On July 23, 2024 AffyImmune, a clinical-stage biotechnology company committed to developing novel, first-in-class chimeric antigen receptor (CAR) T cell therapies, reported the designation of Regenerative Medicine Advanced Therapy (RMAT) by the U.S. Food and Drug Administration (FDA) for its CAR T-cell product candidate, AIC100 as a potential treatment for patients with recurrent anaplastic thyroid cancer (ATC), the most aggressive form of the disease (Press release, AffyImmune Therapeutics, JUL 23, 2024, View Source [SID1234645027]).

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"We believe this important recognition from the FDA further supports the therapeutic potential of AIC100 to change the current treatment paradigm in advanced thyroid cancer and potentially other forms of aggressive solid tumors," said Daniel Janse, Ph.D., CEO at AffyImmune. "RMAT designation was granted following the FDA’s review of safety and efficacy data from the first ten patients dosed with AIC100 in our Phase 1 study. We believe the RMAT designation reinforces the potential ability of AIC100 to meet the high unmet medical need in recurrent ATC, an aggressive disease where a standard of care is currently not available."

RMAT designation was designed to expedite the development and review of regenerative medicine therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious condition, and preliminary clinical evidence indicates the therapy has the potential to address unmet medical needs for the disease. Sponsor companies receiving RMAT designation can benefit from increased interactions with the FDA involving senior managers, with the goal of expediting drug development.

"We remain focused on advancing the development of AIC100 for patients and families living with this devastating cancer," said Sonal Gupta, M.D., Ph.D., Senior Vice President and Head of Clinical Development. "Receiving RMAT designation helps facilitate this goal by enabling increased dialogue with the FDA to expedite our development plan for our affinity-tuned CAR T therapy. We look forward to working closely with the FDA and other regulatory agencies as we continue to advance this program."

At ASCO (Free ASCO Whitepaper) 2024, AffyImmune reported interim results from their Phase 1 study evaluating the safety and efficacy of AIC100, an ICAM-1 targeting and affinity-tuned LFA-1 binder CAR T-cell therapy, in patients with advanced thyroid cancer. Notably, a metabolic complete response was achieved in one patient with ATC, the most aggressive form of the disease.

For more information about the Phase 1 study, visit www.clinicaltrials.gov (NCT04420754).

On July 23, 2024 Triastek Inc. ("Triastek"), a global leader in 3D printing pharmaceuticals, reported that it has entered into a research collaboration and platform technology license agreement with BioNTech SE ("BioNTech"), a next generation immunotherapy company pioneering novel therapies for cancer and other serious diseases (Press release, BioNTech, JUL 23, 2024, View Source [SID1234645026]). Under the agreement, the companies will develop RNA therapeutics for oral delivery based on 3D printing technology. The collaboration aims to provide groundbreaking therapies to address unmet medical needs in an easy to administer oral formulation.

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Triastek will contribute to the collaboration its expertise in innovative oral tablet designs made possible by 3D printing aimed at optimizing delivery of RNA therapeutics across the gastrointestinal mucosa, minimizing degradation in the gastrointestinal tract, and delivering RNA therapeutics to the portion of the gastrointestinal tract where absorption will potentially be the greatest. Triastek’s ability to create tablet structures with unique external and internal tablet geometries, including multiple-layer and multi-compartment designs, will be leveraged, aiming to optimize delivery of novel RNA therapeutics.

"We are immensely honored to announce our collaboration with BioNTech, a leader in revolutionizing patient care with transformative medicines," stated Dr. Senping Cheng, Founder, and CEO of Triastek. "We believe this collaboration stands as a promising milestone in advancing oral RNA therapeutics using 3D printing technology and aims to set new benchmarks in the development of large molecule oral drugs. We are committed to working diligently together to make breakthroughs in oral delivery of RNA therapeutics."

Under the terms of the agreement, Triastek will receive an upfront payment of $10 million, and will be eligible to receive development, regulatory and commercial milestone payments potentially totaling over $1.2 billion as well as tiered royalties on potential future product sales.

On July 23, 2024 Nuvation Bio Inc. (NYSE: NUVB), a late clinical-stage, global biopharmaceutical company tackling some of the greatest unmet needs in oncology, reported multiple updates for its taletrectinib program (Press release, Nuvation Bio, JUL 23, 2024, View Source [SID1234645025]). Data from the global, pivotal Phase 2 TRUST-II study has been accepted for an oral presentation at WCLC 2024 taking place September 7-10 in San Diego, California. Pooled data from both pivotal Phase 2 studies, TRUST-I and TRUST-II, has been accepted for a poster presentation at ESMO (Free ESMO Whitepaper) 2024 taking place September 13-17, in Barcelona, Spain. The pooled data presented at ESMO (Free ESMO Whitepaper) will support the Company’s NDA in the United States. Additionally, the U.S. FDA has granted Orphan Drug Designation to taletrectinib for the treatment of multiple NSCLC indications, including ROS1-positive NSCLC.

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"We are excited to share these program updates as we continue toward our goal of bringing taletrectinib to patients with ROS1-positive NSCLC. We look forward to sharing the latest TRUST-II data at WCLC 2024 and pooled TRUST-I and TRUST-II data at ESMO (Free ESMO Whitepaper) 2024. The pooled data to be presented at ESMO (Free ESMO Whitepaper) will support our NDA in the U.S. and, we believe, position us to commercialize taletrectinib in 2025," said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. "Further, we are pleased with the FDA’s recent determination that taletrectinib qualifies for Orphan Drug Designation, which represents another key regulatory milestone for this important program."

Taletrectinib was granted Orphan Drug Designation for the treatment of ROS1- positive, NTRK-positive, ALK-positive, LTK-positive, ACK1-positive, or DDR1-positive NSCLC. The FDA’s Office of Orphan Drug Products grants this designation to support drug candidates in development for underserved patient populations or rare disorders that affect fewer than 200,000 people in the United States. Orphan Drug Designation qualifies a candidate for various development incentives, including tax credits for eligible clinical trials, waiver of application fees and potential market exclusivity for seven years upon FDA approval.

Taletrectinib is being evaluated for the treatment of patients with ROS1-positive NSCLC in two pivotal Phase 2 studies, TRUST-I (NCT04395677) in China and TRUST-II (NCT04919811), a global pivotal study.

WCLC Presentation Overview:

Title: Efficacy and Safety of Taletrectinib in Patients with ROS1+ Non–Small Cell Lung Cancer: The Global TRUST-II Study
Presenter: Geoffrey Liu, MD
Date: September 10, 2024
Session Time: 11:15 a.m. – 12:30 p.m. PDT
Session: MA06 – New Strategies in ALK, ROS1, NTRK, BRAF, and MET NSCLC
Abstract: 1752

ESMO Presentation Overview:

Title: Pooled Efficacy and Safety From 2 Pivotal Phase 2 Trials of Taletrectinib in Patients (Pts) With Advanced or Metastatic ROS1+ Non–Small Cell Lung Cancer (NSCLC)
Presenter: Maurice Perol, M.D.
Date: September 14, 2024
Session Time: Poster Lunch, 12:00-1:00 p.m. CEST (on display from 9:00 a.m. – 5:00 p.m. CEST)
Session: Poster Display, NSCLC, Metastatic
Abstract: 1289P

The materials will be made available on the Publications section of nuvationbio.com the day of the respective presentations.

About Taletrectinib

Taletrectinib is an oral, potent, central nervous system-active, selective, next-generation ROS1 inhibitor specifically designed for the treatment of patients with advanced ROS1-positive NSCLC. Taletrectinib is being evaluated for the treatment of patients with advanced ROS1-positive NSCLC in two Phase 2 single-arm pivotal studies: TRUST-I (NCT04395677) in China, and TRUST-II (NCT04919811), a global study. Taletrectinib has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with ROS1-positive NSCLC and Breakthrough Therapy Designations by both the U.S. FDA and China’s National Medical Products Administration (NMPA) for the treatment of patients with advanced or metastatic ROS1-positive NSCLC. Based on results of the TRUST-I clinical study, China’s NMPA has accepted and granted Priority Review Designations to New Drug Applications for taletrectinib for the treatment of adult patients with locally advanced or metastatic ROS1-positive NSCLC who either have or have not previously been treated with ROS1 tyrosine kinase inhibitors (TKIs).

About ROS1-positive NSCLC

More than one million people globally are diagnosed with NSCLC annually, the most common form of lung cancer. It is estimated that approximately 1-3% of people with NSCLC are ROS1-positive. Up to 35% of people newly diagnosed with metastatic ROS1-positive NSCLC have tumors that have spread to their brain, increasing up to 55% for those whose cancer has progressed following initial treatment. While people with other types of lung cancer have seen great advances, there has been limited progress for people with ROS1-positive NSCLC who remain in need of new options.

On July 23, 2024 ForDoz Pharma Corp., a private specialty pharmaceutical company focused on product development, manufacturing and commercialization of complex injectables, like liposomes, microspheres, nano-suspensions, etc., reported the ANDA approval of DOXOrubicin Hydrochloride Liposome Injection 20 mg/10 mL (2 mg/mL) and 50 mg/25 mL (2 mg/mL) from the United States Food and Drug Administration (US FDA) (Press release, ForDoz Pharma, JUL 23, 2024, View Source [SID1234645024]). DOXOrubicin Hydrochloride Liposome Injection is indicated for the treatment of patients with ovarian cancer and AIDS-related Kaposi’s sarcoma. Doxorubicin hydrochloride liposome injection, in combination with bortezomib, is indicated for the treatment of patients with multiple myeloma.

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"The approval of DOXOrubicin Hydrochloride Liposome Injection marks a major milestone for ForDoz Pharma as our 1st liposomal injectable pharmaceutical product to enter the US market. It will be distributed by Lupin Pharmaceuticals, Inc.," said James He, Founder and CEO of ForDoz Pharma.

On July 23, 2024 BioVaxys Technology Corp. (CSE: BIOV) (FRA: 5LB) (OTCQB: BVAXF) ("BioVaxys" or the "Company") reported that it intends to complete a non-brokered private placement (the "Private Placement") consisting of up to 10,000,000 units ("Units") at a price of $0.05 per Unit for total gross proceeds of CAD $500,000, before deducting any offering-related expenses (Press release, BioVaxys Technology, JUL 23, 2024, View Source [SID1234645023]). Each Unit consists of one common share (a "Common Share") and one whole Common Share purchase warrant (a "Warrant"). Each Warrant is exercisable for one additional Common Share at an exercise price of $0.15 for a period of 24 months. Closing of the proposed financing is expected to occur on or before July 31st, 2024.

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Closing of the private placement is conditional upon finalizing all contractual documentation and receipt of all applicable regulatory approvals and the policies of the Canadian Securities Exchange ("CSE"). All securities issued pursuant to the Private Placement are subject to a statutory hold period of four months and one day from the date of issuance. Closing of the Private Placement is conditional upon a number of conditions, including finalizing all contractual documentation and receipt of all applicable regulatory approvals and the policies of the CSE.

The Company intends to use the net proceeds of the Private Placement for general working capital purposes including, enabling the Company to fund and advance its business plans in regard to its successful recent acquisition of the entire portfolio of discovery, preclinical and clinical development stage assets in oncology, infectious disease, antigen desensitization, and other immunological fields based on the DPX immune educating platform technology, developed by the former Canadian biotechnology company, IMV Inc., Immunovaccine Technologies Inc., which was purchased from IMV USA ("IMV") on February 16th, 2024..The Company may pay a finder’s fee related to the financing.

In addition, the Company announces that it intends to fully settle up to a maximum of CAD $733,600 in debt through the issuance of a maximum of 14,672,000 common shares issued at a deemed price of $0.05 per Common Share. The board of directors of the Company has determined that it is in the best interests of the Company to settle the outstanding debts by the issuance of Common Shares in order to preserve the Company’s cash for working capital. The debt settlement is expected to include the participation of certain related parties including, BioVaxys CEO and director, James Passin, BioVaxys COO and President Kenneth Kovan, BioVaxys directors Anthony Dutton and Craig Loverock and BioVaxys consultant Loverrock Consulting Corp., and as such it will constitute a "related party transaction" within the meaning of Multilateral Instrument 61-101 – Protection of Minority Security Holders in Special Transactions ("MI 61-101"). The Company is relying on the exemptions from the valuation and minority shareholder approval requirements of MI 61-101 contained in sections 5.5(a) and 5.7(1)(a) of MI 61-101, as the fair market value of the shares for debt transaction with the forgoing related parties does not exceed 25% of the market capitalization of the Company, as determined in accordance with MI 61-101. Closing of the proposed financing is expected to occur by July 31, 2024.

All securities proposed to be issued in connection with the Debt Settlement will be subject to a statutory hold period of four months plus a day from the date of issuance in accordance with applicable securities legislation. Closing of the Debt Settlement is conditional upon a number of conditions, including finalizing all contractual documentation and receipt of all applicable regulatory approvals and the policies of the CSE.