On June 1, 2022 Midatech Pharma PLC (AIM: MTPH.L; Nasdaq: MTP), an R&D biotechnology company focused on improving the bio-delivery and biodistribution of medicines, reported that upon submitting an application to the U.S. Food and Drug Administration ("FDA"), its development programme of MTX110 for the treatment of recurrent glioblastoma ("rGBM") has been granted Fast Track designation by the agency (Press release, Midatech Pharma, JUN 1, 2022, View Source [SID1234615608]).

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Fast Track is a process designed to facilitate the development and expedite the review of treatments for serious conditions and that potentially address unmet medical needs. Drugs that are granted this designation are given the opportunity for more frequent interactions with the FDA, as well as potential pathways for expedited approval.

Commenting, Dmitry Zamoryakhin, CSO of Midatech, said: "GBM is a devastating cancer marked by short survival rate and universal recurrence. Receiving Fast Track designation for MTX110 is an important milestone for the development of the drug as it demonstrates the need for novel and effective treatment options for this currently universally fatal disease. MTX110, our water-soluble formulation of Panobinostat, will soon start recruitment into a company-sponsored Phase I study in patients with recurrent GBM".

This announcement contains inside information for the purposes of Article 7 of the Market Abuse Regulation (EU) 596/2014, as it forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018 (as amended).

About MTX110

MTX110 is a water-soluble form of panobinostat free base, achieved through complexation with hydroxypropyl-β-cyclodextrin (HPBCD), that enables convection-enhanced delivery (CED) at potentially chemotherapeutic doses directly to the site of the tumour. Panobinostat is a hydroxamic acid and acts as a non-selective histone deacetylase inhibitor (pan-HDAC inhibitor). The currently available oral formulation of panobinostat lactate (Farydak) is not suitable for treatment of brain cancers owing to poor blood-brain barrier penetration and inadequate brain drug concentrations. Based on favourable translational science data, MTX110 is being evaluated clinically as a treatment for DIPG (NCT03566199, NCT04264143) and recurrent medulloblastoma (NCT04315064), and preclinically for treatment of glioblastoma (SNO 2020 Abstract TMOD-27). MTX110 is delivered directly into and around the patient’s tumour via a catheter system (e.g. CED or fourth ventricle infusions) to bypass the blood-brain barrier. This technique exposes the tumour to very high drug concentrations while simultaneously minimising systemic drug levels and the potential for toxicity and other side effects. Panobinostat has demonstrated high potency against DIPG tumour cells in in vitro and in vivo models, and in a key study it was the most promising of 83 anticancer agents tested in 14 patient-derived DIPG cell lines (Grasso et al, 2015. Nature Medicine 21(6), 555-559).

On June 1, 2022 Oncorus, Inc. (Nasdaq: ONCR), a viral immunotherapies company focused on driving innovation to transform outcomes for cancer patients, reported that President and Chief Executive Officer, Theodore (Ted) Ashburn, M.D., Ph.D., will participate in a fireside chat at the Jefferies Global Healthcare Conference on Wednesday, June 8, 2022 at 3:30 p.m. ET in New York, NY (Press release, Oncorus, JUN 1, 2022, View Source [SID1234615321]).

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A live webcast of the fireside chat can be accessed by visiting the Investors & Media section of Oncorus’ website at View Source A replay of the webcast will be archived on Oncorus’ website for 90 days following the event.

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."