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]).

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"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.

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]).

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"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.

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]).

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"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.

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]).

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"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:

•
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
•
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.
•
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
•
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

•
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

•
PMV Pharma ended the third quarter with $238.1 million in cash, cash equivalents, and marketable securities.
•
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.
•
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.
•
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.

On November 9, 2023 Pliant Therapeutics, Inc. (Nasdaq: PLRX), a clinical-stage biotechnology company and leader in the discovery and development of novel therapeutics for the treatment of fibrotic diseases, reported a corporate update and provided third quarter 2023 financial results (Press release, Pliant Therapeutics, NOV 9, 2023, View Source [SID1234637413]).

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"The third quarter was highlighted by positive interim data from the INTEGRIS-PSC trial of bexotegrast demonstrating a favorable safety profile and encouraging antifibrotic activity in PSC. Coupled with positive data reported from INTEGRIS-IPF, these data illustrate the broad potential of bexotegrast in fibrotic diseases across multiple organ systems," said Bernard Coulie, M.D., Ph.D., President and Chief Executive Officer of Pliant Therapeutics. "Our pipeline continues to advance, led by the ongoing enrollment of our Phase 2b BEACON-IPF trial. We have also taken active steps to strengthen our leadership team with the appointments of Minnie Kuo as Chief Development Officer and Mishima Gerhart as Chief Regulatory Officer, coming at an important time in Pliant’s evolution into a late-stage clinical development company."

Third Quarter and Recent Highlights

Bexotegrast Highlights
•Enrollment continues in BEACON-IPF, a Phase 2b trial of bexotegrast in patients with idiopathic pulmonary fibrosis (IPF). BEACON-IPF is a 52-week, multinational, randomized, dose-ranging, double-blind, placebo-controlled trial evaluating bexotegrast at once-daily doses of 160 mg or 320 mg. BEACON-IPF is expected to enroll approximately 270 patients with IPF.
•Positive safety and efficacy data from INTEGRIS-PSC Phase 2a trial in patients with primary sclerosing cholangitis (PSC). At once-daily doses of 40 mg, 80 mg and 160 mg, bexotegrast was well tolerated over 12 weeks of treatment with no drug-related severe or serious adverse events. At all doses tested, bexotegrast reduced both Enhanced Liver Fibrosis (ELF) scores and PRO-C3 levels at Week 12 relative to placebo, with statistically significant differences at the 160 mg dose. These data were selected for an oral late-breaker presentation at next week’s American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting 2023.
•Positive independent Data Safety Monitoring Board (DSMB) review of the ongoing of INTEGRIS-PSC Phase 2a trial. This regularly scheduled DSMB review was held in October after the completion of enrollment of the 320 mg dose cohort. The DSMB examined the safety data from all patients enrolled, with all patients completing at least 12 weeks of treatment, and recommended the INTEGRIS-PSC trial continue without modification.
•INTEGRIS-PSC interim 12-week 320 mg dose data expected in the first quarter of 2024. This trial is evaluating the safety, tolerability and pharmacokinetics of bexotegrast at 320 mg versus placebo at 12 and 24 weeks of treatment in approximately 28 patients with PSC. The trial is also evaluating exploratory efficacy endpoints including fibrosis biomarkers such as serum PRO-C3 and ELF score, changes in alkaline phosphatase (ALP) and liver imaging. Twenty-four week data from the 320 mg dose group is expected in mid-2024.
Pipeline Programs
•Phase 1 trial of PLN-101095 in solid tumors is enrolling. This is a Phase 1 open-label trial of PLN-101095, an oral, small-molecule, dual selective inhibitor of αvβ8 and αvβ1 integrins designed to block TGF-β activation in the tumor microenvironment. This trial is enrolling patients with solid tumors that are resistant to immune checkpoint inhibitors.

•Muscular dystrophy program on track for regulatory filing in the first quarter of 2024. PLN-101325 is a monoclonal antibody designed to act as an allosteric agonist of integrin α7β1. Filing for first-in-human clinical studies in Duchenne muscular dystrophy (DMD) is expected in the first quarter of 2024.
Corporate Highlights
•Appointment of Minnie Kuo as Chief Development Officer. Ms. Kuo is an experienced biopharma executive hired to oversee all clinical and non-clinical development activities.
•Appointment of S. Mishima Gerhart as Chief Regulatory Officer. Ms. Gerhart is a recognized leader in the biotechnology and pharmaceutical industries hired to lead all regulatory activities and quality functions.
Third Quarter 2023 Financial Results
•Research and development expenses were $32.3 million, as compared to $24.6 million for the prior-year quarter. The increase was due primarily to higher employee-related expenses and increased clinical and manufacturing-related costs associated with our lead program, bexotegrast, partially offset by a decrease in preclinical manufacturing costs for our pipeline product candidates.
•General and administrative expenses were $15.3 million, as compared to $8.8 million for the prior-year quarter. The increase was due to higher employee-related expenses.
•Net loss of $41.5 million as compared to $30.6 million for the prior-year quarter due to an increase in operating expenses coupled with a decrease in collaboration revenues under the Novartis collaboration during the quarter.
•As of September 30, 2023, the Company had cash, cash equivalents and short-term investments of $523.6 million which the Company expects to be sufficient to fund operations into the second half of 2026.