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Repare Therapeutics Provides Business Update and Reports Third Quarter 2023 Financial Results

On November 9, 2023 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported financial results for the third quarter ended September 30, 2023 (Press release, Repare Therapeutics, NOV 9, 2023, View Source [SID1234637418]).

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"We substantially advanced our pipeline during the third quarter, particularly our Phase 1 MYTHIC trial evaluating lunresertib as a monotherapy and in combination with camonsertib. The initial data that was presented in a plenary session at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in October 2023 showed early efficacy signals across multiple tumor types and in each genotype selected, most notably in gynecological tumors, along with a favorable safety and tolerability profile," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "Additionally, we look forward to hosting an investor event focused on our preclinical programs, RP-1664 and RP-3467, next week, on November 15th, where we will showcase the strength of our growing pipeline."

Third Quarter 2023 Review and Operational Updates:

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Advancing lunresertib (RP-6306), a first-in-class, oral PKMYT1 inhibitor, for the treatment of molecularly selected advanced solid tumors.
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Presented initial positive data from Modules 1 and 2 of its ongoing Phase 1 MYTHIC trial evaluating lunresertib alone and in combination with camonsertib in patients with advanced solid tumors harboring CCNE1 amplification or FBXW7 or PPP2R1A deleterious alternations (NCT04855656) at the 2023 AACR (Free AACR Whitepaper)-NCI-EORTC International Conference and additional data from a later cut-off date in a virtual webcast event hosted by Repare.
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As of the September 5, 2023 data cut-off date as presented during the company virtual webcast event, the Company reported that 67 patients were enrolled in Module 1 (monotherapy) and 59 patients in Module 2 (combination therapy).
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In the Module 2 cohort at the combination preliminary recommended Phase 2 dose (RP2D):
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Protocol-defined overall response (OR) (RECIST or GCIG CA-125 responses) was observed to be 33.3% (N=18). Clinical benefit rate (CBR) (overall response or stable disease of at least 16 weeks without tumor progression) was 50.0%.
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In the cohort of patients with gynecologic tumors, the RECIST response was 50%, OR was 60%, and CBR was 70%. These patients also had a median of 3 and up to 9 prior lines of therapy, before administration of lunresertib.

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In all evaluable patients in the trial, across all doses (N=55), OR was 23.6% and CBR was 41.8%.
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RECIST responses in this ongoing combination trial included 8 confirmed and 3 unconfirmed partial responses (PR). Additionally, 3 patients with ovarian tumors had cancer antigen 125 (CA-125) responses.
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Encouraging and highly manageable safety and tolerability was observed for the combination therapy arm of the trial (N=59). The most common treatment-related adverse event (TRAE) was anemia, with Grade 3 occurring in 42% of patients enrolled in the trial:
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35% of patients did not develop anemia at the preliminary RP2D. Generally, those with Grade 3 anemia had the lowest hemoglobin values at the time of trial enrollment, were intensely pretreated with greater than 4 prior therapies and were of advanced age.
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The anemia reported by patients in the trial usually improved after a one-week treatment interruption and standard supportive care, and did not lead to any therapy discontinuations for patients who received treatment at the preliminary RP2D.
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There were no Grade 4 or Grade 5 TRAEs reported at the preliminary RP2D.
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This data indicates that anemia management can be individualized and alleviated with simple patient monitoring. This approach is now being tested in the expansion cohorts of the MYTHIC trial.
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Repare expects to report additional combination therapy data from the expansion cohorts of the MYTHIC trial in the second half of 2024.
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Repare expects to report initial data from its ongoing Phase 1 MINOTAUR trial evaluating lunresertib in combination with FOLFIRI (NCT05147350) in the first half of 2024. Additionally, the Company expects to report initial updated data from its ongoing Phase 1 MAGNETIC trial evaluating lunresertib in combination with gemcitabine (NCT05147272) in the second half of 2024.
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Repare is collaborating with Princess Margaret Cancer Center to initiate clinical testing, as part of an investigator-sponsored trial (IST), of a fourth lunresertib combination with carboplatin and paclitaxel for the treatment of recurrent gynecological malignancies, with first patient dosing expected to take place by the end of this year.
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Repare is also collaborating with the Canadian Cancer Trials Group in an ongoing basket Phase 2 IST that is enrolling patients with selected, advanced cancers receiving lunresertib as combination with gemcitabine (NCT05605509), and in a second active trial that will evaluate lunresertib in combination with gemcitabine in patients with CDK4/6 inhibitor treated ER+/HER2- metastatic breast cancer (NCT05601440).
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Advancing camonsertib (RP-3500 / RG6526), a potent and selective oral small molecule inhibitor of ATR (Ataxia-Telangiectasia and Rad3-related protein kinase) for the treatment of tumors with specific synthetic lethal genomic alterations in partnership with Roche.
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Roche has included a camonsertib-based arm in its Phase 2, global, multicenter, open-label, multi-cohort TAPISTRY trial (NCT04589845) and its Phase 1/2 study of multiple immunotherapy-based treatment combinations in participants with metastatic non-small cell lung cancer (Morpheus Lung; NCT03337698). Repare is eligible to receive a milestone payment of $40 million upon enrollment of the first patient with camonsertib in the TAPISTRY trial, which is expected by year-end, and could be eligible for an additional $15 million milestone payment if this study becomes registrational.
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Repare is continuing to conduct dose optimization and efficacy assessments in tumor specific expansions in the ATTACC clinical trial in collaboration with Roche to support future clinical development plans for camonsertib combinations with PARP inhibitors.
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Repare also presented clinical and preclinical data from its ongoing Phase 1b TRESR clinical trial evaluating camonsertib in combination with gemcitabine in patients with solid tumors with ATR inhibitor sensitizing mutations at the AACR (Free AACR Whitepaper)-NCI-EROTC conference. The latest data cut from the trial continues to show the benefits of combination therapy, which has led to anti-tumor activity in heavily pretreated patients, including 7 patients (N=28) with confirmed or unconfirmed PRs per RECIST, or GCIG CA-125 responses (N=28), with responses observed primarily in patients with gynecologic cancers. Overall molecular response rate (MRR) of 57%, along with 82% decrease in circulating tumor DNA (ctDNA). The combination therapy was found to be safe and well-tolerated to date, with no drug-drug interactions observed. Efficacy assessment is ongoing at the proposed RP2D in patients with ovarian cancer.
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Advancing preclinical programs into clinical development.
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RP-1664 IND-enabling studies, which began in the first quarter of 2023, are nearing completion and Repare expects to initiate a clinical trial in the first half of 2024.
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RP-3467 is Repare’s wholly-owned Polθ inhibitor, currently in IND-enabling studies, which began in the second quarter of 2023 and remain ongoing. Repare expects to initiate a clinical trial in the second half of 2024.
Third Quarter 2023 Financial Results:

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Cash and cash equivalents and marketable securities: Cash and cash equivalents and marketable securities as of September 30, 2023 were $250.1 million, which Repare believes will be sufficient to fund its planned operations into 2026.
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Revenue from collaboration agreements: Revenue from collaboration agreements were $2.2 million and $38.1 million for the three and nine months ended September 30, 2023, respectively, as compared to $112.5 million and $113.6 million for the three and nine months ended September 30, 2022. The decrease in revenue for the three- and nine-month periods were primarily due to a decrease in revenue recognized under the Roche collaboration mainly as a result of the $108.0 million revenue recognized in the third quarter of 2022 pursuant to the satisfaction of our performance obligations for the issuance of the combined licenses and the clinical trial materials transferred. The decrease in the nine-month period was partially offset by higher deferred revenue recognized from the Roche collaboration, the BMS collaboration and the Ono collaboration.
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Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $32.7 million and $98.3 million for the three and nine months ended September 30, 2023, respectively, as compared to $31.2 million and $89.2 million for the three and nine months ended September 30, 2022. The increase in Net R&D expenses for the three- and nine-month periods were primarily due to higher personnel-related costs and direct external costs related to the progress of our lunresertib clinical program, as well as the advancement of preclinical programs into IND-enabling studies.
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General and administrative (G&A) expenses: G&A expenses were $7.9 million and $25.1 million for the three and nine months ended September 30, 2023, respectively, compared to $7.9 million and $24.6 million for the three and nine months ended September 30, 2022. The increase in G&A was primarily due to higher personnel related costs, offset by lower D&O insurance premiums and lower professional fees.
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Net income (loss): Net loss was $18.9 million, or $0.45 per share, and $65.8 million, or $1.56 per share, in the three and nine months ended September 30, 2023, respectively, and net income was $75.5 million, or $1.71 per share, and $2.6 million, or $0.06 per share, in the three and nine months ended September 30, 2022, respectively.

Rain Oncology Reports Third Quarter 2023 Financial Results and Provides Business Update

On November 9, 2023 Rain Oncology Inc. (NasdaqGS: RAIN), (Rain), reported financial results for the third quarter ended September 30, 2023, along with an update on the Company’s key developments and business operations (Press release, Rain Therapeutics, NOV 9, 2023, View Source [SID1234637417]).

"Rain continues to evaluate a number of strategic opportunities to add value for its stockholders," said Avanish Vellanki, co-founder and chief executive officer of Rain. "We anticipate being able to provide a public update on our efforts before the end of the year."

Third Quarter 2023 Financial Highlights

For the three and nine months ended September 30, 2023, Rain reported a net loss of $7.0 million and $49.6 million, respectively, as compared to a net loss of $18.0 million and $53.0 million for the same periods in 2022, respectively.

Research and development (R&D) expenses were $4.0 million and $35.6 million for the three and nine months ended September 30, 2023, respectively, as compared to $14.5 million and $42.3 million for the same periods in 2022, respectively. The decrease in R&D expenses was primarily related to the winding down of the MANTRA and MANTRA-2 clinical trials for milademetan and lower payroll-related costs for the Company’s R&D personnel due to the reduction-in-force implemented in the second quarter of 2023.

General and administrative (G&A) expenses were $4.1 million and $14.6 million for the three and nine months ended September 30, 2023, respectively, as compared to $3.9 million and $11.3 million for the same periods in 2022, respectively. The increase in G&A expenses was primarily due to higher professional services, legal and payroll-related costs.

Total non-cash stock-based compensation expenses were approximately $1.2 million and $3.6 million for the three and nine months ended September 30, 2023, respectively, as compared to $0.9 million and $3.6 million for the same periods in 2022, respectively.

As of September 30, 2023, Rain had $77.3 million in cash, cash equivalents and short-term investments.

As of November 3, 2023, Rain had approximately 36.4 million shares of common stock outstanding.

Precigen Reports Third Quarter 2023 Financial Results and Progress of Clinical Programs

On November 9, 2023 Precigen, Inc. (Nasdaq: PGEN), a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, reported third quarter 2023 financial results and progress of clinical programs (Press release, Precigen, NOV 9, 2023, View Source [SID1234637416]).

"Precigen has made tremendous progress in reducing our operating costs and we are actively reprioritizing our programs to enable commercial readiness for our lead asset, PRGN-2012. We anticipate completing the PRGN-2012 Phase 2 study in the second quarter of 2024 and, given the FDA’s guidance in August 2023 that the ongoing Phase 1/2 study of PRGN-2012 will serve as the pivotal study to support an accelerated approval request, we are working to expedite the submission of a BLA as quickly as possible," said Helen Sabzevari, PhD, President and CEO of Precigen. "We have recently published exciting new data for both of the Company’s core platforms, AdenoVerse and UltraCAR-T, including presentations at the ESGCT 30th Annual Congress for PRGN-3007 UltraCAR-T and PRGN-2012 AdenoVerse immunotherapy, and publication of a manuscript in Science Translational Medicine that includes full Phase 1 data from the PRGN-2012 clinical study. Each publication builds the body of clinical evidence for the potential of our innovative therapeutic platforms in meeting unmet medical needs for patients."

Program Highlights

PRGN-2012 AdenoVerse Immunotherapy in RRP

· PRGN-2012 is an investigational off-the-shelf AdenoVerse immunotherapy designed to elicit immune responses directed against cells infected with human papillomavirus (HPV) 6 or HPV 11 for the treatment of recurrent respiratory papillomatosis (RRP). The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation and Orphan Drug Designation for PRGN-2012 for the treatment of RRP.

· The Company announced that the FDA has agreed that the ongoing Phase 1/2 (NCT04724980) single-arm study will serve as pivotal for the purpose of filing an accelerated approval request for licensure. Based on this FDA guidance, the Company plans to initiate a confirmatory study prior to submission of the biologics license application (BLA).

· The Company presented positive Phase 1 data showing clinical benefit and enhanced T-cell responses with repeated administration from the ongoing Phase 1/2 single-arm study at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress in an oral presentation (Abstract# OR04) titled, "Significant clinical benefit and enhanced T-cell responses with repeated administration of PRGN-2012, a novel gorilla adenoviral vector based immunotherapy, in adult patients with severe recurrent respiratory papillomatosis."

o The presentation included full results of the Phase 1 study and add to the previously presented data for PRGN-2012 which showed significant response in RRP patients with 50% of patients in Complete Response, requiring no post-treatment surgeries, following PRGN-2012 treatment at Dose Level 2 with a favorable safety profile, no dose-limiting toxicities and no treatment-related adverse events (TRAEs) greater than Grade 2.

o Complete Responses are durable and all complete responders remain surgery-free (follow-up range: 18-24 months) after PRGN-2012 treatment completion.

o PRGN-2012 treatment induced robust HPV-specific T cell responses which were correlated with clinical responses in the study.

· Full results of the Phase 1 portion of the ongoing Phase 1/2 study of PRGN-2012 were published in the peer-reviewed journal, Science Translational Medicine, a leading publication from the American Association for the Advancement of Science (AAAS), in a manuscript titled, "The tumor microenvironment state associates with response to HPV therapeutic vaccination in patients with respiratory papillomatosis."

· Enrollment and dosing in the Phase 2 portion of the study (N=23) is complete bringing the total number of enrolled patients to 35 at Dose Level 2. Patient follow up is currently ongoing and the Phase 2 study is expected to be complete by the second quarter of 2024.

PRGN 2009 AdenoVerse Immunotherapy in HPV-associated Cancers

· PRGN-2009 is an investigational off-the-shelf AdenoVerse immunotherapy designed to activate the immune system to recognize and target HPV-positive solid tumors.

· The Company completed the Phase 1 (NCT04432597) study and presented positive Phase 1 clinical data from the monotherapy and combination therapy arms in patients with recurrent or metastatic HPV-associated cancers at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

· Enrollment was completed in the Phase 2 monotherapy arm with 20 evaluable patients in newly diagnosed oropharyngeal squamous cell carcinoma (OPSCC) patients. The Phase 2 (NCT05996523) combination arm (PRGN-2009 in combination with pembrolizumab) in OPSCC is enrolling patients.

· The Company plans to initiate a Phase 2 randomized, open-label, two-arm study of PRGN-2009 in combination with pembrolizumab in patients with recurrent or metastatic cervical cancer.

PRGN-3006 UltraCAR-T in AML

· PRGN-3006 is an investigational multigenic, autologous chimeric antigen receptor T (CAR-T) cell therapy engineered to express a CAR specifically targeting CD33, membrane bound IL-15 (mbIL15), and a kill switch. The FDA granted Orphan Drug Designation and Fast Track Designation for PRGN-3006 UltraCAR-T for patients with relapsed or refractory acute myeloid leukemia (AML).

· The Company completed the Phase 1 (NCT03927261) dose escalation study and presented positive data at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

· The Phase 1b dose expansion study of PRGN-3006 is ongoing and an interim clinical data presentation is expected in 2024.

PRGN-3005 UltraCAR-T in Ovarian Cancer

· PRGN-3005 UltraCAR-T is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR specifically targeting the unshed portion of MUC16, mbIL15, and a kill switch.

· The Company completed the Phase 1 (NCT03907527) dose escalation cohorts of the intraperitoneal (IP) and intravenous (IV) arms without lymphodepletion as well as in the lymphodepletion cohort in the IV arm and presented positive Phase 1 clinical data in patients with advanced platinum resistant ovarian cancer at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting.

· As previously communicated, based on portfolio reprioritization efforts, the Company will not add an extensive number of new sites this year. Instead, a new site will be activated under the Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) to continue the advancement of the PRGN-3005 Phase 1b dose expansion study without incurring major clinical/contract research organization (CRO) costs.

PRGN-3007 UltraCAR-T in Advanced ROR1+ Hematological and Solid Tumors

· PRGN-3007, based on the next generation of the UltraCAR-T platform, is an investigational multigenic, autologous CAR-T cell therapy engineered to express a CAR targeting receptor tyrosine kinase-like orphan
receptor 1 (ROR1), mbIL15, a kill switch, and a novel mechanism for the intrinsic blockade of PD-1 gene expression.

· The Phase 1 dose escalation portion of the Phase 1/1b study is ongoing. The target patient population for the Phase 1/1b study includes ROR1+ advanced hematological and solid tumors.

· The Company presented additional preclinical data (Abstract# P469) for PRGN-3007 at the ESGCT 30th Annual Congress in a poster presentation titled, "Overnight-manufactured UltraCAR-T cells with first-in-class miRNA-based PD1 blockade demonstrates enhanced polyfunctionality and sustained cytotoxicity against hematological and solid tumors."

Financial Highlights

· Cash, cash equivalents, short-term and long-term investments totaled $79.0 million as of September 30, 2023.

· Selling, general, and administrative (SG&A) costs decreased versus the prior year, by 9% and 17% for the three and nine months ended September 30, 2023, respectively.

"Following our portfolio reprioritization and other cost-saving measures announced last quarter, Precigen continues to manage the balance sheet to enable rapid progression of our lead assets," said Harry Thomasian Jr., CFO of Precigen. "As we scale-up areas of our business to prepare for commercialization, we are focused on fiscal management and exploring new non-dilutive capital opportunities, including potential strategic partnerships, to maximize and extend our runway."

Third Quarter 2023 Financial Results Compared to Prior Year Period

Research and development expenses decreased $1.0 million, or 8%, compared to the three months ended September 30, 2022. This decrease was primarily due to continued reprioritization of clinical product candidates.

SG&A expenses decreased $0.9 million, or 9%, compared to the three months ended September 30, 2022. This decrease was primarily driven by a reduction in professional fees of $0.6 million, due to decreased legal fees associated with certain litigation matters, and $0.3 million in decreased insurance related expenses.

Total revenues decreased $15.3 million, or 92%, compared to the three months ended September 30, 2022. Collaboration and licensing revenues decreased $14.6 million, or 100%, compared to the three months ended September 30, 2022, primarily due to the prior year period non-cash recognition of revenue related to historical collaboration agreements for which revenue was previously deferred. Product and service revenues decreased $0.7 million, or 34%, compared to the three months ended September 30, 2022. This decrease is related to reductions in services performed at Exemplar.

Total other income, net, increased $2.1 million compared to the three months ended September 30, 2022. This is primarily due to $2.0 million in reduced interest expense associated with the Company’s Convertible Notes as they were fully retired in the second quarter of 2023, and $0.8 million increased interest income due to higher interest rates on investments. This increase was offset by a $0.9 million gain recorded on the early retirement of a portion of the Convertible Notes in the third quarter of 2022 that did not occur in the third quarter of 2023.

Loss from continuing operations was $19.8 million, or $(0.08) per basic and diluted share, compared to loss from continuing operations of $7.6 million, or $(0.04) per basic and diluted share, in the three months ended September 30, 2022.

First Nine months 2023 Financial Results Compared to Prior Year Period

Research and development expenses decreased $0.8 million, or 2%, compared to the nine months ended September 30, 2022. This decrease was primarily due to continued reprioritization of clinical product candidates.

SG&A expenses decreased $6.3 million, or 17%, compared to the nine months ended September 30, 2022. This decrease was primarily driven by a reduction in professional fees of $4.8 million, due to decreased legal fees
associated with certain litigation matters, as well as a $1.2 million reduction in salaries, benefits, and other personnel costs due to reduced head count, and $0.3 million in decreased insurance related expenses.

Total revenues decreased $20.1 million, or 80%, from the nine months ended September 30, 2022. Collaboration and licensing revenues decreased $14.6 million or 100% from the nine months ended September 2022, primarily due to the prior year period non-cash recognition of revenue related to historical collaboration agreements for which revenue was previously deferred. Product and services revenues decreased $5.4 million, or 52%, from the nine months ended September 30, 2022. This decrease primarily related to reductions in services performed at Exemplar as well as the recognition of revenue in the first quarter of 2022 related to agreements for which revenue was previously deferred that did not occur in 2023 of $1.0 million at Exemplar.

Total other income, net, increased $7.3 million compared to the nine months ended September 30, 2022. This is primarily due to $5.7 million reduced interest expense associated with the Convertible Notes as they were retired in the second quarter of 2023, and $2.1 million increased interest income due to higher interest rates on the Company’s investments. This increase was offset by $0.8 million reduction in the gain recorded on the early retirement of a portion of the Convertible Notes in 2023 compared to 2022.

Loss from continuing operations was $62.8 million, or $(0.26) per basic and diluted share, compared to loss from continuing operations of $57.6 million, or $(0.29) per basic and diluted share, in the nine months ended September 30, 2022.

Poseida Therapeutics Provides Updates and Financial Results for the Third Quarter of 2023

On November 9, 2023 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, reported updates and financial results for the third quarter ended September 30, 2023 (Press release, Poseida Therapeutics, NOV 9, 2023, View Source [SID1234637415]).

"In the third quarter, we continued to execute on our key priorities for 2023 while strengthening our financial position with a $50 million strategic investment from Astellas that closed in August 2023. Additionally, we have made strong progress in our Roche Collaboration, accelerating certain milestone payments," said Mark Gergen, Chief Executive Officer of the Company. "With the recent announcement of my upcoming transition to the role of Executive Chairman on January 1, 2024, I am excited about the future of Poseida under the leadership of Kristin Yarema, Ph.D., who will assume the role of President and CEO."

Based upon substantial progress in its P-BCMA-ALLO1 and P-CD19CD20-ALLO1 programs, the Company is announcing today that certain payments as well as the expected timing of achievement of upcoming milestones, have been accelerated to reflect progress in the programs and better align with expected upcoming further clinical development and manufacturing needs and timelines. Poseida may also receive additional funding and resources for select expanded research, clinical development, and manufacturing activities under the existing Roche Collaboration Agreement. As a result of this progress, the Company expects to receive certain payments sooner and/or with more certainty than originally anticipated.

"The combination of the Astellas investment and the progress in our Roche Collaboration has strengthened our financial position in the last quarter," said Johanna Mylet, Chief Financial Officer at the Company. "In addition to extending our baseline cash runway, we continue to have potential further upside in the near term under the Roche Collaboration Agreement as well as additional business development opportunities to further extend our cash runway."

The Company previously announced the acceptance of three poster presentations at the ASH (Free ASH Whitepaper) Annual Meeting, taking place in San Diego and virtually in December 2023. In separate presentations, the Company plans to present interim safety and efficacy data on P-BCMA-ALLO1, the Company’s Phase 1 allogeneic cell therapy program in multiple myeloma partnered with Roche, and P-FVIII-101, the Company’s preclinical non-viral gene therapy program in Hemophilia A.

"As we advance our cell therapy pipeline, we continue to be excited about the significant progress we are making in our Roche partnership. The advancements we have made are being recognized with acceleration of, and increased certainty around achievement of additional near-term milestones, which extends our cash runway and further validates the progress we are making across our allogeneic cell therapies," said Dr. Yarema, President, Cell Therapy at the Company. "In addition to the clinical progress for our allo BCMA program that will be presented at ASH (Free ASH Whitepaper), we continue to expect dosing to begin in our P-CD19CD20-ALLO1 program in B-cell malignancies in early 2024. Across our allogeneic portfolio, we have seen significant improvement in raising product yields through unit operation optimization at our clinical manufacturing facility, as recently highlighted in our CAR-TCR Summit presentation. In our lead solid tumor program, P-MUC1C-ALLO1, in order to gain the full benefit of our recently implemented program learnings, such as preconditioning regimen, as well as due to market factors and slightly slower than expected enrollment in some newer key cohorts of this basket study, we have made the decision to shift the timing for an interim data update to a medical meeting in the first half of next year. As we look to 2024, we plan to provide an overall Company update and outlook in early January."

Program Updates

Cell Therapy Programs

MUC1-C Program

P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate targeting solid tumors derived from epithelial cells, including breast and ovarian cancers. The Company is currently evaluating P-MUC1C-ALLO1 in a Phase 1 clinical trial. Poseida plans to provide an interim clinical update at a medical meeting in the first half of 2024.

BCMA Program

P-BCMA-ALLO1 is an allogeneic CAR-T product candidate being developed to target relapsed/refractory multiple myeloma (R/R MM) in partnership with Roche. The Company is currently evaluating P-BCMA-ALLO1 in a Phase 1 clinical trial and plans to share early safety and preliminary efficacy results at the ASH (Free ASH Whitepaper) Annual Meeting, taking place in San Diego and virtually in December 2023.

CD19CD20 Program

P-CD19CD20-ALLO1 is an allogeneic CAR-T product being developed to target B-cell malignancies in partnership with Roche. P-CD19CD20-ALLO1 is the Company’s first dual CAR program and contains two fully functional CAR molecules to target cells that express either CD19 or CD20, or both. Poseida expects to dose the first patient with P-CD19CD20-ALLO1 in early 2024.

Gene Therapy Programs

The Company is in the process of strategically evaluating its gene therapy and gene editing programs including the programs previously licensed to Takeda to determine which programs it will prioritize and progress internally. In addition, the Company is actively evaluating the potential to leverage these programs and technologies through business development. The Company intends to provide an update on this evaluation when complete, which is expected to be in the first half of 2024.

FVIII Program

The Company is advancing its P-FVIII-101 preclinical program, which is in development for the in vivo treatment of Hemophilia A. P-FVIII-101 utilizes piggyBac gene modification delivered via lipid nanoparticle that has demonstrated stable and sustained Factor VIII expression in animal models. The Company is presenting preclinical data from this program at the upcoming ASH (Free ASH Whitepaper) Annual Meeting.

OTC Program

P-OTC-101 is an in vivo program for the treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene. The Company is developing the P-OTC-101 program utilizing a hybrid delivery system and working on an updated timeline for the program. The Company received orphan drug designation for this program from the FDA in July 2023.

PAH Program

P-PAH-101 is a liver-directed gene therapy to treat Phenylketonuria (PKU), an inherited genetic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene resulting in buildup of phenylalanine in the body. If left untreated, PKU can affect a person’s cognitive development. P-PAH-101 is currently in preclinical development.

Other Operational Updates and Upcoming Events

Strategic Investment

In August 2023 the Company announced a $50 million strategic investment by Astellas and granted Astellas certain strategic rights.

Leadership Updates

As previously announced, effective January 1, 2024, Dr. Yarema, the Company’s current President, Cell Therapy, will transition to the role of President and CEO of the Company and Mr. Gergen, the Company’s current CEO and Chairman, will transition to the role of Executive Chairman of the board of directors. Brent Warner, President, Gene Therapy, will continue to report to Mr. Gergen.

Poseida R&D Days

In recognition of its continued development and growth, and to highlight its proprietary platform technologies and preclinical research in 2024, the Company plans to hold two R&D Days – the first focusing on gene therapy in April 2024 and the second focusing on cell therapy in the fall of 2024. Additional details are expected to be announced early next year.

Financial Results for the Third Quarter 2023

Revenues

Revenues were $9.4 million for the three months ended September 30, 2023, compared to $116.3 million for the same period in 2022. The decrease was primarily due to initial license revenue recognized from the collaboration and license agreement with Roche, which became effective in the third quarter of 2022, offset by the revenue recognized related to the research services performed under the collaboration and license agreements with Roche and Takeda.

For the nine months ended September 30, 2023, revenues were $39.7 million, compared to $120.4 million for the same period in 2022. The decrease was primarily due to initial license revenue recognized from the collaboration and license agreement with Roche, which became effective in the third quarter of 2022, offset by the revenue recognized related to the research services performed under the collaboration and license agreements with Roche and Takeda, including $8.9 million of

previously deferred revenue recognized as a result of the termination of its collaboration agreement with Takeda in July 2023.

Research and Development Expenses

Research and development expenses were $37.5 million for the three months ended September 30, 2023, compared to $35.1 million for the same period in 2022. The increase was primarily due to an increase in personnel expenses as a result of increased headcount, an increase in preclinical stage programs and other unallocated expenses due to an increase in research collaboration activity, offset by a decrease in clinical stage programs, primarily driven by the wind-down of the Company’s clinical development activities associated with its autologous programs.

For the nine months ended September 30, 2023, research and development expenses were $114.7 million, compared to $119.0 million for the same period in 2022. The decrease was primarily due to a decrease in external costs related to the Company’s autologous clinical stage programs, partially offset by an increase in personnel expenses as a result of increased headcount, an increase in external costs related to its preclinical stage programs and other unallocated expenses due to an increase in research collaboration activity.

General and Administrative Expenses

General and administrative expenses were $8.1 million for the three months ended September 30, 2023, compared to $9.4 million for the same period in 2022. The decrease was primarily due to lower professional fees and facility costs.

For the nine months ended September 30, 2023, general and administrative expenses were $28.6 million, compared to $28.2 million for the same period in 2022. The increase was primarily due to an accelerated stock-based compensation expense in the first quarter of 2023 related to a one-time modification associated with the retirement of the Company’s former Executive Chairman, offset by lower facility costs.

Net Income (Loss)

Net loss was $31.8 million and $98.1 million for the three and nine months ended September 30, 2023, respectively, compared to net income of $70.4 million and net loss of $30.7 million for the three and nine months ended September 30, 2022, respectively.

Cash Position

As of September 30, 2023, the Company’s cash, cash equivalents and short-term investments balance was $238.8 million. The Company expects that its cash, cash equivalents and short-term investments together with the remaining near-term milestones and other payments from Roche as well as the proceeds from the Astellas strategic investment will be sufficient to fund operations into the second half of 2025. Potential additional payments under the Roche Collaboration Agreement and/or potential additional business development could extend cash runway beyond the second half of 2025.

PMV Pharmaceuticals Reports Third Quarter 2023 Financial Results and Corporate Highlights

On November 9, 2023 PMV Pharmaceuticals, Inc. (Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule, tumor agnostic therapies targeting p53, reported financial results for the third quarter ended September 30, 2023, and provided a corporate update (Press release, PMV Pharma, NOV 9, 2023, View Source [SID1234637414]).

"We were very pleased to recently share updated Phase 1 data from our PYNNACLE clinical trial with the oncology community, demonstrating clinical efficacy and safety of PC14586 in heavily pretreated patients across multiple solid tumor types," said David Mack, Ph.D., President and Chief Executive Officer. "On the strength of the positive findings and guidance from the FDA, we selected the recommended Phase 2 dose and are aligned on the clinical and regulatory pathway for further development of PC14586. We look forward to initiating a registrational Phase 2 study in the first quarter of 2024."

Third Quarter 2023 and Recent Corporate Highlights:

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Updated clinical results from the Phase 1 PYNNACLE study evaluating PC14586 were featured in a late-breaking poster at the 2023 AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) on October 12, 2023. Confirmed responses were observed in patients whose tumors were TP53 Y220C and KRAS wild-type in the efficacious dose range, in multiple tumor types including ovarian, breast, prostate, small-cell lung, and endometrial cancer. An overall response rate of 38% was achieved at the Recommended Phase 2 Dose (RP2D) of 2000 mg daily (6/16 evaluable patients) reflective of the planned Phase 2 patient population (TP53 Y220C and KRAS wild-type). The median duration of response was seven months. A copy of the poster can be found on the PMV corporate website here: View Source
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The RP2D of 2000 mg once daily was selected based on overall safety, pharmacokinetics (PK), and efficacy in alignment with the U.S. Food and Drug Administration (FDA) at an End of Phase 1 meeting held in Q3 2023. PMV plans to initiate a registrational tumor-agnostic Phase 2 clinical trial in early 2024.
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The PYNNACLE clinical trial results were also highlighted in a KOL webinar which included a presentation by Aparna Parikh, M.D, M.S., Director of the Global Cancer Care Program at Mass General Hospital Cancer Center. A copy of the webinar presentation can be accessed here: View Source
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Ongoing enrollment in the combination arm of PYNNACLE evaluating PC14586 with KEYTRUDA (pembrolizumab). PMV and Merck entered into a collaboration in 2022 under the terms of which Merck is supplying KEYTRUDA for this study.

Third Quarter 2023 Financial Results

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During the nine months ended September 30, 2023, the Company raised $35.1 million in net proceeds through an At-the-Market facility (ATM).
Exhibit 99.1

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PMV Pharma ended the third quarter with $238.1 million in cash, cash equivalents, and marketable securities.
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Net loss for the nine months ended September 30, 2023, was $53.2 million compared to $54.0 million for the nine months ended September 30, 2022.
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Research and development (R&D) expenses were $42.5 million for the nine months ended September 30, 2023, compared to $37.0 million for the nine months ended September 30, 2022. The increase in R&D expenses was primarily related to increased headcount and clinical expenses to advance research on PC14586, the Company’s lead drug candidate.
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General and administrative (G&A) expenses were $18.7 million for the nine months ended September 30, 2023, compared to $18.9 million for the nine months ended September 30, 2022. The decrease in G&A expenses was primarily due to facility-related costs now allocated to research as our new laboratory building in Princeton, New Jersey began operations.

KEYTRUDA (pembrolizumab) is a registered trademark of Merck Sharp & Dohme LLC., a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.