KRAS

Phase 1 analysis from the PYNNACLE Phase 1/2 study showed promising efficacy of rezatapopt (PC14586) in heavily pre-treated patients with advanced ovarian cancer harboring a TP53 Y220C mutation featured in a late-breaking oral presentation at 2024 SGO Annual MeetingOf the 15 patients in the efficacy evaluable population, seven patients achieved a confirmed partial response with a seven-month median duration of response and a favorable safety profileRezatapopt is a first-in-class precision oncology investigational therapy in patients with advanced solid tumors with a TP53 Y220C mutation and KRAS wild-type which is being evaluated in a registrational Phase 2 study PRINCETON, N.J., March 18, 2024 (GLOBE NEWSWIRE) -- PMV Pharmaceuticals, Inc. (Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53, today announced that a Phase 1 analysis reported promising anti-tumor activity of rezatapopt (PC14586) in heavily pre-treated patients with advanced ovarian cancer harboring a TP53 Y220C mutation. Rezatapopt is a first-in-class precision oncology small molecule investigational therapy that selectively targets the TP53 Y220C mutation in solid tumors. These data were featured today in a late-breaking oral presentation at the 2024 Society for Gynecologic Oncology (SGO) Annual Meeting on Women’s Cancer being held March 16-18, 2024 in San Diego, CA. The presentation entitled, “Phase 1 Analysis from the PYNNACLE Phase 1/2 Study PC14586 in the Subgroup of Patients with Advanced Ovarian Cancer Harboring a TP53 Y220C Mutation,” was delivered by Alison M. Schram, M.D., Medical Oncologist, Memorial Sloan Kettering Cancer Center. “In this Phase 1 study, we observed promising efficacy of rezatapopt in heavily pre-treated patients with advanced ovarian cancer harboring a TP53 Y220C mutation. In addition, rezatapopt showed a favorable safety profile,” said Dr. Schram. “These data are encouraging given the significant unmet medical need for patients with advanced solid tumors, particularly in patients with ovarian cancer who are platinum resistant. Further study of rezatapopt as monotherapy in ovarian cancer is warranted.” Deepika Jalota, Pharm.D., Chief Development Officer of PMV Pharma added, “These Phase 1 PYNNACLE data presented at the SGO meeting showed that rezatapopt, a first-in-class precision oncology investigational therapy, continues to demonstrate clinical benefit in a patient population of high unmet need. Our registrational, tumor-agnostic PYNNACLE Phase 2 trial, which includes an ovarian cancer cohort, remains on track to initiate in the first quarter of this year. The trial will assess rezatapopt as monotherapy at the recommended Phase 2 dose of 2000 mg daily in patients with TP53 Y220C and KRAS wild-type advanced solid tumors.” Results of Phase 1 Analysis of PYNNACLE in Advanced Ovarian Cancer Phase 1 data from the PYNNACLE trial (NCT04585750) demonstrated that rezatapopt has a favorable safety profile and induced responses in heavily pre-treated patients across multiple tumor types. This subgroup analysis investigated the efficacy of rezatapopt in patients with advanced ovarian cancer treated across the efficacious dose range (1150 mg daily to 1500 mg twice daily). Patient Characteristics As of September 5, 2023, the median age of patients with ovarian cancer (N=22) was 66 years (range 49 – 81 years)At baseline, 20 patients had high-grade serous ovarian cancer and two had endometrioid cancer Nineteen patients were platinum resistant and one was platinum refractoryTwo patients had a BRCA2 mutationSix patients were homologous recombination deficiency positiveAll patients were KRAS wild-typeMedian number of prior lines of systemic therapy was four (range 1 – 9) Efficacy The efficacy evaluable population consisted of 15 patients with measurable disease at baseline and ≥1 post-baseline tumor assessment. Seven patients achieved a confirmed partial response (PR), seven had stable disease (SD), and one had progressive diseaseMedian duration of response was seven monthsOf the 15 patients with measurable serum CA-125 at baseline, six had a CA-125 response. Among these, five patients achieved radiographic PR and one had SD Safety In the overall population of 67 patients assessed in the efficacious dose range (≥1150mg daily), including this subset of patients with ovarian cancer, treatment-related adverse events (TRAEs) were mostly grade 1 and 2. Most frequent TRAEs were nausea (51%), vomiting (43%), and increased blood creatinine (27%)Frequency and severity of TRAEs were similar in the ovarian cancer population compared with the overall populationRezatapopt administration with food led to an improvement in nausea and vomiting About Rezatapopt Rezatapopt (PC14586) is a first-in-class, small molecule, p53 reactivator designed to selectively bind to the pocket in the p53 Y220C mutant protein, restoring the wild-type, or normal, p53 protein structure and tumor-suppressing function. The U.S. Food and Drug Administration (FDA) granted Fast Track designation to rezatapopt for the treatment of patients with locally advanced or metastatic solid tumors with a p53 Y220C mutation. About the PYNNACLE Clinical Trial The ongoing Phase 1/2 PYNNACLE study is evaluating rezatapopt in patients with advanced solid tumors harboring a TP53 Y220C mutation. The primary objective of the Phase 1 portion of the trial was to determine the maximum tolerated dose and recommended Phase 2 dose (RP2D) of rezatapopt when administered orally to patients. Safety, tolerability, pharmacokinetics and effects on biomarkers will also be assessed. Phase 2 will be an expansion study with the primary objective of evaluating the efficacy of rezatapopt at the RP2D in patients with TP53 Y220C advanced solid tumors. For more information about the Phase 1/2 PYNNACLE clinical trial, refer to www.clinicaltrials.gov (NCT study identifier NCT04585750). About PMV Pharma PMV Pharma is a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53. p53 mutations are found in approximately half of all cancers. Our co-founder, Dr. Arnold Levine, established the field of p53 biology when he discovered the p53 protein in 1979. Bringing together leaders in the field to utilize over four decades of p53 biology, PMV Pharma combines unique biological understanding with a pharmaceutical development focus. PMV Pharma is headquartered in Princeton, New Jersey. For more information, please visit www.pmvpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the Company’s future plans or expectations for rezatapopt, including our ability to obtain approval on a tumor-agnostic basis, ongoing safety and response rate of participants in our clinical trials and expectations regarding timing and success of the Phase 2 portion of its current clinical trial for rezatapopt, including rezatapopt’s potential efficacy and safety profile in ovarian cancer patients. Any forward-looking statements in this statement are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of the Company’s product candidate development activities and planned clinical trials, the Company’s ability to execute on its strategy and operate as a clinical stage company, the potential for clinical trials of rezatapopt or any future clinical trials of other product candidates to differ from preclinical, preliminary, interim or expected results, the Company’s ability to fund operations, and the impact that any current or future global pandemic or geopolitical emergency may have on the Company’s clinical trials, supply chain, and operations, as well as those risks and uncertainties set forth in the section entitled “Risk Factors” in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (the “SEC”) on February 29, 2024, and its other filings filed with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investors Contact:Tim SmithSenior Vice President, Head of Corporate Development and Investor Relationsinvestors@pmvpharma.com Media Contact:Kathy VincentGreig Communicationskathy@greigcommunications.com

EmendoBio’s technology will aid Anocca in delivering tailored therapies to patients more rapidly. Credit: Natali _ Mis / Shutterstock.com. Swedish biotech company Anocca has entered a non-exclusive licensing agreement with EmendoBio to use the latter’s OMNI-A4 nuclease, a gene editing technology [that cleaves chains of nucleotides in nucleic acids into smaller units]. This strategic move is set to expedite developing and manufacturing Anocca’s pipeline of T cell receptor-engineered T cell (TCR-T) therapies, targeting difficult-to-treat solid cancers. The latest development comes after Anocca received good manufacturing practice compliance certification and a manufacturing licence from regulators in Sweden for its cell therapy production facility. Anocca CEO and co-founder Reagan Jarvis stated: “Integrating EmendoBio’s nuclease into our manufacturing process supports Anocca’s aim of generating the highest-quality cell therapy products. “This next-generation gene editing system provides the precision and efficiency needed to scale out production of our growing libraries of TCR-T products in a high-precision manufacturing process. See Also: Eli Lilly-backed Zephyr AI secures $111m for precision medicine tech Almirall to license Eloxx’s ZKN-013 for rare skin conditions “We are excited to work with EmendoBio to develop gene-edited TCR-T cell therapies as we prepare for our first clinical programme targeting the KRAS driver mutation in a hard-to-treat solid cancer.” EmendoBio’s technology is expected to significantly enhance Anocca’s manufacturing expertise. It will also aid the company in delivering tailored therapies to patients more rapidly, focusing on the genetic causes of hard-to-treat cancer types. The licensed gene editing technology is part of EmendoBio’s nuclease suite designed for high activity and specificity within cell therapy applications. EmendoBio director Dr Ei Yamada stated: “This non-exclusive licensing agreement marks a significant milestone in the field of T cell therapy. “Together with Anocca, we are embracing the shared vision of harnessing the potential of gene editing and cellular therapies to make a profound impact on patient outcomes. Our combined expertise will unlock novel avenues for therapeutic development and push the boundaries of what’s possible in advanced medicine.” In May 2023, Anocca secured Skr400m ($38.9m) in an equity financing round to develop next-generation cell therapies for cancer on an industrial scale. AMF and Mellby Gård led the funding round. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

Enabling Technology Supports the Development of Anocca’s Deep Pipeline of TCR-T therapeutics NEW YORK and SÖDERTÄLJE, Sweden, March 14, 2024 (GLOBE NEWSWIRE) -- Anocca AB (Anocca), a leading T cell receptor-engineered T cell (TCR-T) cellular therapeutics company, and EmendoBio Inc. (EmendoBio), a nuclease discovery and gene editing therapeutics company, today announced a non-exclusive licensing agreement for the use of EmendoBio’s novel OMNI-A4 nuclease to accelerate the manufacture and development of Anocca’s deep pipeline of TCR-T cell therapies for difficult-to-treat solid cancers. “Integrating EmendoBio’s nuclease into our manufacturing process supports Anocca’s aim of generating the highest-quality cell therapy products. This next-generation gene editing system provides the precision and efficiency needed to scale out production of our growing libraries of TCR-T products in a high-precision manufacturing process. We are excited to work with EmendoBio to develop gene-edited TCR-T cell therapies as we prepare for our first clinical program targeting the KRAS driver mutation in a hard-to-treat solid cancer” said Anocca’s CEO and co-founder, Reagan Jarvis. “This non-exclusive licensing agreement marks a significant milestone in the field of T cell therapy,” said Dr. Ei Yamada, Director of EmendoBio. “Together with Anocca, we are embracing the shared vision of harnessing the potential of gene editing and cellular therapies to make a profound impact on patient outcomes. Our combined expertise will unlock novel avenues for therapeutic development and push the boundaries of what's possible in advanced medicine.” Anocca recently received certification of GMP compliance and a manufacturing license from the Swedish regulators for their cell therapy production facility, the largest in the Nordics. EmendoBio’s technology strengthens Anocca’s manufacturing capability and provides a foundation for the ambitious goal of reaching more patients faster with personalised treatments targeting the underlying genetic drivers of hard-to-treat cancers. The licensed gene editing technology is part of EmendoBio’s portfolio of proprietary nucleases developed to be highly active and specific for cell therapy applications. Anocca’s and Emendo’s commitment to innovation and scientific excellence sets the stage for a promising future in T cell therapies. The agreement not only underscores the significance of collaborative efforts in the biotechnology sector but also reinforces both companies' dedication to improving global healthcare outcomes. Notes to editors About EmendoBio EmendoBio is a next-generation CRISPR gene editing company leveraging its proprietary technology to enable high precision gene editing, which is critical for the development of safe medical applications of genome editing. Driven by cutting-edge computational biology, novel gene editing strategies, and the latest of protein engineering tools, EmendoBio’s novel nuclease discovery platform, OMNI, broadens the targetable range of the genome, while its target-specific optimisation platform enables highly precise editing that maintains high efficiencies. EmendoBio’s proprietary OMNI nucleases also are designed to reduce "off-target” effects, i.e., cleavage at locations other than the targeted genes. Achieving such pinpoint precision is a substantial challenge for genome editing therapies. The capabilities of the OMNI technology platform, along with deep expertise in genomic medicine and protein engineering, provide EmendoBio with a unique advantage when addressing indications, including within haematology, cardiovascular, ophthalmology, oncology, and other disease areas. About Anocca Anocca is a fully integrated biopharmaceutical company developing libraries of T cell receptor-engineered T cell (TCR-T) therapies to redefine the treatment of solid tumours. Its proprietary technologies have been designed to vastly expand TCR-T development, allowing the systematic generation of treatments for the broadest patient populations that equip the immune system against the most difficult to treat solid cancers. Anocca operates an advanced research and development infrastructure, underpinned by a custom software ecosystem and in-house clinical manufacturing and process development facilities. Its unique discovery platform uses programmable human cells to recreate and manipulate T cell immunity. Follow Anocca on LinkedIn and visit . Media Inquiries Anocca AB Mark Farmery, CDO media@anocca.com Scius Communications (for Anocca AB) Katja Stout Tel: +44 7789 435 990 katja@sciuscommunications.com Daniel Gooch Tel: +44 7747 875 479 daniel@sciuscommunications.com EmendoBio Inc. Naoya Satoh, CEO Tel: +81 3 5730 2480 nsato@emendobio.com Noa Wertheimer Tel: +972 54 6437741 noaw@emendobio.com