On June 1, 2022 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company, reported it has entered into a worldwide license and collaboration agreement with Roche for the development and commercialization of camonsertib (also known as RP-3500), 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 including those in the ATM gene (Ataxia-Telangiectasia mutated kinase) (Press release, Repare Therapeutics, JUN 1, 2022, View Source [SID1234615337]). Under the collaboration, Roche will assume development of camonsertib with the potential to expand development into additional tumors and multiple combination studies.

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"Camonsertib has the potential to help cancer patients across numerous solid tumors as a monotherapy and possibly in combination with other agents," said Kim Seth, Ph.D., EVP and Head of Business & Corporate Development at Repare. "Given the encouraging data Repare has generated for camonsertib as a potentially best-in-class ATR inhibitor with a promising tolerability profile and patient selection insights in areas of high unmet medical need, and Roche’s leading global footprint and unique expertise in precision oncology, we are confident that Roche is the ideal partner for us to drive the broad global development and commercialization of camonsertib."

"Roche is excited about the emerging DNA damage response field, which represents a promising new approach to precision oncology," said James Sabry, M.D., Ph.D., Global Head of Pharma Partnering, Roche. "We are looking forward to partnering with Repare Therapeutics to further develop camonsertib as a new potential treatment option for patients with significant unmet medical needs across a range of tumor types. The collaboration with Repare builds on Roche’s strategy of personalized healthcare and further strengthens our leadership in oncology."

Under the terms of the agreement, Repare will receive a $125 million upfront payment, and is eligible to receive up to $1.2 billion in potential clinical, regulatory, commercial and sales milestones, including up to $55 million in potential near-term payments, and royalties on global net sales ranging from high-single-digits to high-teens. The collaboration also provides Repare with the ability to opt-in to a 50/50 U.S. co-development and profit share arrangement, including participation in U.S. co-promotion if U.S. regulatory approval is received. If Repare chooses to exercise its co-development and profit share option, it will continue to be eligible to receive certain clinical, regulatory, commercial and sales milestone payments, in addition to full ex-U.S. royalties.

The transaction is subject to clearance under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary closing conditions.

Company Conference Call:

The Company will host a conference call with accompanying slides for analysts and investors today at 5:00 p.m. Eastern Time to further discuss the collaboration. To access the call, please dial (877) 870-4263 (U.S.) or (855) 669-9657 (Canada) or (412) 317-0790 (international) at least 10 minutes prior to the start time and ask to be joined to the Repare Therapeutics call. A live video webcast will be available in the Investor section of the Company’s website at View Source A webcast replay will also be archived for at least 30 days.

About Repare Therapeutics’ SNIPRx Platform

Repare’s SNIPRx platform is a genome-wide CRISPR-based screening approach that utilizes proprietary isogenic cell lines to identify novel and known synthetic lethal gene pairs and the corresponding patients who are most likely to benefit from the Company’s therapies based on the genetic profile of their tumors. Repare’s platform enables the development of precision therapeutics in patients whose tumors contain one or more genomic alterations identified by SNIPRx screening, in order to selectively target those tumors in patients most likely to achieve clinical benefit from resulting product candidates.

On June 1, 2022 Priothera, a late-clinical stage biotechnology company pioneering the development of its S1P receptor modulator compound, mocravimod, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation (FTD) for mocravimod in combination with allogeneic Hematopoietic Stem Cell Transplant (HSCT) for post remission therapy of Acute Myeloid Leukemia (AML) patients (Press release, Priothera, JUN 1, 2022, View Source [SID1234615355]). FDA’s Fast Track designation is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening diseases and that demonstrate the potential to address unmet medical needs.

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Priothera is working to initiate the MO-TRANS global Phase 2b/3 study in Europe, US and Japan, to assess the efficacy and safety of mocravimod as an adjunctive and maintenance therapy in adult AML patients undergoing allogeneic HSCT. The MO-TRANS study is expected to start in the second half of 2022 and preliminary data from this study are expected by the end of 2024.

Karen Von Graevenitz, Head of Regulatory Affair at Priothera, commented ""The Fast Track designation grant for mocravimod in combination with allogeneic HSCT is an important milestoneand underlines the significant unmet need in AML patients undergoing HSCT, a serious disease where currently no available therapy exists. The designation means mocravimod will be eligible for expedited review and we will work closely with the US FDA to advance the global Phase 2/3 trial which is due to start in the second half of 2022."

Florent Gros, Co-Founder and CEO of Priothera, added: "Following being granted orphan drug designations for mocravimod in the US and Europe, we are pleased to have been granted Fast Track designation for this highly promising compound. This important regulatory milestone moves us a step closer to bringing mocravimod to patients with AML and other hematologic malignancies."

About Mocravimod
Mocravimod (also known as KRP203), is a synthetic, sphingosine 1-phosphate receptor (S1PR) modulator. This novel investigational drug has been assessed in Phase 1 and Phase 2 trials for safety and tolerability, as well as for efficacy in several autoimmune indications. Promising data from a Phase 1b/2a clinical study in patients with hematological malignancies led Priothera to further develop mocravimod for the treatment of blood cancers.

Mocravimod will be investigated as an adjunctive and maintenance treatment in a Phase 2b/3 study as a potential treatment for patients with Acute Myeloid Leukemia (AML) receiving allogeneic hematopoietic stem cell transplantation (HSCT). Allogeneic HSCT is the only potentially curative approach for AML patients, but current treatments have unacceptably high mortality and morbidity rates.

Priothera leverages S1PR modulator’s unique mode of action to maintain anti-leukemia activity – graft-versus leukemia (GVL) while reducing tissue damage resulting from graft-versus-host disease (GVHD), a consequence of allogeneic HSCT. This novel treatment approach – mocravimod being the only S1PR modulator treating blood cancers – tackles a high unmet medical need and intends to add quality life to patients.

On June 1, 2022 Flatiron HealthⓇ reported the appointment of Javier Jimenez, MD, MPH, as Chief Medical Officer, effective June 1, 2022 (Press release, Flatiron Health, JUN 1, 2022, View Source [SID1234615374]). In his new role, Dr. Jimenez will drive our vision for integrated evidence, harnessing new approaches to oncology evidence generation through Flatiron’s engaged care network and fit-for-purpose scientific methods and tools. His clinical expertise and proven leadership, as well as his extensive experience with real-world evidence, will continue to advance our scientific efforts and a future where we accelerate R&D and realize a more equitable and sustainable cancer care ecosystem. Dr. Jimenez succeeds Dr. Michael Vasconcelles, who will remain at Flatiron as a senior advisor through August 1, 2022.

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"Our commitment to reimagining the infrastructure of cancer care is unwavering. Mike has been instrumental in elevating and deepening the expertise and focus of our scientific, clinical, and regulatory efforts, establishing the foundational building blocks for our path forward. I would like to thank him for his significant contributions to Flatiron, and for doing so with dedication and passion," said Carolyn Starrett, Flatiron CEO. "I’m pleased to welcome Javier as our new Chief Medical Officer, joining our executive team. Javier is a visionary and one of the most deeply experienced healthcare leaders in the field of real-world evidence and novel study design. Flatiron is approaching our ten-year anniversary as a company, and we are more passionate than ever to lay the groundwork for a world where cancer research and care are integrated and accelerated globally. Javier has been pursuing a similar vision as a Flatiron customer since our inception."

Prior to Flatiron, Dr. Jimenez served as Executive Vice President, Real World Evidence and Late Phase at Syneos Health, with expertise spanning Clinical Development, Medical Affairs, Market Access and Product Commercialization. During his time at Syneos Health, Dr. Jimenez grew the RWE function, integrating RWE solutions experts, data and analytics, innovation and RWE clinical operations, while driving the RWD and analytics and technology company strategy. Dr. Jimenez started his journey in the pharmaceutical industry over twenty years ago, first spending sixteen years leading Medical Evidence, Observational Research, Epidemiology, Regulatory, and HTA teams across the US and Europe at AstraZeneca, while building Phase IIIb/IV design and delivery capabilities as well. Dr. Jimenez’s vast experience in Oncology includes leading AstraZeneca’s collaboration with ASCO (Free ASCO Whitepaper)’s CancerLinQ. Following his time at AstraZeneca, Dr. Jimenez spent four years building the RWE, Advance Analytics and Clinical Outcomes functions at Sanofi to support all Sanofi Global Business units, as well as developing technology, tools and capabilities to leverage RWD and generate insights. As an MD and Epidemiologist by training, Dr. Jimenez is a passionate champion and key opinion leader in the use of real-world data to transform regulatory decision making and in the design of novel clinical trials. Dr. Jimenez’s recent research contributions span hypoglycemia predictive modeling in patients with Type 2 diabetes and cardiovascular risk prevention, management in clinical practice settings and use of RWD to identify novel indications for products in development and build research-grade RWD.

"I am incredibly honored to join Flatiron, a global leader and pioneer in real-world evidence in oncology," said Dr. Jimenez. "Flatiron is at a transformative point in its trajectory, working to create a more modern, connected oncology ecosystem – and I am inspired to be a part of an organization that is committed to improving lives by learning from the experience of every cancer patient."

On June 1, 2022 Iterion Therapeutics, Inc., a venture-backed, clinical-stage biotechnology company developing novel cancer therapeutics, reported that results from a Phase 1 study of tegavivint in patients with desmoid tumors will be featured in a poster presentation and discussion session at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO 2022) (Press release, Iterion Therapeutics, JUN 1, 2022, View Source [SID1234615391]). ASCO (Free ASCO Whitepaper) 2022 is being held June 3-7, 2022, in Chicago, Illinois.

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Tegavivint is a potent and selective first-in-class small molecule inhibitor of Transducin Beta-like Protein One (TBL1), a novel downstream co-factor in the Wnt/beta-catenin signaling pathway. Increased expression of beta-catenin and TBL1 are associated with metastasis and poor prognosis in a broad range of tumor types. Tegavivint’s targeting of TBL1 prevents TBL1/beta-catenin complex formation, specifically inhibiting beta-catenin’s oncogenic transcriptional activity without disrupting key cell membrane functions that have been linked to toxicity common to other drugs in this pathway.

The poster, titled, "Results of a phase I dose escalation study of a tegavivint (BC2059), a first-in-class TBL1 inhibitor for patients with progressive, unresectable desmoid tumors," will be presented on Sunday, June 5, 2022, at 11:30 a.m., CDT, during the Sarcoma session (poster number 428; abstract number 11523) by Lee D. Cranmer, M.D., Ph.D., F.A.C.P., principal investigator of the trial. The poster details results from the Phase 1 trial in adult patients with progressive, unresectable desmoid tumors. The primary objectives of the study were to evaluate safety and to determine the maximum tolerated dose (MTD), the recommended Phase 2 dose (RP2D), and exploratory efficacy. No dose-limiting toxicities were observed and a maximum tolerated dose (MTD) was not determined. Treatment related adverse events were mostly Grade 1-2 with none resulting in treatment discontinuation. The RP2D was declared at 5 mg/kg based on pharmacologically relevant plasma concentrations and preliminary efficacy. Responses were observed at all dose levels with an overall response rate of 25% at the RP2D. Additionally, the 9 month progression free survival rate was 79% among those treated at the RP2D. Overall, these data demonstrated that tegavivint is well tolerated, does not appear to have the toxicity historically associated with WNT inhibition, and has promising clinical activity.

"The data presented at ASCO (Free ASCO Whitepaper) complements research presented earlier this year at AACR (Free AACR Whitepaper), which cumulatively demonstrate the safety, tolerability and clinical activity of tegavivint in the treatment of desmoid tumors," said Rahul Aras, PhD, CEO of Iterion. "We are particularly excited to present these results at ASCO (Free ASCO Whitepaper), the world’s preeminent oncology conference, because the data also serve to highlight the potential of our ongoing tegavivint programs in AML, non-small cell lung cancer, and pediatric solid tumors. These cancer indications, like desmoid tumors, are associated with nuclear beta-catenin overexpression, which has historically been considered undruggable. We believe tegavivint’s unique mechanism of action provides an opportunity to selectively disrupt the interaction of beta-catenin and TBL1, which in turn, allows for the specific degradation of nuclear beta-catenin without impacting key cell membrane functions that have been linked to previously reported toxicity."

On June 1, 2022 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that the company will present at the Jefferies Healthcare Conference in New York, NY on Wednesday, June 8, 2022, at 9:00 a.m. ET and the JMP Securities Life Sciences Conference in New York, NY on Wednesday, June 15, 2022, at 12:00 p.m. ET (Press release, BioCryst Pharmaceuticals, JUN 1, 2022, View Source [SID1234615322]).

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Links to a live audio webcast and replay of these presentations may be accessed in the Investors & Media section of BioCryst’s website at http://www.biocryst.com.