On August 3, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, reported its financial results for the quarter ended June 30, 2023 and provided its financial guidance for the year (Press release, Ultragenyx Pharmaceutical, AUG 3, 2023, View Source [SID1234633792]).

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"In the second quarter, our global commercial efforts have resulted in continued meaningful growth of Crysvita, Dojolvi and Mepsevii revenue, including in our key territories outside of the U.S.," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "At the same time, we have continued to advance key clinical programs for osteogenesis imperfecta (OI), Angelman syndrome and Wilson disease, which are expected to generate a number of data catalysts over the next few quarters."

Second Quarter 2023 Selected Financial Data Tables and Financial Results

Revenues (dollars in thousands), (unaudited)
Three Months Ended June 30, Six Months Ended June 30,
2023 2022 2023 2022
Crysvita
Collaboration revenue in profit-share territory $ 19,799 $ 51,609 $ 69,705 $ 96,773
Royalty revenue 29,061 — 29,061 —
Non-cash royalty revenue 17,270 5,423 22,152 10,261
Product sales 16,884 12,402 38,118 21,796
Total Crysvita revenue 83,014 69,434 159,036 128,830
Dojolvi 16,491 13,497 30,794 25,926
Mepsevii 8,439 4,933 16,919 9,794
Evkeeza 365 — 577 —
Daiichi Sankyo — 1,479 1,479 4,728
Total revenues $ 108,309 $ 89,343 $ 208,805 $ 169,278

Total Revenues
Ultragenyx reported $108.3 million in total revenue for the second quarter of 2023, which represents 21% growth compared to the second quarter 2022. This includes second quarter 2023 Crysvita revenue in North America of $61.3 million, which represents 19% growth versus the same time period in 2022. The growth in the region continued on its current trajectory while commercialization responsibilities for Crysvita in North America were transitioned from Ultragenyx to its collaboration partner Kyowa Kirin in April 2023.

Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended June 30, Six Months Ended June 30,
2023 2022 2023 2022
Total revenues $ 108,309 $ 89,343 $ 208,805 $ 169,278
Operating expenses:
Cost of sales 9,914 8,270 22,171 14,370
Research and development 164,949 154,529 330,647 297,684
Selling, general and administrative 81,403 68,137 158,049 135,449
Total operating expense 256,266 230,936 510,867 447,503
Net loss $ (159,828 ) $ (158,162 ) $ (323,800 ) $ (310,482 )
Net loss per share, basic and diluted $ (2.25 ) $ (2.26 ) $ (4.58 ) $ (4.45 )

Operating Expenses
Total operating expenses for the second quarter of 2023 were $256.3 million, including non-cash stock-based compensation of $34.7 million and a $9.0 million milestone expense upon initiation of the UX143 Phase 3 Orbit study. In 2023, annual operating expenses are expected to decrease as the company manages headcount and increases operational leverage while executing on high-value programs.

Net Loss
For the second quarter of 2023, Ultragenyx reported net loss of $159.8 million, or $2.25 per share basic and diluted, compared with a net loss for the second quarter of 2022 of $158.2 million, or $2.26 per share, basic and diluted.

Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were approximately $618.4 million as of June 30, 2023.

2023 Financial Guidance
For the full year 2023, the company expects:

Total revenue in the range of $425 million to $450 million
Crysvita revenue in the range of $325 million to $340 million. This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
Dojolvi revenue in the range of $65 million to $75 million
Net Cash Used in Operations to be around $400 million

Recent Updates and Clinical Milestones

UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Two Phase 3 studies enrolling, additional Phase 2 data expected in mid-October

Positive data from the dose-selection Phase 2 portion of the Phase 2/3 Orbit study were announced in June 2023 and showed that setrusumab rapidly induced bone production in patients with OI. Setrusumab treatment induced statistically significant increases in levels of serum P1NP, a sensitive marker of bone formation, and a substantial and significant improvement in bone mineral density (BMD) by three months. Serum P1NP levels through at least 1 month of treatment were available from all 24 patients enrolled in Orbit and demonstrated that treatment with setrusumab significantly increased serum P1NP, peaking at one to two weeks and again, as expected, after the 2-month dosing timepoint. The Phase 2 Orbit data demonstrated meaningful response in serum P1NP and BMD across both cohorts, with the majority of the effect observed at 20 mg/kg when compared to the 40 mg/kg cohort.

The large increase in BMD observed in the Orbit patient population over the first 3 months was consistent with the rapid increase in serum P1NP levels and was similar to results that took 1 year to achieve in the ASTEROID study in adult OI patients. Treatment with setrusumab for 3 months resulted in an increase in lumbar spine BMD from baseline of 9.4% at 20 mg/kg (n=10), which represents a substantial mean change in Z-score of +0.65 from -2.12 (n=11) at baseline. Treatment with 40 mg/kg (n=7) resulted in a 9.8% BMD increase. Patients on placebo at the 3-month timepoint (n=2) showed no significant change in BMD or change in lumbar spine Z-score.

As of the data cut-off, there had been no treatment-related serious adverse events observed in the study. Reported adverse events were generally consistent with those observed in the ASTEROID study and included infusion associated events, headache and sinusitis. There were no reported hypersensitivity reactions related to setrusumab. There were no safety-related differences observed between dosing groups or age groups.

In July we announced, the first patients were dosed in both of the late-stage clinical trials, Orbit and Cosmic, which evaluate setrusumab in pediatric and young adult patients with OI. Orbit is expected to enroll approximately 195 patients at more than 40 sites across 12 countries. The Phase 3 Cosmic study is an active-controlled study evaluating the effect of setrusumab compared to intravenous bisphosphonate (IV-BP) therapy on annualized total fracture rate in patients aged 2 to <5 years. Cosmic is expected to enroll approximately 65 patients at more than 20 global sites.

Additional data, including fracture frequency information, from the Phase 2 portion of the Orbit study are expected to be shared at an Analyst Day planned for mid-October.

GTX-102 antisense oligonucleotide for Angelman syndrome: Phase 1/2 expansion cohorts enrolling; program update planned for mid-October 2023

In May 2023, the company announced that the FDA reviewed and agreed to a protocol amendment to the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome that enables the harmonization of dose ranges in the U.S. with those being used in ex-U.S. cohorts of the study. The Phase 1/2, open-label, dose-escalating study is evaluating the safety and tolerability of GTX-102 in pediatric patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The study is looking to verify the GTX-102 dose range and treatment regimen that will be used in the Phase 3 program.

As of August 3, 2023, 19 patients have had more than 12 months of exposure to GTX-102, with the longest approaching two years. The dose escalation phase of this study was completed earlier in the year and dosing in the expansion cohorts is ongoing. As of the date of this release, patients from the dose escalation cohorts continue to exhibit encouraging dose and time-dependent clinical activity following longer-term treatment and maintenance dosing. No additional treatment-related serious adverse events or lower extremity weakness adverse events have occurred since the prior update in January 2023.

Globally, sites are enrolling patients in the expansion cohorts and will evaluate the same safety, pharmacokinetic, and efficacy measures as the dose escalating cohorts. Enrollment has accelerated in the last two months as additional sites, including the U.S., have been activated and there are currently more than 20 patients enrolled at sites around the world. No lower extremity weakness safety events have been observed in patients in the expansion cohorts to date.

An interim program update is expected at an Analyst Day in mid-October 2023. In the first half of 2024, the company expects to share data from the dose expansion cohorts on at least 20 patients who have been therapy for at least 6 months, which is expected to provide a meaningful comparison with natural history in this disease.

UX701 AAV gene therapy for Wilson Disease: Stage 1 of pivotal clinical study dosing patients; expect Stage 1 enrollment completion around the end of the year
Dosing in Stage 1 of the pivotal study is ongoing and is expected to enroll five patients per escalating dose cohort. In July 2023, the data safety monitoring board reviewed safety data from Cohort 1 and recommended escalating to the second dose level (1.0 x 10^13 GC/kg dose). Dosing in Cohort 2 has begun and Stage 1 is on track to complete enrollment around the end of the year. Interim Stage 1 data is expected in the first half of 2024 that would include safety and potentially initial signs of clinical activity.

DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Dosing in Phase 3 study complete
In May 2023, Ultragenyx announced the last patient had been dosed in the Phase 3 study. The 48-week study has fully enrolled patients eight years of age and older, randomized 1:1 to DTX401 or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch while maintaining glucose control. Phase 3 data are expected in the first half of 2024.

DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients
Ultragenyx is randomizing and dosing patients in the ongoing Phase 3 study. The pivotal, 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels.

Analyst Day planned for mid-October
The company intends to host an Analyst Day in mid-October to highlight additional Phase 2 Orbit clinical data, including fracture frequency information in patients with OI, and provide program updates on the pipeline, including GTX-102 for Angelman syndrome.

Corporate updates
Howard Horn was appointed as Ultragenyx’s Chief Financial Officer (CFO) and Executive Vice President, Corporate Strategy, effective October 16, 2023. In this role, Howard will be responsible for leading the company’s finance, accounting, corporate strategy, and investor relations functions. He will report to Emil D. Kakkis and will serve on the Executive Leadership Team.

Conference Call and Webcast Information

Ultragenyx will host a conference call today, Thursday, August 3, 2023, at 2 p.m. PT/5 p.m. ET to discuss the second quarter 2023 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call, please register by clicking on the following link (https://register.vevent.com/register/BI0d56bd62c7cc44be8c2fe4ce58f2318c), and you will be provided with dial in details. The replay of the call will be available for one year.

On August 3, 2023 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported financial results for the second quarter ended June 30, 2023 (Press release, Puma Biotechnology, AUG 3, 2023, View Source [SID1234633790]). Unless otherwise stated, all comparisons are for the second quarter of 2023 compared to the second quarter of 2022.

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Product revenue, net consists entirely of sales revenue from NERLYNX, Puma’s first commercial product. Product revenue, net in the second quarter of 2023 was $51.6 million, an increase from $51.3 million reported for the second quarter of 2022. Product revenue, net in the first six months of 2023 was $98.3 million, compared to $92.0 million in the first six months of 2022.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported net income of $2.1 million, or $0.05 per basic and diluted share, for the second quarter of 2023, compared to net income of $9.4 million, or $0.21 per basic and diluted share, for the second quarter of 2022. Net income for the first six months of 2023 was $3.5 million, or $0.08 per basic share and $0.07 per diluted share, compared to net income of $6.0 million, or $0.14 per basic and diluted share, for the first six months of 2022.

Non-GAAP adjusted net income was $4.6 million, or $0.10 per basic and diluted share, for the second quarter of 2023, compared to $12.6 million, or $0.28 per basic and diluted share, for the second quarter of 2022. Non-GAAP adjusted net income for the first six months of 2023 was $8.8 million, or $0.19 per basic and diluted share, compared to $12.3 million, or $0.28 per basic and diluted share, for the first six months of 2022. Non-GAAP adjusted net income excludes stock-based compensation expense. For a reconciliation of GAAP net income to non-GAAP adjusted net income and GAAP net income per share to non-GAAP adjusted net income per share, please see the financial tables at the end of this news release.

Net cash provided by operating activities for the second quarter of 2023 was $3.3 million, compared to net cash used in operating activities of $13.9 million in the second quarter of 2022. Net cash provided by operating activities for the first six months of 2023 was $5.9 million, compared to net cash used in operating activities of $40.8 million in the first six months of 2022. At June 30, 2023, Puma had cash, cash equivalents and marketable securities of $74.4 million, compared to cash, cash equivalents and marketable securities of $81.1 million at December 31, 2022.

"We are pleased to report both positive net income and positive cash flow for the second quarter of 2023," said Alan H. Auerbach, Chairman, Chief Executive Officer, and President of Puma. "In addition, we announced positive biomarker data from the randomized trial of alisertib plus paclitaxel versus paclitaxel alone in hormone receptor positive, HER2-negative breast cancer at the recent 2023 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), which will be helpful as we move forward with the clinical development of alisertib in this indication."

Mr. Auerbach added, "We anticipate the following key milestones over the next 12 months: (i) initiating a Phase II clinical trial of alisertib in small cell lung cancer (H2 2023); (ii) conducting a meeting with the FDA to discuss the clinical development and registration pathway for alisertib in hormone receptor positive, HER2-negative breast cancer (Q4 2023); and (iii) reporting data from an ongoing investigator sponsored Phase I/II trial of alisertib plus pembrolizumab for the treatment of patients with Rb-deficient head and neck squamous cell cancer (H2 2023)."

Revenue

Total revenue consists of product revenue, net from sales of NERLYNX, Puma’s first commercial product, license revenue from Puma’s sub-licensees and royalty revenue. For the second quarter of 2023, total revenue was $54.6 million, of which $51.6 million was net product revenue and $3.0 million was royalty revenue. This compares to total revenue of $59.5 million in the second quarter of 2022, of which $51.3 million was net product revenue and $8.2 million was royalty revenue. For the first six months of 2023, total revenue was $107.3 million, of which $98.3 million was net product revenue and $9.0 million was royalty revenue. This compares to total revenue for the first six months of 2022 of $105.3 million, of which $92.0 million was net product revenue and $13.3 million was royalty revenue.

Operating Costs and Expenses

Total operating costs and expenses were $49.7 million for the second quarter of 2023, compared to $47.4 million for the second quarter of 2022. Operating costs and expenses in the first six months of 2023 were $98.0 million, compared to $94.0 million in the first six months of 2022.

Cost of Sales

Cost of sales was $11.9 million for the second quarter of 2023, compared to $14.9 million for the second quarter of 2022. Cost of sales was $25.1 million for the first six months of 2023, compared to $25.8 million for the first six months of 2022. The decrease was due to lower royalty expense related primarily to the timing of sales made in China by Puma’s sub-licensee, partially offset by increased intangible amortization related to the $12.5 million paid to Pfizer for meeting a commercial sales milestone as of December 31, 2022.

Selling, General and Administrative Expenses

Selling, general and administrative (SG&A) expenses were $24.4 million for the second quarter of 2023, compared to $20.6 million for the second quarter of 2022. SG&A expenses for the first six months of 2023 were $46.8 million, compared to $41.0 million for the first six months of 2022. The $5.8 million year-over-year increase for the first six months resulted primarily from an increase in payroll and related costs of approximately $4.7 million, consisting of approximately $2.5 million due to salary and headcount increases in 2023, as well as a $2.0 million tax credit related to the CARES Act received during the period ended June 30, 2022. In addition, professional fees and expenses increased approximately $0.8 million from the first six months of 2022.

Research and Development Expenses

Research and development (R&D) expenses were $13.4 million for the second quarter of 2023, compared to $11.9 million for the second quarter of 2022. R&D expenses for the first six months of 2023 were $26.1 million, compared to $27.2 million for the first six months of 2022. The $1.1 million year-over-year decrease for the first six months resulted primarily from a decrease in clinical trial expense of approximately $3.6 million, primarily due to the reduction and closure of SUMMIT clinical trial sites, partially offset by an increase in internal R&D of approximately $2.9 million, due primarily to a $1.8 million tax credit related to the CARES Act received during the period ended June 30, 2022, as well as an increase in payroll-related expenses in 2023.

Total Other Income (Expenses)

Total other expenses were $2.6 million for the second quarter of 2023, essentially unchanged from the second quarter of 2022. Total other expenses were $5.4 million for the first six months of 2023, compared to total other expenses of $5.2 million for the first six months of 2022. The $0.2 million year-over-year increase in other expenses for the first six months of 2023 reflects higher interest rates on our outstanding notes as well as imputed interest on a legal settlement, largely offset by increased interest income.

Third Quarter and Full Year 2023 Financial Outlook

Third Quarter 2023

Full Year 2023

Product Revenue, Net

$51 million – $53 million

$205 million – $210 million

Royalty Revenue

$3 million – $5 million

$25 million – $30 million

Net Income

$3 million – $4 million

$20 million – $24 million

Gross to Net Adjustment

17.5% – 18.5%

19% – 20%

Conference Call

Puma Biotechnology will host a conference call to report its second quarter 2023 financial results and provide an update on the Company’s business and outlook at 1:30 p.m. PDT/4:30 p.m. EDT on Thursday, August 3, 2023. The call may be accessed by dialing (877) 709-8150 (domestic) or (201) 689-8354 (international). Please dial in at least 10 minutes in advance and inform the operator that you would like to join the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available shortly after completion of the call and will be archived on Puma’s website for 90 days.

On August 3, 2023 Omega Therapeutics, Inc. (Nasdaq: OMGA) ("Omega"), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, reported financial results for the second quarter ended June 30, 2023, and highlighted recent Company progress (Press release, Omega Therapeutics, AUG 3, 2023, View Source [SID1234633789]).

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"This quarter, we continued to make progress with our clinical development plans and further establish our ability to generate a new class of programmable epigenomic mRNA medicines," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "We continue to enroll patients in our Phase 1/2 first-in-human MYCHELANGELO I study, from which we expect to announce preliminary monotherapy dose-escalation data later this year. In addition to our clinical progress, we presented new preclinical data at the ASCO (Free ASCO Whitepaper) 2023 Annual Meeting that further validates the potential to combine MYC-targeting Omega Epigenomic Controllers (OECs) with checkpoint blockade immunotherapies and advanced multiple programs in preclinical studies. We also welcomed industry veteran Chris Schade to our Board of Directors, whose wealth of biopharma experience will support the long-term growth objectives of our Company. We look forward to building on this foundational work and steady momentum in the second half of this year and beyond."

Recent Corporate Highlights

Development Pipeline and Platform

•
Advanced the MYCHELANGELO I Clinical Trial Evaluating OTX-2002: The Phase 1/2 trial is evaluating OTX-2002, the Company’s lead OEC, as a monotherapy (Part 1) and in combination with standard of care therapies (Part 2) in patients with relapsed or refractory hepatocellular carcinoma (HCC) and other solid tumor types known for association with the c-Myc (MYC) oncogene. Trial enrollment continues to progress as planned at clinical sites across the U.S. and Asia. Preliminary data from the Phase 1 monotherapy dose escalation portion of the study, including initial safety, tolerability, pharmacologic and translational data, are expected in the fourth quarter of 2023.

•
Presented New Preclinical Data Demonstrating the Potential of MYC-targeting OECs to Synergize with Immunotherapies at the ASCO (Free ASCO Whitepaper) 2023 Annual Meeting: Results further validate the OMEGA platform, with MYC-targeting OECs demonstrating consistent anti-tumor activity across multiple tumor types, including HCC and non-small cell lung cancer (NSCLC). MYC OECs modulated the tumor microenvironment, enhanced response to checkpoint blockade immunotherapies and conferred immune memory in preclinical models. These data support Omega’s clinical strategy and planned combination with standard of care, including anti-PD-1 and anti-PD-L1 therapies, in the ongoing Phase 1/2 MYCHELANGELO I clinical trial.

•
Progressed Preclinical Development of Multiple OEC Programs: The Company continues to advance multiple OECs from the OMEGA platform through preclinical studies. OTX-2101, the Company’s development candidate for the treatment of MYC-driven NSCLC, is being evaluated in Investigational New Drug (IND)-enabling studies. Additional preclinical work is ongoing for other OEC development programs, including HNF4a in liver disease and a CXCL 1-8-targeting OEC with potential in multiple indications including neutrophilic asthma, acute respiratory distress syndrome (including COVID-related), oncology, and dermatological and rheumatological indications, representing a potential franchise opportunity.

Corporate

•
Appointed Chris Schade to Board of Directors: Mr. Schade joined the Board on July 10, 2023, and brings over 30 years of experience across the biopharma industry including deep expertise in building companies, strategic planning, financing, and business development. He has proven leadership in several executive roles and as a board member across private and public biopharma companies.

Second Quarter 2023 Financial Results

As of June 30, 2023, the Company had cash, cash equivalents and marketable securities totaling $113.0 million.

Research and development (R&D) expenses for the second quarter of 2023 were $25.0 million, compared to $19.4 million for the second quarter of 2022. The $5.6 million increase in R&D expenses was primarily driven by increases in clinical development costs, external manufacturing costs, and study costs to support the advancement of our programs, as well as facilities and personnel-related expenses, including stock-based compensation to support business growth.

General and administrative (G&A) expenses were $6.2 million for the second quarter of 2023 and 2022.

Net loss for the second quarter of 2023 was $29.7 million, compared to $25.9 million for the second quarter of 2022, driven predominantly by increased R&D expenses to support the Company’s growth.

On August 3, 2023 NGM Biopharmaceuticals, Inc. (NGM Bio) (Nasdaq: NGM), a clinical-stage biotechnology company focused on discovering and developing transformative therapeutics for patients, reported financial results for the quarterly period ended June 30, 2023 and provided business highlights (Press release, NGM Biopharmaceuticals, AUG 3, 2023, View Source [SID1234633788]).

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"We continue to make steady progress in clinical trials of our lead solid tumor oncology programs NGM707, NGM438 and NGM831," said David J. Woodhouse, Ph.D., Chief Executive Officer at NGM Bio. "We also announced positive data in the second quarter from a Phase 2 investigator-sponsored trial of aldafermin for the treatment of patients with IBS-D and BAM, and from a Phase 2b trial of aldafermin in compensated cirrhosis (F4) patients, furthering our belief in the therapeutic potential of aldafermin. These latest data deepen the strong body of clinical evidence we have amassed demonstrating the activity and safety of aldafermin, which we will leverage as we engage in potential partnering discussions for continued development of this product candidate."

Key Second Quarter and Recent Highlights

Aldafermin
•Announced positive data from Phase 2 investigator-sponsored trial of aldafermin for the treatment of patients with diarrhea-predominant IBS-D and BAM in May 2023 at Digestive Disease Week 2023. Aldafermin demonstrated statistically significant reductions in serum 7αC4 (a marker of bile acid synthesis) and fecal bile acids versus placebo in patients with idiopathic BAM with IBS-D.

•Announced positive topline data from the Phase 2b ALPINE 4 trial of aldafermin in 160 patients with compensated cirrhosis due to NASH (liver fibrosis stage 4, or, F4) in May 2023. The study met its primary endpoint with a statistically significant reduction in Enhanced Liver Fibrosis, or ELF, score from baseline to week 48 in patients treated with 3 mg of aldafermin versus patients receiving placebo. Aldafermin was generally well tolerated with no treatment-related serious adverse events and a safety and tolerability profile generally consistent with prior trials of aldafermin.

Solid Tumor Oncology

•Following the initiation of the first two Phase 2b expansion cohorts, continued enrollment in the Phase 1/2 trial evaluating NGM707, an ILT2/ILT4 antagonist antibody product candidate, in combination with KEYTRUDA (pembrolizumab).
•Continued enrollment in the Phase 1/1b trial evaluating NGM438, a LAIR1 antagonist antibody product candidate, as a monotherapy and in combination with pembrolizumab for the treatment of patients with advanced or metastatic solid tumors.

•Continued enrollment in the Phase 1/1b trial evaluating NGM831, an ILT3 antagonist antibody product candidate, as a monotherapy and in combination with pembrolizumab for the treatment of patients with advanced or metastatic solid tumors.
Corporate Updates
•Announced Siobhan Nolan Mangini will step down as President and Chief Financial Officer at NGM Bio. To support a smooth transition, she will stay on until December 1, 2023. NGM Bio has initiated a search to identify the company’s next Chief Financial Officer.

•Substantially completed activities related to the restructuring of NGM Bio’s workforce announced in April 2023. NGM Bio incurred restructuring charges of approximately $5.0 million, the majority of which were paid in the second quarter.
Second Quarter 2023 Financial Results
•NGM Bio reported a net loss of $38.3 million for the quarter ended June 30, 2023, compared to a net loss of $46.5 million for the same period in 2022.
•Related party revenue from our collaboration with Merck Sharp & Dohme LLC, or Merck, was $1.4 million for the quarter ended June 30, 2023 compared to $8.3 million for the same period in 2022. Our related party revenue from Merck will continue to decrease in 2023 and we expect approximately $0.6 million of funding from Merck from July 1, 2023 through March 31, 2024.
•R&D expenses were $32.4 million for the quarter ended June 30, 2023, compared to $45.4 million for the same period in 2022. R&D expenses included restructuring charges of $3.9 million in the three and six months ended June 30, 2023.
•General and administrative expenses were $9.6 million for the quarter ended June 30, 2023, compared to $9.9 million for the same period in 2022. G&A expenses included restructuring charges of $1.1 million in the three and six months ended June 30, 2023.
•Cash, cash equivalents and short-term marketable securities were $193.5 million as of June 30, 2023, compared to $271.5 million as of December 31, 2022. NGM Bio expects its cash, cash equivalents and marketable securities will be sufficient to fund its planned operations into the second quarter of 2025. NGM Bio has based this estimate on plans and assumptions that may prove to be insufficient or inaccurate (for example, with respect to anticipated costs, timing or success of certain activities), and the company could utilize its available financial resources sooner than it currently expects.

On August 3, 2023 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported business updates and preliminary financial results for the six-month period ending June 30, 2023 (Press release, Cellectis, AUG 3, 2023, View Source [SID1234633786]). Full report of the financial results for the second quarter of 2023 will be released in the coming days.

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"We are proud of our team and the strong execution this quarter. Our clinical data presented for UCART22 at the European Hematology Association (EHA) (Free EHA Whitepaper) were positive for patients with r/r B-ALL who have failed multiple lines of treatment including multi-agent chemoimmunotherapy, CD19 directed CAR T-cell therapy, and allogeneic stem cell transplant. We are looking forward to releasing new data later this year on our UCART22 product candidate manufactured in-house," said André Choulika, Ph.D., Chief Executive Officer at Cellectis.

"In addition, we have made progress with our pipeline in 2023 and continue to focus on our core clinical trials BALLI-01 (evaluating UCART22), NATHALI-01 (evaluating UCART20x22) and AMELI-01 (evaluating UCART123). This quarter, Cellectis presented an encore of the clinical data on the AMELI-01 clinical trial at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2023 annual meeting. These preliminary data support the continued administration of UCART123 after FCA lymphodepletion in patients with r/r AML.

Cellectis’ innovation team also presented preclinical data on a gene editing process to develop a bona fide HBB gene correction of sickle cell mutation, and a comprehensive analysis to better design efficient TALE Base Editors (TALE-BE) at the International Society for Cell and Gene Therapy (ISCT) 2023 annual meeting. These achievements showcase once more the power of our gene-editing platform and our TALEN, the technology of choice for therapeutic gene editing, and that we are continuing to deliver constant breakthrough innovation to treat diseases with unmet medical needs.

Despite an unprecedented challenging market environment for cell and gene therapy companies, Cellectis remains focused in its mission to develop innovative cancer therapy product candidates."

Pipeline Highlights

UCART Clinical Development Programs

BALLI-01 (evaluating UCART22) in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)

UCART22 is an allogeneic CAR T-cell product candidate targeting CD22 and is being evaluated in patients with r/r B-ALL in the BALLI-01 Phase 1/2a clinical study.
On June 9, Cellectis presented updated clinical and translational data at the European Hematology Association (EHA) (Free EHA Whitepaper). These data support the preliminary safety and efficacy of UCART22 in a heavily pretreated r/r B-ALL population. UCART22 is currently the most advanced allogeneic CAR T-cell product in development for r/r B-ALL.
In the poster presented at EHA (Free EHA Whitepaper), Cellectis included data from patients who received UCART22 after fludarabine, cyclophosphamide (FC) and FC with alemtuzumab (FCA) lymphodepletion (LD). Compared to the clinical update on BALLI-01 at ASH (Free ASH Whitepaper) 2021, we have included data from six additional patients who received UCART22 at dose level 3 (DL3), as of the December 31, 2022 data cutoff.
UCART22 administered after FC or FCA LD regimen was well tolerated. No dose limiting toxicities (DLTs) nor immune effector cell-associated neurotoxicity syndrome (ICANS) were observed.
For FCA-dose level 3, 50% of the six patients responded. Host lymphocytes remained suppressed through Day 28 for all patients who received FCA LD. Peak ferritin levels correlated with UCART22 cell expansion and cytokine release syndrome (CRS). UCART22 continues to be well tolerated, with no treatment emergent serious adverse events (TESAEs) or DLTs reported. UCART22 cell expansion was detected in 9 of 13 patients in the FCA LD arm and associated with clinical activity.
The BALLI-01 study is currently enrolling patients after FCA LD with Cellectis’ in-house manufactured product. The next data set is expected to be released later this year.
NATHALI-01 (evaluating UCART20x22) in relapsed or refractory B-cell non-Hodgkin lymphoma (r/r B-NHL)

UCART20x22 is Cellectis’ first dual allogeneic CAR T-cell product candidate targeting both CD20 and CD22 and is being evaluated in patients with r/r B-NHL in the NATHALI-01 Phase 1/2a clinical study2.
The NATHALI-01 clinical study is ongoing. Cellectis expects to provide first-in-human data later this year.AMELI-01 (evaluating UCART123) in relapsed or refractory acute myeloid leukemia (r/r AML)
UCART123 is an allogeneic CAR T-cell product candidate targeting CD123 and is being evaluated in patients with r/r AML in the AMELI-01 Phase 1 dose-escalation clinical study.
On May 17, Cellectis presented an encore of the clinical data on the AMELI-01 clinical trial that were unveiled at the ASH (Free ASH Whitepaper) 2022 annual meeting, at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2023 annual meeting. These preliminary data support the continued administration of UCART123 after FCA lymphodepletion in patients with r/r AML.
The oral presentation reviewed preliminary data from patients who received UCART123 at one of the following dose levels: dose level 1 (DL1) 2.5×105 cells/kg; dose level 2 (DL2) 6.25×105 cells/kg; intermediate dose level 2 (DL2i) 1.5×106 cells/kg; or dose level 3 (DL3) 3.30×106 cells/kg after lymphodepletion with FC ([n=8], DL1 – DL3) or FCA ([n=9], DL2 & DL2i).
The data presented showed that adding alemtuzumab to the FC LD regimen was associated with sustained host lymphodepletion and significantly higher UCART123 cell expansion, that correlated with improved anti-tumor activity.
25% (n=2) of patients at DL2 in the FCA arm achieved meaningful response; one patient who failed five prior lines of therapy including allogeneic stem cell transplant experienced a durable minimal residual disease (MRD)-negative complete response that continued beyond 12 months, as of December 2022.
The AMELI-01 study is currently enrolling patients after FCA lymphodepletion in a two-dose regimen arm.
Research Data & Preclinical Programs

UCART20x22

On June 5, 2023, Cellectis presented preclinical data on its product candidate UCART20x22, at the International Society for Cell & Gene Therapy (ISCT) 2023 annual event.
Cellectis provided pre-clinical proof-of-concept data for UCART20x22 to overcome current mechanisms of resistance to CAR T-cell therapies in B-NHL, while providing a potential therapeutic alternative to CD19 targeting and allowing a reduction in the time from treatment decision to infusion.
Cellectis demonstrated that UCART20x22 displays robust activity in vitro and in vivo, against targets expressing heterogeneous levels of CD22 and CD20. We have used in vitro cytotoxicity assays against different tumor cell lines, showing strong activity whether these cells express a single antigen (CD20 or CD22) or both antigens simultaneously, as well as IFNg release in response to antigen specific stimulation.
Article published in Cancer Immunology Research

On May 31, 2023, Cellectis published an article in Cancer Immunology Research demonstrating pre-clinical proof-of-concept data of UCART20x22 product candidate, to overcome current mechanisms of resistance to CAR T-cell therapies in B-NHL.
In this study, we demonstrated that allogeneic CD20x22 CAR T-cells exhibit robust, sustained and dose-dependent activity in vitro and in vivo, while efficiently targeting primary Non-Hodgkin Lymphoma samples with heterogeneous levels of CD20 and CD22.
HBB gene correction of sickle cell mutation

Encouraging preclinical data on gene editing process using Cellectis TALEN technology to develop highly efficient HBB gene correction of sickle cell mutation, were presented in a poster presentation at the ISCT 2023 annual event.
These results showed that non-viral DNA delivery associated with TALEN gene editing reduces the toxicity usually observed with viral DNA delivery and allows high levels of HBB gene correction in long-term repopulating hematopoietic stem cells.
Cellectis leveraged TALEN technology to develop a gene editing process leading to highly efficient HBB gene correction via homology directed repair, while mitigating potential risks associated to HBB gene knock-out. Furthermore, we compared viral (TALEN-V) and non-viral (TALEN-NV) DNA template delivery strategies in mobilized healthy donor (HD) or non-mobilized homozygous sickle patient (HbSS) hematopoietic stem and progenitor cells (HSPCs).
Both strategies led to high and comparable efficiencies of HBB gene correction in vitro in HD and HbSS, without affecting viability, purity or clonogenic potential of corrected HSPCs.
The poster presentation highlighted the following data:

TALEN-mediated engineering efficiently corrects the mutated HBB gene in clinically relevant HSPCs and promote phenotype correction in fully mature RBCs.
Cellectis optimized TALEN gene editing process mitigates potential safety challenges by reducing the frequency of HBB gene inactivation (<10% β-thal cells).
Non-viral DNA template-mediated HBB repair mitigates p53 DNA damage response activation, preserves edited LT-HSCs fitness and enables their efficient engraftment in vivo using an immunodeficient murine model.
TALE Base Editors (TALE-BE)

A comprehensive analysis to better design efficient TALE Base Editors (TALE-BE) using Cellectis’ TALEN technology was presented in a poster at ISCT annual meeting.
Cellectis developed a strategy that allowed to comprehensively characterize editing efficiencies in function of the TC position within the TALE-BE editing windows. This method is specifically taking advantage of the highly precise and efficient TALEN mediated ssODN knock-in in primary T cells, allowing to focus on how target composition and spacer variations can affect TALE-BE activity/efficiency.
The poster presentation highlighted the following data:

Determined optimal spacer length (13/15 bp) for highly efficient TALE-BE for both C40/C40 and C11/C11 scaffolds.
Determined optimal common sequence context for high editing rates.
Determined that editing efficiency of the C11/C11 scaffold is highly dependent on Cytosine position requirements, resulting in more stringent activity in a context of 15 bp spacer size and decreasing the effects of bystander editing.
We believe that the knowledge obtained will allow to better design efficient TALE-BE while improving the specificity profiles of this innovative editing platform.

Novel treatment paradigm for successful CAR T immunotherapy against stroma-rich solid tumors

On May 12, 2023, Cellectis published an article in Frontiers Bioengineering demonstrating the efficacy of its TALEN engineered FAP UCART-cells in cancer-associated fibroblast (CAF) depletion, reduction of desmoplasia and tumor infiltration.
Over 90% of epithelial cancers including breast, colorectal, pancreatic and lung adenocarcinomas express the CAF-specific surface marker, fibroblast activation protein α (FAP), which makes it a promising CAR T-cell target. In this study, Cellectis proposed a novel and versatile approach of combination CAR T-cell therapy that can be extended to most stroma-rich cold tumors with relevant tumor-antigen targeting CAR T-cells which otherwise are recalcitrant to cell therapy.
Preclinical data showed that:

In a mouse xenograft model, successful implantation of injected CAFs in the tumors was confirmed by positive staining of spindle-like cells with human-specific FAP antibody, recapitulating a physiologically relevant TNBC tumor with tumor and stromal compartments.
FAP UCART-cells alone significantly reduced tumor growth.
In vitro and in vivo results show that FAP UCART-cells enable the reprogramming of the cold, stroma-rich triple negative breast cancer (TNBC) TME, making the tumor susceptible to subsequent Meso UCART infiltration and cytotoxicity and improving the overall antitumor activity of the treatment.
In the context of combination therapy with anti-PD1 checkpoint inhibitor, maximal anti-tumor activity and survival benefits were observed upon FAP UCART-cell treatment followed by Meso UCART-cell treatment.
Licensed Allogeneic CAR T-cell Development Programs

Servier and Allogene: anti-CD19 programs

Allogene announced that it is enrolling patients "in the industry’s first potentially pivotal Phase 2 allogeneic CAR T clinical trial with ALLO-501A across sites in the United States and Canada". The European Medicines Agency (EMA) recently approved the ALPHA2 Clinical Trial Application (CTA).
Allogene’s single-arm ALPHA2 trial in relapsed/refractory (R/R) large B cell lymphoma (LBCL) will enroll approximately 100 patients who have received at least two prior lines of therapy and have not received prior anti-CD19 therapy. Allogene has announced that it expects to complete enrollment in 1H 2024 with the first data readout by the end of 2024.
Long-term follow up data from the Phase 1 of Allogene’s ALPHA/ALPHA2 trials in LBCL was presented at both the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting with an encore presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress and International Conference on Malignant Lymphoma (ICML) Lugano in June 2023. The Phase 1 trials enrolled heavily pre-treated patients with a median of three prior lines of therapy. Data from 33 CAR T-naïve LBCL patients receiving Alloy cell product including 12 patients treated with the Phase 2 regimen, are the first to demonstrate the potential for an allogeneic CAR T product to induce complete responses at rates and durability similar to approved autologous therapies.
Allogene: anti-BCMA and anti-CD70 program

The ongoing Phase 1 dose escalation of Allogene’s TRAVERSE study is enrolling patients with advanced or metastatic renal cell carcinoma (RCC) who have progressed on standard therapies including an immune checkpoint inhibitor and a VEGF-targeting therapy.
Allogene’s TRAVERSE trial is now deploying an investigational in vitro companion diagnostic (IVD) assay designed to prospectively assess CD70 expression levels in patients. Allogene has announced that dose escalation in the TRAVERSE trial is expected to be completed in 2023.
Corporate Updates

On June 28, 2023, Cellectis reported results from the annual shareholders’ general meeting held on June 27, 2023, at the Company’s Paris headquarters. At the meeting, during which more than 72% of shares were exercised, Resolutions 1 through 28 were adopted and resolution 29 was rejected, according to the management recommendations. The detailed results of the vote and the resolutions are available on the company’s website: View Source
At the end of the meeting, the terms of office of Ms. Annick Schwebig and Mr. Hervé Hoppenot ended and Ms. Annick Schwebig and Mr. Hervé Hoppenot departed the board of directors as of such date.

During the annual shareholders meeting, Cécile Chartier, Ph.D., was appointed as a director of the Cellectis’ Board of Directors, with immediate effect.

Cécile Chartier currently serves as Chief Scientific Officer at NextVivo, Inc. Prior to her tenure at NextVivo, Dr. Chartier was Vice President of Research at Iovance Biotherapeutics, Inc. where she led the development of next generation tumor-infiltrating lymphocytes (TIL) therapies through research to early-stage clinical trials. Cécile’s extensive experience in the development of next generation cell and gene therapies coupled with her deep knowledge of the U.S. biotechnology industry will be a huge asset to Cellectis.

Financial Results

The interim condensed consolidated financial statements of Cellectis, have been prepared in accordance with International Financial Reporting Standards, as issued by the International Accounting Standards Board ("IFRS").

We present certain financial metrics broken out between our two reportable segments – Therapeutics and Plants – in the appendices of this Q2 2023 financial results press release.

On January 13, 2023, Calyxt, Cibus Global LLC (Cibus) and certain other parties named therein, entered into an Agreement and Plan of Merger (the "Merger Agreement"), pursuant to which, subject to the terms and conditions thereof, Calyxt and Cibus will merge in an all-stock transaction (the "Calyxt Merger"). As a consequence of the foregoing, Calyxt met the "held-for-sale" criteria specified in IFRS 5 and was classified as a discontinued operation until May 31, 2023.

On June 1, 2023, Calyxt and Cibus closed the merger transaction and now operate under the name Cibus, Inc. Consequently, Calyxt was deconsolidated and Calyxt’s cash, cash equivalent and restricted cash are no longer included in the Group’s cash, cash equivalent and restricted cash since June 1, 2023.

Cash: As of June 30, 2023, Cellectis, had $89 million in consolidated cash, cash equivalents, and restricted cash. This compares to $95 million in consolidated cash, cash equivalents and restricted cash as of December 31, 2022. This $6 million difference mainly reflects $55 million of cash out, which include $15 million for R&D suppliers, $7 million for SG&A suppliers, $23 million for staff costs, $7 million for rents and taxes, $3 millions of reimbursement of the "PGE" loan, and a $1 million unfavorable impact on Forex partially offset by a $23 million net cash inflow from the capital raise closed in February, a $21 million net cash inflow from EIB loan, a $1 million cash inflow related to the grant and refundable advance from BPI, $2 millions of financial investments’ capital gain and interests, a $1 million reimbursement of social charges paid on stock options, and a $2 million net cash inflow from licenses and other cash receipts.

Based on the current operating plan, Cellectis anticipates that the cash, cash equivalents, and restricted cash as of June 30, 2023 will fund Cellectis’ operations into the third quarter of 2024.

Revenues and Other Income: Consolidated revenues and other income were $5.6 million for the six months ended June 30, 2023 compared to $6.5 million for the six months ended June 30, 2022. The decrease of $1.0 million reflects the recognition of two milestones related to Cellectis’ agreement with Cytovia for $1.5 million in 2022 and a milestone of $1.0 million with another partner while recognition of revenues in 2023 is not material, and partially offset by the increase of the research tax credit for $0.8 million and the partial recognition of a grant signed with "BPI" of $0.8 million.

R&D Expenses: Consolidated R&D expenses were $43.2 million for the six months ended June 30, 2023, compared to $52.2 million for the six months ended June 30, 2022. The $9.0 million decrease was primarily attributable to (i) a $3.4 million decrease in personal expenses due to departures not replaced (ii) a $4.7 million decrease in purchases, external expenses and other (from $28.0 million in 2022 to $23.2 million in 2023) mainly explained by internalization of our manufacturing and quality activities to support our R&D pipeline and (iii) a $0.8 million decrease of non-cash stock-based compensation expenses (from $3.1 million to $2.3 million).

SG&A Expenses: Consolidated SG&A expenses were $8.9 million for the six months ended June 30, 2023, compared to $10.9 million for the six months ended June 30, 2022. The $2.0 million decrease primarily reflects (i) a $1.6 million decrease in purchases, external expenses and other (from $6.4 million in 2022 to $4.9 million in 2023) mainly explained by the implementation of our ERP in 2022 (ii) a $0.2 million decrease in personal expenses and non-cash stock-based compensation expenses.

Net financial gain (loss): Consolidated net financial gain was $11.6 million for the six months ended June 30, 2023, compared to $9.2 million for the six months ended June 30, 2022. The $2.4 million increase primarily reflects (i) a $20.8 million increase of financial income, mainly attributable to the profit from Calyxt’s deconsolidation, partially offset by (ii) the loss in fair value on our retained investment in Calyxt since deconsolidation for $10.2 million, (iii) a $6.8 million decrease in the fair value of Cytovia’s note receivable.

Net income (loss) from discontinued operations: Pursuant to Calyxt deconsolidation income from discontinued operation for the six-month period ended June 30, 2023, 2023 only include five months of activity. The $3.5 million increase of net loss from discontinued operations between the six-month period ended June 30, 2022 and 2023 is primarily driven by (i) the increase of $9.2 million of net financial loss and (ii) the increase of $1.5 million of other operating expenses partially offset by (i) the decrease of $2.8 million of R&D expenses (from $6.3 million in 2022 to $3.5 in 2023) and (ii) the decrease of $4.5 million of SG&A expenses (from $6.8 million in 2022 to $2.3 million in 2023).

Net Income (loss) Attributable to Shareholders of Cellectis: The consolidated net loss attributable to shareholders of Cellectis was $40.7 million (or $0.76 per share) for the six months ended June 30, 2023, of which $35.7 million was attributed to Cellectis continuing operations, compared to $50.9 million (or $1.12 per share) for the six months ended June 30, 2022, of which $47.3 million was attributed to Cellectis continuing operations. This $10.1 million decrease in net loss between the first six months of 2023 and 2022 was primarily driven by (i) a $9.0 million decrease of R&D expenses, (ii) a $2.0 million decrease of SG&A expenses and (iii) an increase of $2.4 million of the financial gain due to the deconsolidation of Calyxt compensated in part by the decrease of fair value of Cytovia’s note receivable. These downward impacts on the net loss were partially offset by (i) a decrease of $1.0 million of revenues and other income, (ii) an increase of $1.5 million of loss from discontinued operations attributable to Shareholders of Cellectis.

Adjusted Net Income (Loss) Attributable to Shareholders of Cellectis: The consolidated adjusted net loss attributable to shareholders of Cellectis was $36.7 million (or $0.68 per share) for the six months ended June 30, 2023, compared to a net loss of $45.5 million (or $1.00 per share) for the six months ended June 30, 2022.

Please see "Note Regarding Use of Non-IFRS Financial Measures" for reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to adjusted net income (loss) attributable to shareholders of Cellectis.

We currently foresee focusing our cash spending at Cellectis for 2023 in the following areas:

Supporting the development of our pipeline of product candidates, including the manufacturing and clinical trial expenses of UCART123, UCART22, UCART 20×22 and potential new product candidates;
Operating our state-of-the-art manufacturing capabilities in Paris (France), and Raleigh (North Carolina, USA); and
Continuing to strengthen our manufacturing and clinical departments.
The selected, preliminary financial information set forth above are unaudited and should be considered preliminary and subject to change. We have provided such selected, preliminary results above as our final results remain subject to the completion of our normal closing procedures, final adjustments, developments that may arise between now and the time the financial results are finalized, and management’s and the audit and finances committee’s final reviews. Accordingly, you should not place undue reliance on this preliminary information, which may differ materially from our actual final results. These preliminary results should not be viewed as a substitute for our full quarterly financial statements prepared in accordance with IFRS. In addition, they are not necessarily indicative of the results to be achieved in any future period. These preliminary results have been prepared by and are the responsibility of management. Our independent registered public accounting firm has not audited, compiled, performed any procedures on or revised the preliminary financial information, and accordingly does not express an opinion or any other form of assurance with respect of the preliminary information. We plan to report our full results for the second quarter in the coming days.