
•At the end of September 2023, approximately 2,320 children with achondroplasia were being treated with VOXZOGO across 38 active markets. In the third quarter, patient growth remained strong worldwide. Based on these trends, and the expectation that approximately 2,600 children will be receiving VOXZOGO treatment by year-end, today the company updated full-year 2023 VOXZOGO guidance to between $435 million and $455 million. Additionally, based on increased fill-finish manufacturing commitments, VOXZOGO supply is planned to increase from 2023 levels through the first and second quarters of 2024 and is expected to be fully unconstrained by mid-year 2024.
•On October 24, 2023, the European Commission adopted the decision to expand the indication for VOXZOGO to treat children with achondroplasia aged 4 months and older with open growth plates. VOXZOGO is approved for the treatment of children with achondroplasia of all ages with open growth plates in Japan and the U.S.
VOXZOGO and ROCTAVIAN Market Expansion Opportunities
•In the coming weeks, the company plans to begin the pivotal program with VOXZOGO for the treatment of children with hypochondroplasia, a condition characterized by impaired bone growth. Hypochondroplasia is a genetic statural condition caused by a mutation (gene change) in the fibroblast growth factor receptor-3 (FGFR3) gene. The 6-month observation arm of the study will be followed by the 52-week randomized, double-blind, placebo-controlled phase of the 80-participant clinical trial. If successful, this study is expected to support regulatory approval in this large indication.
•The company is also preparing to initiate two additional clinical programs in 2024 with VOXZOGO, one in idiopathic short stature and one in genetic short stature conditions.
•Additional product expansion opportunities with ROCTAVIAN continue, including a clinical study investigating ROCTAVIAN treatment in those with active or prior inhibitors and continued exploration of methods of administering ROCTAVIAN in people with pre-existing antibodies against AAV5.
Earlier-stage Development Portfolio On-track; Seven Product Candidates Advancing
•BMN 255 for hyperoxaluria in chronic liver disease. The company believes the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in a patient population with significant unmet need. The company expects to have a determination of clinical proof of concept in 2024.
•BMN 331 gene therapy for Hereditary Angioedema (HAE) is in the Phase 1/2 HAERMONY study to evaluate this investigational AAV5-mediated gene therapy for people living with HAE. The company expects to dose additional patients with an optimized corticosteroid regimen with an anticipated clinical proof of concept determination by 2025.
•BMN 351 for Duchenne Muscular Dystrophy (DMD), is an antisense oligonucleotide therapy for individuals with exon 51-skip-amenable DMD. The company is currently enabling a global clinical development plan and expects to have a determination of clinical proof of concept in 2025.
•BMN 349 for alpha-1 antitrypsin deficiency, an orally bioavailable, small molecule that preferentially sequesters mutant protein, preventing polymerization in liver cells that drive the progressive liver disease form of the illness. The company plans to initiate a global clinical program with BMN 349 in 2024 and expects to have a determination of clinical proof of concept in 2025.
•BMN 293 for MYBPC3 hypertrophic cardiomyopathy (HCM). IND enabling studies are underway and have incorporated pre-IND feedback from the FDA. The company plans to initiate a global clinical program with BMN 293 in 2024 and expects to have a determination of clinical proof of concept in 2026.
•BMN 365 for AAV gene therapy for PKP2 arrhythmogenic cardiomyopathy. The company is currently conducting IND-enabling studies and expects to initiate global clinical programs in 2025 with an anticipated clinical proof of concept determination by 2027.
•BMN 355 monoclonal antibody for long-QT syndrome. The company is currently conducting IND-enabling studies and expects to initiate global clinical programs in 2025 with an anticipated clinical proof of concept determination by 2026.

On November 1, 2023 Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, reported recent business highlights including an expanded global partnership with Pierre Fabre Laboratories for tabelecleucel (tab-cel), financial results for the third quarter 2023, and key upcoming catalysts (Press release, Atara Biotherapeutics, NOV 1, 2023, View Source [SID1234636625]).

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!

"We are proud to expand our global tab-cel partnership with Pierre Fabre Laboratories, who is committed to delivering this first-of-its-kind treatment to patients in need across the globe," said Pascal Touchon, President and Chief Executive Officer of Atara. "In light of our expanded tab-cel partnership and to strategically position the company going forward, we are also restructuring our operations to significantly reduce expenses, meaningfully extend our cash runway to nearly two years, and enable organizational focus on generating the greatest value from our transformative pipeline: ATA188 and our differentiated allogeneic CAR-T assets. I wish to personally thank the talented colleagues who will be departing Atara for their essential contributions in getting us to this critical point in our journey."

Expanded Global Partnership for Tabelecleucel (tab-cel or EBVALLO)

Atara has entered into an expanded partnership with Pierre Fabre Laboratories for the U.S. and remaining global commercial markets for tab-cel for up to USD 640 million and significant double-digit tiered royalties on net sales. In addition, Pierre Fabre Laboratories has agreed to reimburse Atara for expected tab-cel global development costs through Biologics License Application (BLA) transfer, and purchase current and future tab-cel inventory through the BLA transfer date. Near-term payments to Atara include:
Approximately USD 30 million in cash upfront and initial inventory purchase at closing
USD 100 million in potential regulatory milestones through BLA approval
Substantially all tab-cel manufacturing, clinical, and regulatory activities are planned to transition from Atara to Pierre Fabre Laboratories at the time of BLA transfer
Atara expects to submit the tab-cel post-transplant lymphoproliferative disease (PTLD) BLA in Q2 2024
"We are eager to progress tabelecleucel toward approval in the U.S. so that American patients can access this innovative treatment already approved and commercialized in Europe," said Eric Ducournau, CEO of Pierre Fabre Laboratories.

The closing of the transaction, subject to expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary closing conditions, is expected to occur in December 2023. PJT Partners served as the exclusive financial advisor to Atara and Fenwick & West LLP served as legal counsel to Atara.

Strategic Restructure and Financial Impacts

Concurrent with the execution of the global tab-cel partnership, Atara is undertaking a strategic restructuring and is reducing its current workforce by approximately 30 percent. This will enable Atara to execute its remaining responsibilities under the tab-cel collaboration with Pierre Fabre Laboratories, while focusing on the advancement of ATA188 and its differentiated allogeneic CAR T (AlloCAR-T) programs
The strategic restructuring, combined with certain anticipated payments from the expanded global partnership and the Company’s existing cash, cash equivalents and short-term investments as of September 30, 2023, is expected to fund the Company’s planned operations into Q3 2025
Pipeline Focus Moving Forward

The ATA188 Phase 2 EMBOLD study primary analysis and communication remains on track for early November with more than 90 patients to be included
To create the greatest value from its potentially transformative pipeline, Atara will focus capital resources on ATA188 development and to unlock the full promise of its growing and potential best-in-class oncology and autoimmune targeted AlloCAR-T portfolio
Atara will leverage its EBV T-cell biology expertise and novel CAR-T technologies for areas of significant unmet medical need by overcoming limitations of current or investigational autologous or allogeneic CAR-T approaches:
Initiation of Phase 1 study in relapsed/refractory B-cell non-Hodgkin’s lymphoma (NHL) for ATA3219—an allogeneic CD19-1XX CAR+ EBV T cell immunotherapy—expected in the coming months with preliminary clinical data anticipated H2 2024
Progressing efforts toward a potential clinical study evaluating ATA3219 in autoimmune disease in parallel with NHL development
Continued advancement of promising early AlloCAR-T development programs including ATA3431, an allogeneic, bispecific tandem CAR directed against both CD19 and CD20 built on the EBV T-cell platform with a 1XX costimulatory signaling domain. ATA3431 preclinical data has been accepted for poster presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December 2023
Third Quarter 2023 Financial Results (prior to Pierre Fabre Laboratories partnership expansion in October 2023)

Cash, cash equivalents and short-term investments as of September 30, 2023, totaled $102.4 million, as compared to $153.6 million as of June 30, 2023
Net cash used in operating activities was $51.3 million for the third quarter 2023, as compared to $65.1 million in the same period in 2022
Atara reported a net loss of $69.8 million, or $0.66 per share for the third quarter 2023, as compared to a net loss of $84.1 million, or $0.82 per share for the same period in 2022.
Total costs and operating expenses include non-cash stock-based compensation, depreciation and amortization expenses of $12.4 million for the third quarter 2023, as compared to $15.4 million for the same period in 2022
Research and development expenses were $56.9 million for the third quarter 2023, as compared to $70.2 million for the same period in 2022
Research and development expenses include $6.8 million of non-cash stock-based compensation expenses for the third quarter 2023 as compared to $8.0 million for the same period in 2022
General and administrative expenses were $12.2 million for the third quarter 2023, as compared to $18.9 million for the same period in 2022
General and administrative expenses include $4.4 million of non-cash stock-based compensation expenses for the third quarter 2023, as compared to $6.0 million for the same period in 2022
Conference Call and Webcast Details

Atara will host a live conference call and webcast today, Wednesday, November 1, 2023, at 9:00 a.m. EDT. Analysts and investors can participate in the conference call by dialing 877-407-8291 for domestic callers and 201-689-8345 for international callers. A live audio webcast can be accessed by visiting the Investors & Media – News & Events section of atarabio.com. An archived replay will be available on the Company’s website for 30 days following the live webcast.

On November 1, 2023, Apellis Pharmaceuticals, Inc., a Delaware corporation (the "Company"), reported to have entered into a Sales Agreement (the "Agreement") with Cowen and Company, LLC, as agent ("Cowen"), pursuant to which the Company may offer and sell shares of its common stock, $0.0001 par value per share (the "Shares"), from time to time through Cowen (the "Offering") (Filing, 8-K, Apellis Pharmaceuticals, NOV 1, 2023, View Source [SID1234636623]). The Company has also filed a prospectus supplement with the Securities and Exchange Commission (the "SEC") in connection with the Offering (the "Prospectus Supplement") under the Company’s existing automatic shelf Registration Statement on Form S-3 (File No. 333-269899), which became effective on February 22, 2023 (the "Registration Statement"). Pursuant to the Prospectus Supplement, the Company may offer and sell Shares having an aggregate offering price of up to $300.0 million.

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!

Upon delivery of a placement notice and subject to the terms and conditions of the Agreement, Cowen may sell the Shares at market prices by any method deemed to be an "at the market offering" as defined in Rule 415(a)(4) promulgated under the Securities Act of 1933, as amended (the "Securities Act"), including sales made directly on or through The Nasdaq Global Select Market ("Nasdaq"), the existing trading market for the Company’s common stock.

The Company or Cowen may suspend or terminate the offering of Shares upon notice to the other party, subject to certain conditions. Cowen will act as sales agent on a commercially reasonable efforts basis consistent with its normal trading and sales practices and applicable state and federal law, rules and regulations and the rules of Nasdaq.

The Company has agreed to pay Cowen commissions for its services of acting as agent of up to 3.0% of the gross proceeds from the sale of the Shares pursuant to the Agreement. The Company has also agreed to provide Cowen with customary indemnification and contribution rights.

A copy of the Agreement is attached as Exhibit 1.1 hereto and is incorporated herein by reference. The foregoing description of the material terms of the Agreement is qualified in its entirety by reference to such exhibit.

Wilmer Cutler Pickering Hale and Dorr LLP, counsel to the Company, has issued a legal opinion relating to the Shares. A copy of such legal opinion, including the consent included therein, is attached as Exhibit 5.1 hereto.

The Shares will be sold pursuant to the Registration Statement, and offerings of the Shares will be made only by means of the Prospectus Supplement. This Current Report on Form 8-K shall not constitute an offer to sell or solicitation of an offer to buy the Shares, nor shall there be any sale of the Shares in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities law of such state or jurisdiction.