Investigational New Drug

Artiva Biotherapeutics Receives FDA Fast Track Designation for AlloNK® in Lupus Nephritis

Retrieved on: 
Thursday, February 22, 2024

Artiva Biotherapeutics, Inc. , a clinical stage company whose mission is to deliver highly effective, off-the-shelf, allogeneic natural killer (NK) cell-based therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Artiva’s lead program AlloNK® (also known as AB-101) for the treatment of lupus nephritis (LN) in combination with rituximab or obinutuzumab.

Key Points: 
  • Artiva Biotherapeutics, Inc. , a clinical stage company whose mission is to deliver highly effective, off-the-shelf, allogeneic natural killer (NK) cell-based therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Artiva’s lead program AlloNK® (also known as AB-101) for the treatment of lupus nephritis (LN) in combination with rituximab or obinutuzumab.
  • Artiva previously received FDA clearance of an Investigational New Drug (IND) application for AlloNK in combination with rituximab in LN, marking the first IND clearance of an allogeneic, off-the-shelf NK cell therapy in autoimmune disease.
  • AlloNK is a non-genetically modified, allogeneic, cryopreserved NK cell therapy candidate that enhances the activity of B-cell-targeting monoclonal antibodies to drive B-cell depletion.
  • “The FDA Fast Track designation gives us an opportunity to accelerate our efforts to bring our AlloNK cell therapy to autoimmunity patients,” said Fred Aslan, M.D., Chief Executive Officer of Artiva.

ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Study of Inhaled mRNA-Based Genetic Medicine, RCT2100, for the Treatment of Cystic Fibrosis

Retrieved on: 
Wednesday, February 21, 2024

“While progress has been made for many people with CF, those who do not benefit from existing modulators report feeling left behind and continue to face profound physical and emotional hardship from the disease.

Key Points: 
  • “While progress has been made for many people with CF, those who do not benefit from existing modulators report feeling left behind and continue to face profound physical and emotional hardship from the disease.
  • The Phase 1 double-blind, placebo-controlled, first-in-human study is designed to assess the safety and tolerability of a single ascending dose of inhaled RCT2100 administered via nebulizer.
  • The study is being conducted in New Zealand and ReCode anticipates enrolling approximately 32 healthy adults who will receive a single dose of either placebo or RCT2100.
  • ReCode plans to submit an Investigational New Drug application (IND) to the U.S. Food and Drug Administration for a Phase 1 trial of RCT2100 in CF patients in the first half of 2024.

Wheeler Bio Seals Agreement with Charles River to Offer Portable CMC® to Discovery Clients to Accelerate Their Journey to First-in-Human Use

Retrieved on: 
Thursday, February 29, 2024

The collaboration is designed to enable a swift transition from pre-clinical stages directly to human clinical trials.

Key Points: 
  • The collaboration is designed to enable a swift transition from pre-clinical stages directly to human clinical trials.
  • Combining Charles River's industry-leading experience in antibody discovery services, safety, and analytics with Wheeler's Portable CMC® platform will accelerate therapeutic discovery to Investigational New Drug (IND) submission timelines.
  • Wheeler Bio's mission is to solve translational challenges inherent in advancing from discovery to CMC development and early-clinical trial material supply.
  • Jesse McCool, CEO and Co-Founder of Wheeler Bio, stated, "Wheeler is excited to expand our relationship with Charles River and to link with their discovery organization.

Incannex Completes Successful Pre-IND Meeting with the FDA for CannQuit-N; a Novel Solution for Tobacco Smoking Cessation

Retrieved on: 
Friday, February 16, 2024

The pre-IND teleconference included representatives from various divisions of the FDA covering all regulatory aspects of the CannQuit-N development program.

Key Points: 
  • The pre-IND teleconference included representatives from various divisions of the FDA covering all regulatory aspects of the CannQuit-N development program.
  • The Agency had reviewed the CannQuit-N meeting package and provided recommendations on key design aspects of the proposed clinical trials, including the timing of efficacy endpoints and specific details on safety endpoints.
  • The FDA also confirmed proposed strategies for manufacturing and quality control are appropriate.
  • The productive discussion with the FDA is an important milestone for the development of CannQuit-N and will guide the continued development of the drug product.

Inventiva reports preliminary 2023 fiscal year financial Information¹ and provides an update on its clinical trial NATiV3

Retrieved on: 
Thursday, February 15, 2024

Frédéric Cren, Chairman, Chief Executive Officer and cofounder of Inventiva, stated: “2023 has been an eventful year for the company.

Key Points: 
  • Frédéric Cren, Chairman, Chief Executive Officer and cofounder of Inventiva, stated: “2023 has been an eventful year for the company.
  • This allowed us to draw down the second tranche of €25 million of the €50 million EIB loan in January 2024.
  • We have advanced our pivotal NATiV3 Phase III clinical trial with lanifibranor in NASH after the implementation of the revised study design in early 2023, with 913 patients randomized to date.
  • An adverse event of elevated aminotransferases has been reported in a patient enrolled in the trial following a scheduled visit.

OnKure Announces the Appointment of Samuel Agresta, M.D., as Chief Medical Officer

Retrieved on: 
Thursday, February 15, 2024

BOULDER, Colo., Feb. 15, 2024 (GLOBE NEWSWIRE) -- OnKure, Inc. today announced the appointment of Samuel Agresta, M.D., M.P.H.

Key Points: 
  • BOULDER, Colo., Feb. 15, 2024 (GLOBE NEWSWIRE) -- OnKure, Inc. today announced the appointment of Samuel Agresta, M.D., M.P.H.
  • as Chief Medical Officer.
  • “Sam’s leadership experience and expertise in oncology drug development will be invaluable as we advance our portfolio,” said Nick Saccomano, Ph.D., Chief Executive Officer of OnKure.
  • Dr. Agresta also served as a Director and Chief Medical Officer at Infinity Pharmaceuticals, Inc. where he was responsible for the Phase 1 and 2 developments of the PI3Ky inhibitor, IPI-549.

Ampio Provides Update on Results from Pre-IND Enabling Studies

Retrieved on: 
Wednesday, February 14, 2024

ENGLEWOOD, Colo., Feb. 14, 2024 /PRNewswire/ -- Ampio Pharmaceuticals, Inc. (NYSE American: AMPE) (the "Company" or "Ampio"), a pre-revenue stage biopharmaceutical company focused on development of a potential treatment for osteoarthritis as part of its OA-201 program, today announced the efficacy results from recently completed nonclinical pre-IND enabling studies with OA-201, Ampio's only product development opportunity.

Key Points: 
  • ENGLEWOOD, Colo., Feb. 14, 2024 /PRNewswire/ -- Ampio Pharmaceuticals, Inc. (NYSE American: AMPE) (the "Company" or "Ampio"), a pre-revenue stage biopharmaceutical company focused on development of a potential treatment for osteoarthritis as part of its OA-201 program, today announced the efficacy results from recently completed nonclinical pre-IND enabling studies with OA-201, Ampio's only product development opportunity.
  • Ampio reported that the efficacy results of these studies do not support an Investigational New Drug ("IND") submission which was anticipated in early 2025.
  • The Company's management and the Board are currently assessing both internal and external options.
  • The Company will be taking immediate actions to preserve its cash in order to be able to adequately fund any option identified by the Board.

Starton Therapeutics Announces Opening of Second Clinical Site for STAR-LLD Phase 1b Trial in Multiple Myeloma and Provides Study Update

Retrieved on: 
Wednesday, February 14, 2024

The ongoing study evaluates the safety, pharmacokinetics, and efficacy of continuous subcutaneous administration of low-dose lenalidomide (STAR-LLD) in conjunction with dexamethasone and bortezomib (Velcade®).

Key Points: 
  • The ongoing study evaluates the safety, pharmacokinetics, and efficacy of continuous subcutaneous administration of low-dose lenalidomide (STAR-LLD) in conjunction with dexamethasone and bortezomib (Velcade®).
  • Remarkably, the trial is progressing ahead of schedule and with the addition of RMOC is now projected to conclude enrollment by Q1/2024.
  • I’m excited about the potential opportunity STAR-LLD can bring to multiple myeloma patients.”
    Earlier last month, Starton disclosed preliminary trial findings regarding the safety and efficacy of STAR-LLD for multiple myeloma.
  • Notably, no hematologic toxicities greater than Grade 1 have been observed following up to 3 cycles of treatment thus far.

Atara Biotherapeutics Announces Submission of Investigational New Drug Application for ATA3219 for Treatment of Lupus Nephritis

Retrieved on: 
Wednesday, February 14, 2024

“We are dedicated to advancing medical breakthroughs with innovative cell therapies that truly make a difference.

Key Points: 
  • “We are dedicated to advancing medical breakthroughs with innovative cell therapies that truly make a difference.
  • Treatment will be facilitated for patients and physicians in avoiding apheresis and lengthy patient-by-patient manufacturing as ATA3219 would be rapidly available as an off-the-shelf treatment from finished product inventory.
  • The ATA3219 IND submission includes robust in vitro data reflecting the CD19 antigen-specific functional activity of ATA3219 and CAR-mediated activity against B cells from SLE patients.
  • Despite recent advances in treatment strategies, the response rate using existing therapies remains low, with significant risk of long-term morbidity and mortality associated with refractory LN.

Pasithea Therapeutics Announces Opening of Enrollment in the U.S. for its Phase 1 Trial of PAS-004

Retrieved on: 
Tuesday, February 13, 2024

SOUTH SAN FRANCISCO, Calif. and MIAMI, Feb. 13, 2024 (GLOBE NEWSWIRE) -- Pasithea Therapeutics Corp. (NASDAQ: KTTA) (“Pasithea” or the “Company”), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) and other cancer indications, today announced the activation of four clinical trial sites in the United States. These U.S. clinical trial sites in Texas and Virginia are now open and actively enrolling patients.

Key Points: 
  • These U.S. clinical trial sites in Texas and Virginia are now open and actively enrolling patients.
  • The Company's clinical development plan for PAS-004 following the Phase 1 study is to begin a Phase 2 clinical trial in NF1 pediatric and adult patients as soon as safety and PK are established.
  • There are also three other clinical trial sites in Eastern Europe that are expected to open in the coming months.
  • “Activating our four clinical trial sites in the U.S. is a significant milestone in Pasithea’s mission towards developing PAS-004 as a potential best-in-class next-generation MEK inhibitor.