PureTech-invented KarXT receives FDA approval for the treatment of schizophrenia in adults
- KarXT (xanomeline and trospium chloride), invented by PureTech, has received FDA approval for treating schizophrenia in adults.
- The approval triggers milestone payments totaling $29 million to PureTech under agreements with Royalty Pharma and Karuna Therapeutics.
- PureTech is entitled to potential future payments and approximately 2% royalties on net annual sales over $2 billion.
- KarXT, now marketed as Cobenfy, will be under the stewardship of Bristol Myers Squibb following their acquisition of Karuna Therapeutics.
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FDA approves new mechanism of action drug for schizophrenia treatment
- The FDA approved Cobenfy (xanomeline chloride and trospium) capsules for oral use in treating schizophrenia in adults.
- Cobenfy is the first antipsychotic approved to act on cholinergic receptors instead of dopamine receptors.
- Efficacy was evaluated in two multicenter, randomized, double-blind, placebo-controlled studies over 5 weeks.
- Participants receiving Cobenfy showed significant symptom reduction compared to placebo, measured by the PANSS score.
- Cobenfy has warnings for potential side effects like urinary retention, increased heart rate, and angioedema.
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FDA approves Bristol Myers Squibb’s COBENFY for schizophrenia treatment
- The FDA has approved COBENFY (xanomeline and trospium chloride) for treating schizophrenia in adults.
- COBENFY is the first new class of medicine for schizophrenia in decades, targeting M1 and M4 receptors without blocking D2 receptors.
- Approval is based on data from the EMERGENT clinical program, including three placebo-controlled trials and two open-label trials.
- In Phase 3 trials, COBENFY showed significant reductions in schizophrenia symptoms compared to placebo, with common side effects including nausea, dyspepsia, and constipation.
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BlueRock Therapeutics’ investigational cell therapy bemdaneprocel for Parkinson’s disease shows positive data at 24 months
- BlueRock Therapeutics announced positive 24-month data from the exPDite Phase 1 trial of bemdaneprocel for Parkinson's disease.
- The trial assessed the safety and tolerability of bemdaneprocel, showing it to be well-tolerated with no adverse events related to the therapy.
- High dose cohort showed a mean reduction of 21.9 points in motor symptoms using MDS-UPDRS Part III, compared to 8.3 points in the low dose cohort.
- Participants in the high dose cohort experienced a mean increase of 1.8 hours in 'Good ON' state time and a mean decrease of 1.9 hours in 'OFF' state time.
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Septerna initiates phase 1 trial for SEP-786 in hypoparathyroidism
- Septerna has started dosing participants in a Phase 1 clinical trial for SEP-786, an oral small molecule PTH1R agonist.
- The trial includes single-ascending dose (SAD) and multiple-ascending dose (MAD) phases to assess safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD).
- SEP-786 aims to treat hypoparathyroidism, a condition caused by parathyroid hormone deficiency, leading to severe symptoms and complications.
- The trial will enroll up to 180 healthy adults, with the SAD phase currently underway.
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Enliven Therapeutics announces positive data update from Phase 1 clinical trial of ELVN-001 in chronic myeloid leukemia
- Updated Phase 1 data presented at ESH-iCMLf 26th Annual John Goldman Conference.
- Cumulative MMR rate of 44% (8/18) by 24 weeks, with stable or deepening responses between weeks 12 and 24.
- ELVN-001 remains well-tolerated with no dose reductions reported; 39 patients enrolled with a median treatment duration of 20 weeks.
- ELVN-001 is a highly selective kinase inhibitor targeting the BCR-ABL gene fusion, showing potential as a best-in-class therapy.
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Estrella Immunopharma achieves complete response in first patient treated with CD19-redirected ARTEMIS T-cells
- Estrella Immunopharma announced a complete response in the first patient treated with EB103 CD19-redirected ARTEMIS T-cells in the STARLIGHT-1 Phase I/II trial.
- The patient, diagnosed with high-risk follicular lymphoma grade 3A, showed no detectable signs of disease one month post-treatment.
- No treatment-related serious adverse events (SAEs) were observed, despite the patient's high tumor burden and multiple comorbidities.
- The STARLIGHT-1 trial aims to assess the safety and determine the Recommended Phase II Dose (RP2D) of EB103 in relapsed/refractory B-cell NHL patients.
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Adicet opens enrollment for ADI-001 phase 1 clinical trial in autoimmune diseases
- Adicet Bio has started enrolling patients for the Phase 1 clinical trial of ADI-001 in autoimmune diseases.
- The trial will include patients with lupus nephritis (LN), systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and ANCA-associated vasculitis (AAV).
- The study has three arms, with patients receiving a single dose of ADI-001 and undergoing safety assessments at multiple intervals.
- ADI-001 has been granted Fast Track Designation by the FDA for relapsed/refractory class III or class IV LN.
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Precision BioSciences submits first clinical trial applications for PBGENE-HBV
- Precision BioSciences has submitted Clinical Trial Applications (CTA) to initiate a Phase 1 study for PBGENE-HBV.
- PBGENE-HBV aims to potentially cure chronic hepatitis B by eliminating cccDNA and inactivating integrated HBV DNA in hepatocytes.
- The regulatory submissions are supported by robust non-human primate safety studies and efficacy in preclinical models.
- The company plans to initiate the Phase 1 study soon and expects to report data in 2025.
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Prime Medicine announces strategic research collaboration and license agreement with Bristol Myers Squibb
- Prime Medicine and Bristol Myers Squibb to develop and commercialize ex vivo T-cell therapies.
- Prime Medicine to receive $110 million upfront, with potential for over $3.5 billion in milestones.
- Prime Medicine will design Prime Editor reagents; Bristol Myers Squibb will handle development and commercialization.
- Collaboration leverages Prime Medicine’s PASSIGE technology for precise gene editing.
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