Pharmacokinetics

Gain Therapeutics’ GT-02287 Completely Restores Motor Function in Mouse Models of Parkinson’s Disease

Retrieved on: 
Tuesday, February 6, 2024

The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.

Key Points: 
  • The data was accepted as a late-breaker abstract and will be presented at the 20th Annual WORLDSymposium™ being held in San Diego this week.
  • “We believe the data showing complete restoration of motor function in a therapeutic model are remarkable and further support the potential of GT-02287 to slow or stop the progression of Parkinson’s disease, a disease for which only symptomatic treatments are available to patients at this time,” said Matthias Alder, Gain Therapeutics’ Chief Executive Officer.
  • Further, animals in the most challenging treatment group – those that began treatment eight days following onset of the disease – showed motor improvement from day 14 to day 27, which suggests progressive reversal of neuronal deficit associated with continued treatment duration.
  • Further details of the study, including protocol and specific results can be found in the poster, which was presented today and can be accessed here .

Scholar Rock Presents New Preclinical Data Demonstrating Potential Benefit of SRK-439 for Healthy Weight Loss Management

Retrieved on: 
Tuesday, February 6, 2024

“These preclinical data showing that SRK-439 preserves lean mass and improves fat mass loss provide compelling scientific rationale to study SRK-439 in combination with GLP-1 RA therapies for healthy weight loss management,” said Jay Backstrom, M.D., MPH, President and CEO of Scholar Rock.

Key Points: 
  • “These preclinical data showing that SRK-439 preserves lean mass and improves fat mass loss provide compelling scientific rationale to study SRK-439 in combination with GLP-1 RA therapies for healthy weight loss management,” said Jay Backstrom, M.D., MPH, President and CEO of Scholar Rock.
  • “We believe that preserving lean muscle mass through our highly selective approach to myostatin inhibition in combination with GLP-1 RA therapy has the potential to transform the management of weight loss.
  • In parallel, Scholar Rock is developing SRK-439, a novel investigational selective myostatin inhibitor, optimized for the treatment of obesity.
  • SRK-439 also improved fat mass loss (–36.60% to –46.32% from baseline with semaglutide; –17.31% to –19.04% from baseline with liraglutide).

Alterity Therapeutics Phase 2 Data Monitoring Committee Recommends Continuing Clinical Trial as Planned After Second Review

Retrieved on: 
Tuesday, February 6, 2024

MELBOURNE, AUSTRALIA AND SAN FRANCISCO, Feb. 06, 2024 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that an independent Data Monitoring Committee (DMC) has completed its second review of trial data and recommended the ATH434-201 Phase 2 study continue as planned. The ATH434-201 clinical trial is a randomized, double-blind, placebo-controlled investigation of ATH434 in patients with early-stage multiple system atrophy (MSA), a rare neurodegenerative disease with no approved treatments to slow or stop its progression.

Key Points: 
  • MELBOURNE, AUSTRALIA AND SAN FRANCISCO, Feb. 06, 2024 (GLOBE NEWSWIRE) -- Alterity Therapeutics (ASX: ATH, NASDAQ: ATHE) (“Alterity” or “the Company”), a biotechnology company dedicated to developing disease modifying treatments for neurodegenerative diseases, today announced that an independent Data Monitoring Committee (DMC) has completed its second review of trial data and recommended the ATH434-201 Phase 2 study continue as planned.
  • The DMC conducted its second prespecified review of unblinded clinical data from study participants.
  • The plan for the DMC to review initial safety data has been cleared with the U.S. Food and Drug Administration.
  • Additional information on the Phase 2 trial can be found by ClinicalTrials.gov Identifier: NCT05109091 .

Vividion Therapeutics starts Phase I clinical trial in advanced solid and hematologic tumors with oral STAT3 inhibitor

Retrieved on: 
Tuesday, February 6, 2024

Vividion Therapeutics, Inc. (Vividion), announced today that it has initiated dosing of patients in a Phase I clinical trial evaluating VVD-130850, an investigational oral STAT3 inhibitor for the treatment of advanced solid and hematologic tumors.

Key Points: 
  • Vividion Therapeutics, Inc. (Vividion), announced today that it has initiated dosing of patients in a Phase I clinical trial evaluating VVD-130850, an investigational oral STAT3 inhibitor for the treatment of advanced solid and hematologic tumors.
  • The start of the trial represents another major milestone for Vividion’s innovative chemoproteomics platform.
  • The Phase I clinical trial will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary anti-tumor activity of VVD-130850 in patients with advanced solid and hematologic tumors as a single-agent and in combination with immune checkpoint inhibition.
  • “We are excited to announce the initiation and dosing of this highly selective STAT3 inhibitor,” said Jenna Goldberg, M.D., Chief Medical Officer of Vividion.

Rani Therapeutics Announces Positive Topline Results from Phase 1 Study of an Oral Anti-Interleukin 12/23 Antibody (RT-111)

Retrieved on: 
Monday, February 5, 2024

ET / 5:30 a.m. PT –

Key Points: 
  • ET / 5:30 a.m. PT –
    SAN JOSE, Calif., Feb. 05, 2024 (GLOBE NEWSWIRE) -- Rani Therapeutics Holdings, Inc. (“Rani Therapeutics” or “Rani”) (Nasdaq: RANI), a clinical-stage biotherapeutics company focused on the oral delivery of biologics and drugs, today announced positive topline results from its Phase 1 clinical study of RT-111, a RaniPill® capsule containing an ustekinumab biosimilar, CT-P43.
  • In the study, RT-111 was well-tolerated and delivered ustekinumab with high bioavailability.
  • “We are highly encouraged by the positive results from our Phase 1 study for RT-111 – our third successfully completed Phase 1 trial using RaniPill® technology.
  • To our knowledge, RT-111 is the first ever oral monoclonal antibody to achieve high bioavailability in humans,” said Talat Imran, Chief Executive Officer of Rani.

Pliant Therapeutics Announces Positive Safety and Exploratory Efficacy Data from the INTEGRIS-PSC Phase 2a Trial of Bexotegrast 320 mg in Patients with Primary Sclerosing Cholangitis and Suspected Liver Fibrosis

Retrieved on: 
Sunday, February 4, 2024

SOUTH SAN FRANCISCO, Calif., Feb. 04, 2024 (GLOBE NEWSWIRE) -- Pliant Therapeutics, Inc. (Nasdaq: PLRX) today announced 12-week interim data from the 320 mg dose group of INTEGRIS-PSC, a multinational, randomized, double-blind, placebo-controlled Phase 2a clinical trial of bexotegrast in patients with primary sclerosing cholangitis (PSC) and suspected moderate to severe liver fibrosis. The 320 mg group met its primary and secondary endpoints demonstrating that bexotegrast was well tolerated over a 12-week treatment period and its plasma concentrations increased with dose. There was no dose relationship for adverse events. Pruritus and cholangitis occurred less frequently on bexotegrast than on placebo.

Key Points: 
  • The trial’s exploratory efficacy endpoints assessed changes in the liver fibrosis markers, Enhanced Liver Fibrosis (ELF) score and PRO-C3 levels, as well as liver biochemistry and magnetic resonance imaging (MRI) of the liver.
  • In addition, MRI imaging continued to show evidence of improved hepatocyte function and bile flow with bexotegrast at the 320 mg dose relative to placebo.
  • INTEGRIS-PSC is a multinational, randomized, dose-ranging, double-blind, placebo-controlled Phase 2a trial evaluating bexotegrast at once-daily oral doses of 40 mg, 80 mg, 160 mg, 320 mg or placebo for 12 weeks in 121 patients with PSC.
  • The 320 mg group enrolled 27 patients in the active arm and added 9 new patients to the pooled placebo arm.

Nurix Therapeutics Announces Publication in the Journal Science Identifying a New Class of BTK Mutations That Are Susceptible to NX-2127, a Novel BTK and IKZF1/3 Degrader

Retrieved on: 
Thursday, February 1, 2024

SAN FRANCISCO, Feb. 01, 2024 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, today announced the publication of a manuscript in the journal Science titled: “Kinase Impaired BTK Mutations Are Susceptible to Clinical Stage BTK and IKZF1/3 Degrader NX-2127” that elucidates a previously unappreciated oncogenic scaffold function of BTK responsible for clinical resistance to enzymatic inhibitors and shows that NX-2127, a potent targeted protein degrader with differentiated activity against BTK and IKZF1/3, can overcome this resistance across a broad range of acquired mutations.

Key Points: 
  • “While BTK inhibitors have positively changed clinical outcomes for patients with B-cell malignancies, the emergence of acquired resistance to these medicines is a growing clinical problem,” said Alexey Danilov, M.D., Ph.D.
  • Professor and Co-Director, Toni Stephenson Lymphoma Center, City of Hope National Medical Center.
  • “With the ability to target BTK inhibitor resistance mutations and achieve clinical responses, we believe that BTK degraders have the potential to become the next dominant class of agents in the significant BTK-targeted therapy field.
  • We look forward to presenting additional clinical data from this program, and from our NX-5948 BTK degrader program, which is also being evaluated in patients with CLL, at future medical meetings.”

Purple Biotech Reaches Recommended Phase 2 Dose for NT219

Retrieved on: 
Thursday, February 1, 2024

REHOVOT, Israel, Feb. 01, 2024 (GLOBE NEWSWIRE) -- Purple Biotech Ltd. ("Purple Biotech" or "the Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that harness the power of the tumor microenvironment to overcome tumor immune evasion and drug resistance, today announced it has determined 100mg/kg is the recommended Phase 2 dose (RP2D) for NT219 in combination with cetuximab in the treatment of head and neck cancer based on its Phase 1/2 dose escalation study (NCT04474470).

Key Points: 
  • REHOVOT, Israel, Feb. 01, 2024 (GLOBE NEWSWIRE) -- Purple Biotech Ltd. ("Purple Biotech" or "the Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that harness the power of the tumor microenvironment to overcome tumor immune evasion and drug resistance, today announced it has determined 100mg/kg is the recommended Phase 2 dose (RP2D) for NT219 in combination with cetuximab in the treatment of head and neck cancer based on its Phase 1/2 dose escalation study (NCT04474470).
  • The Company recently reported that NT219, in combination with cetuximab, demonstrated a dose dependent anti-tumor activity with confirmed partial responses.
  • The Company is now advancing its upcoming Phase 2 Proof of Concept study of NT219 for the treatment of R/M SCCHN.
  • “We look forward to treating more patients with NT219 in a Phase 2 study.”

Sai Life Sciences augments DMPK capabilities to ace large-scale collaborations

Retrieved on: 
Thursday, February 1, 2024

HYDERABAD, India, Feb. 01, 2024 (GLOBE NEWSWIRE) -- To offer global big pharma clients cutting-edge, end-to-end drug metabolism and pharmacokinetics (DMPK) services, Sai Life Sciences, a global Contract Research, Development & Manufacturing Organization (CRO-CDMO) , has expanded its DMPK capabilities .

Key Points: 
  • HYDERABAD, India, Feb. 01, 2024 (GLOBE NEWSWIRE) -- To offer global big pharma clients cutting-edge, end-to-end drug metabolism and pharmacokinetics (DMPK) services, Sai Life Sciences, a global Contract Research, Development & Manufacturing Organization (CRO-CDMO) , has expanded its DMPK capabilities .
  • Making the announcement, Krishna Kanumuri, CEO & Managing Director, Sai Life Sciences, said, “The focus of innovator companies on diversifying their supply chains bodes well for Indian CROs-CDMOs like ours.
  • Sauri Gudlavalleti, Chief Operating Officer said, “The latest expansion of DMPK capabilities augments our integrated drug discovery (IDD) offerings.
  • Sai Life Sciences also has plans to invest in niche areas such as peptides, oligos and large molecules to strengthen its discovery capabilities.

Jnana Therapeutics Announces Positive Clinical Proof of Concept Achieved with JNT-517, a Potential First-in-Class Oral Treatment for PKU

Retrieved on: 
Tuesday, January 30, 2024

BOSTON, Jan. 30, 2024 (GLOBE NEWSWIRE) -- Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today announced positive, statistically significant interim results from its ongoing clinical study of JNT-517 in individuals with phenylketonuria (PKU). JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes. On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517.

Key Points: 
  • JNT-517, a small molecule inhibitor of the phenylalanine (Phe) transporter SLC6A19, is being evaluated as a potential first-in-class oral treatment for PKU across all ages and genotypes.
  • On the basis of these positive interim results, Jnana has adapted the Phase 1b trial design to support the potential for accelerated progression of JNT-517.
  • “There is an urgent need for an oral, safe, and efficacious therapy for the more than 60% of individuals with PKU not currently on therapy.
  • The study dosed its first participant with PKU in August 2023 and is enrolling individuals aged 18 to 65 at clinical sites in the United States and Australia.