On July 6, 2022 Pyramid Biosciences, Inc., a clinical-stage biotechnology company developing a portfolio of precision therapies targeting a range of serious diseases, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for PBI-200, a next-generation, highly brain penetrant inhibitor of the neurotrophic tyrosine receptor kinase (NTRK) currently in clinical development for the treatment of NTRK fusion-positive solid tumors (Press release, Pyramid Biosciences, JUL 6, 2022, View Source [SID1234616516]).

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"Oncogenic NTRK gene fusions are found in a broad range of cancers," said Pyramid Biosciences CEO Brian Lestini, MD, PhD. "Orphan designation for drugs in development such as PBI-200 are important to ensure continued innovation to address unmet needs for these patients, including primary and metastatic brain tumors."

"We are extremely pleased that the orphan designation was granted, and we look forward to continued enrollment in our global Phase 1/2 clinical trial of PBI-200," said Pyramid Biosciences co-founder and COO Jordan Leef.

The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations or rare disorders, that affect fewer than 200,000 people in the United States.

About PBI-200

PBI-200 is an oral, highly potent, and selective inhibitor of TRK kinase that is currently in clinical development for the treatment of patients with cancers that harbor abnormalities involving the tropomyosin receptor kinases. In some patients, the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. PBI-200 was discovered and developed by Pyramid Biosciences to overcome a wide range of on-target resistance mutations that have been described with first-generation TRK inhibitors. In addition, preclinical studies have shown that PBI-200 achieves high levels of penetration into brain, which translated into superior efficacy in intracranial xenograft models compared to other TRK inhibitors, as well as a favorable safety profile.

The global Phase 1/2 trial of PBI-200 (PBI-200-101) is a multicenter, open-label study in subjects with NTRK fusion-positive advanced or metastatic tumors including primary and metastatic CNS tumors. The trial consists of a dose-escalation phase, followed by a multicohort expansion at the recommended Phase 2 dose. Additional information can be found at: View Source (NCT04901806).

On July 6, 2022 Turnstone Biologics Corp., a clinical-stage biotechnology company developing next-generation immunotherapies to treat and cure solid tumors, and Moffitt Cancer Center ("Moffitt"), a world leader in advancing cellular immunotherapies for the treatment of cancer, reported the formation of a broad strategic alliance, deepening their existing multi-year research collaboration (Press release, Turnstone Biologics, JUL 6, 2022, View Source [SID1234616515]). As part of this expanded partnership, Turnstone will have priority access to Moffitt’s scientific research, manufacturing, and clinical capabilities for the development of novel tumor infiltrating lymphocyte ("TIL") therapies. The parties also announced U.S. Food and Drug Administration ("FDA") clearance of an Investigational New Drug ("IND") application for a Moffitt-sponsored Phase 1 clinical study of TIDAL-01, Turnstone’s lead TIL therapy candidate, in cutaneous and non-cutaneous melanoma. The trial is expected to commence later this year.

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"Our landmark strategic alliance with Turnstone underscores Moffitt’s commitment to bold research initiatives and groundbreaking clinical studies for the benefit of cancer patients who have limited or no effective treatment options," said Patrick Hwu, M.D., President and CEO, Moffitt Cancer Center. "Cell therapy is a key area of focus at Moffitt, and we are impressed by Turnstone’s novel approach to TIL therapy. Our combined team has made strong progress as part of our ongoing partnership, particularly with the IND clearance of TIDAL-01. We look forward to initiating the clinical study in melanoma and continuing to grow this valuable relationship with Turnstone."

Selected TILs represent the foundational therapeutic modality driving Turnstone’s cancer immunotherapy pipeline and leverage decades of work anchored in academia that have demonstrated the promise of this approach. By identifying, selecting, and expanding the most potent subsets of patient specific tumor-reactive T-cells, Turnstone aims to improve and broaden the clinical efficacy of TILs and overcome the limitations of current treatments to achieve positive outcomes in harder to treat, lower mutational burden, cancers. TIDAL-01 builds on the historical clinical success of Selected TIL treatment protocols and is the initial focus of the Turnstone-Moffitt collaboration as the parties advance the program into a Phase 1 clinical study.

Under the terms of the agreement, Moffitt will grant Turnstone prioritized clinical trial activation, enhanced patient screening and data sharing, full access to Moffitt’s cellular therapies research and development infrastructure and expanded molecular data sets and biospecimens for research. Moffitt will also provide extended governance support and allocated GMP manufacturing capacity for Turnstone product candidates. Turnstone will contribute resources and expertise to the partnership and fund research and development activities at Moffitt. Additional financial terms were not disclosed.

"This industry-leading partnership with Moffitt is a direct result of our unwavering efforts to develop promising new cancer medicines in the most innovative and impactful ways possible," said Sammy Farah, Ph.D., M.B.A., President and CEO, Turnstone Biologics. "We believe Moffitt’s translational insights and clinical execution capabilities coupled with our next-generation Selected TIL technology will accelerate the development of our differentiated TIL therapies and increases our opportunity to create curative outcomes for patients with solid tumors."

On July 6, 2022 POINT Biopharma Global Inc. (NASDAQ: PNT) (the "Company" or "POINT"), a company accelerating the discovery, development and global access to life-changing radiopharmaceuticals, reported that it will participate in the following upcoming investor conferences (Press release, Point Biopharma, JUL 6, 2022, View Source [SID1234616514]):

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William Blair & Company – Biotech Focus Conference 2022

Brookline Capital Markets – Newport Biotech Symposium

On July 6, 2022 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that management will be attending William Blair’s Biotech Focus Conference 2022 at The St. Regis New York in New York City (Press release, Autolus, JUL 6, 2022, View Source [SID1234616513]). Autolus’ Chief Executive Officer, Dr. Christian Itin, will participate in a panel discussion on ‘Operationalizing Cell Therapies’ on Tuesday, July 12 at 8.55 – 10.15 am ET (1:55 pm – 3.15 pm BST) and the Company will also be attending one-on-one investor meetings at the event.

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On July 6, 2022 BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, reported it entered into a worldwide strategic collaboration with InnoRNA, a biotechnology company with expertise in LNP-based delivery technology and mRNA drug discovery, to leverage its innovative technology platform for developing mRNA-based therapeutics (Press release, BeiGene, JUL 6, 2022, View Source [SID1234616512]).

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"As a global biotechnology company, BeiGene is committed to delivering next-generation therapies through our own internal discovery engine and leveraging cutting-edge technology from experienced and innovative partners"

"As a global biotechnology company, BeiGene is committed to delivering next-generation therapies through our own internal discovery engine and leveraging cutting-edge technology from experienced and innovative partners," said Lai Wang, Ph.D., Global Head of R&D at BeiGene. "This collaboration with InnoRNA advances and supports our research efforts in the important field of mRNA therapies while securing critical, proprietary delivery tools."

Under the terms of the agreement, InnoRNA will receive an upfront cash payment and will be eligible to receive additional payments based upon the achievement of certain development, regulatory, and commercial milestones, as well as tiered royalties, for the mRNA-LNP research collaboration programs. BeiGene will hold exclusive global development and commercialization rights for the mRNA-LNP therapies jointly discovered by InnoRNA and BeiGene.

InnoRNA will receive an additional upfront cash payment and is eligible for additional milestone payments for granting BeiGene non-exclusive license to its proprietary LNP Technology platform.

"We are very excited to partner with BeiGene to discover and develop innovative mRNA therapies as well as contribute our LNP technology to BeiGene’s internal research," said Linxian Li, Ph.D., Founder and Chief Executive Officer of InnoRNA. "As evidenced by the success of multiple Covid-19 vaccines, mRNA and LNPs will likely play a major role in the future of drug development, potentially in broad fields beyond vaccines. Joining this collaboration with BeiGene represents a big step forward toward this vision for our company."