相关临床试验
8
0 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2005
已完成
5
62.5%
招募中
2
25.0%
终止
1
12.5%
暂无批准数据
- Pomerantz LLP is investigating Larimar Therapeutics for potential securities fraud related to nomlabofusp, a subcutaneously injected treatment for Friedreich's ataxia. - On June 29, 2026, Larimar disclosed that anaphylaxis occurred in 10 of 43 patients in its ongoing open-label study, triggering a 12.57% stock decline. - The company submitted the first section of a rolling Biologics License Application seeking accelerated approval despite the ongoing safety signal. - A prior disclosure on September 29, 2025, had already revealed anaphylaxis in seven participants, causing a 33.66% single-day stock drop.
- Larimar Therapeutics is preparing to submit a Biologics License Application (BLA) for Nomlabofusp in Q2 2026, targeting an accelerated approval pathway using frataxin concentration in skin as a surrogate endpoint. - The company has addressed previous anaphylaxis safety concerns through revised dosing protocols and FDA-approved study design adjustments, with further safety data expected from ongoing Phase 2 extension studies. - With $175.4 million in cash reserves sufficient through Q4 2026, Larimar has completed a strategic capital restructuring with Blue Owl and maintains FDA Fast Track and Rare Pediatric Disease designations for Nomlabofusp. - Analyst consensus rates the company as a "Strong Buy" with price targets exceeding $16, significantly above the current $3.45 trading price, amid speculation of potential acquisition interest from companies like Biogen.
- Larimar Therapeutics is progressing with clinical trials of nomlabofusp, targeting adolescent patients with Friedreich's ataxia, with potential data expected in mid-2025. - The FDA's acceptance of dose escalation in pediatric patients suggests confidence in nomlabofusp's safety profile, supporting a BLA submission planned for the second half of 2025. - Analysts maintain a positive outlook on Larimar, citing the potential of nomlabofusp to address the root cause of Friedreich's ataxia and fill a market gap, especially for younger patients. - Larimar's strong cash position is expected to fund operations into 2026, supporting the ongoing clinical development and regulatory processes for nomlabofusp.
• The FDA's START program, designed to expedite rare disease therapy development, has chosen its first candidates for enhanced guidance and support. • Denali Therapeutics' DNL12 for mucopolysaccharidosis IIIA, Neurogene's NGN-401 for Rett syndrome, and Larimar Therapeutics' nomlabofusp for Friedreich's ataxia are among the selected therapies. • Grace Science's GS-100 gene therapy for NGLY1 deficiency is also included, marking a significant step forward in addressing this life-threatening condition with no approved treatments. • The START program aims to provide comprehensive support in clinical trial design and data generation, facilitating efficient development of potentially life-saving therapies.
- Larimar Therapeutics' nomlabofusp demonstrates positive initial data in a long-term open label extension study for Friedreich's Ataxia, showing increased frataxin levels. - The company has initiated dosing of adolescents in a pediatric pharmacokinetic run-in study, using a weight-based dose equivalent to the 50 mg adult dose. - Modeling predicts that 50 mg daily of nomlabofusp could achieve frataxin levels ≥ 50% of those in healthy controls, potentially impacting metabolic pathways disrupted by the disease. - Larimar remains on track to initiate a registrational study in mid-2025 and plans to submit a Biologics License Application in the second half of 2025.
- Nomlabofusp treatment demonstrates modification of gene expression and lipid profiles, alongside increased frataxin (FXN) levels in Friedreich's ataxia (FA) patients. - Modeling predicts daily 50 mg nomlabofusp dosage could achieve FXN levels ≥50% of healthy controls in most FA patients, similar to asymptomatic carriers. - Initial data from the open-label extension study shows that 25mg of nomlabofusp increased and maintained tissue FXN levels over time. - Early trends suggest potential clinical benefits across various outcomes with long-term 25 mg daily nomlabofusp, supporting its therapeutic potential.
- Larimar Therapeutics reported a Q3 2024 net loss of $15.5 million, with a strong cash position of $203.7 million, expected to last into 2026. - The company's lead program, nomlabofusp for Friedreich's ataxia, is progressing with a program update expected in mid-December 2024. - Larimar plans to initiate a PK run-in study in adolescents by the end of 2024 and a global confirmatory study by mid-2025, targeting BLA submission in 2H 2025. - The company received ILAP designation from the MHRA, aiming to accelerate market access for nomlabofusp in the UK.
- Larimar Therapeutics anticipates a mid-December update on its nomlabofusp program, including safety, pharmacokinetic, and frataxin data from the ongoing open-label extension study. - The company is on track to initiate a PK run-in study in adolescent Friedreich's ataxia patients by the end of 2024, marking a step towards pediatric evaluation. - A global confirmatory/registration study is planned for mid-2025, with a Biologics License Application submission targeted for the second half of 2025. - Larimar's strong financial position, with $203.7 million in cash reserves, is expected to support operations into 2026, facilitating continued development efforts.
- Larimar Therapeutics presented positive early clinical trial results for nomlabofusp (CTI-1601) in Friedreich's ataxia (FRDA) patients, highlighting its potential to address frataxin deficiency. - A Phase 2 dose exploration trial demonstrated that nomlabofusp increased frataxin levels in FRDA patients, with the 50-mg dose group showing the most significant improvement over 28 days. - An ongoing open-label extension study, initiated in March 2024, is evaluating the long-term effects of nomlabofusp, including clinical efficacy and frataxin concentrations, with preliminary data expected in Q4 2024. - Larimar Therapeutics aims for accelerated approval and plans to submit a Biologics License Application (BLA) by mid-2025, positioning nomlabofusp as a potential protein replacement therapy in the FRDA pipeline.
• Industry leaders from Stealth BioTherapeutics, Cohbar, and GenSight Biologics will gather at the exclusive Mitochondria-Targeted Drug Development Digital Summit to share breakthrough research developments. • The summit will showcase innovative approaches including Mitokinin's PINK1 activation for Parkinson's disease and Larimar Therapeutics' CTI-1601 protein replacement therapy for Friedreich's Ataxia. • Over 80 key industry experts will participate in 20+ hours of content and workshops, focusing on advancing therapeutics for mitochondrial and age-related diseases.
