Omaveloxolone

Larimar Therapeutics Advances Nomlabofusp Clinical Trials for Friedreich's Ataxia

4 months ago

• 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.

Biogen Executives Address Investor Pressure and Strategic Focus at JPM25

4 months ago

At the J.P. Morgan Healthcare Conference, Biogen executives discussed the pressure from investors for new deals amidst a declining stock price. They emphasized the company's focus on growth, scientific conviction, and strategic acquisitions, highlighting recent milestones and the disciplined approach to business development.

Biogen and Stoke Therapeutics Collaborate to Advance Zorevunersen for Dravet Syndrome

5 months ago

• Biogen and Stoke Therapeutics will collaborate to develop and commercialize zorevunersen for Dravet syndrome outside the U.S., Canada, and Mexico. • Stoke's Phase 3 EMPEROR study of zorevunersen, expected to begin in mid-2025, aims to reduce seizure frequency and improve cognition and behavior in children with Dravet syndrome. • Zorevunersen, an antisense oligonucleotide targeting the SCN1A gene, has shown promising results in earlier trials, including an 87% median reduction in convulsive seizure frequency. • Biogen will provide Stoke with an upfront payment of $165 million, potential milestone payments up to $385 million, and tiered royalties on net sales in Biogen's territory.

Omaveloxolone Shows Sustained Safety and Efficacy in Friedreich Ataxia Treatment

5 months ago

• Long-term data from the MOXIe trial extension confirms omaveloxolone's favorable safety profile, with manageable gastrointestinal side effects and transient transaminase elevations. • Analyses from ICAR 2024 demonstrate omaveloxolone's efficacy across various age groups, reinforcing its potential to benefit a broad range of Friedreich ataxia patients. • The SKYCLARYS PASS registry will track real-world safety and effectiveness, providing insights into the drug's performance beyond clinical trial settings. • Clinicians can mitigate gastrointestinal adverse events by initiating treatment with lower doses and gradually increasing to the target dose of 150 mg.

FDA Grants Priority Review to Vatiquinone for Friedreich's Ataxia Treatment

5 months ago

• The FDA has accepted PTC Therapeutics' NDA for vatiquinone, a potential treatment for Friedreich's ataxia (FA), granting it priority review. • Vatiquinone aims to address the unmet need for FA patients, particularly children, by targeting energetic and oxidative stress pathways. • The NDA is supported by data from the MOVE-FA study and long-term studies, showing slowed disease progression and a favorable safety profile. • The FDA's target action date for a decision on vatiquinone is set for August 19, 2025, marking a significant step toward potential approval.

Solid Biosciences' SGT-212 Receives FDA Fast Track Designation for Friedreich's Ataxia Treatment

5 months ago

• Solid Biosciences' SGT-212, a dual-route gene therapy for Friedreich's ataxia (FA), has received Fast Track designation from the FDA, expediting its development and review process. • SGT-212 delivers the frataxin gene via intravenous and direct intradentate nucleus infusions, targeting both neurological and cardiac manifestations of FA. • A Phase 1b clinical trial is planned for the second half of 2025 to assess the safety and tolerability of SGT-212 in adult FA patients. • The Fast Track designation will allow Solid Biosciences to have more frequent interactions with the FDA and the potential to be eligible for priority review.

Long-Term Safety of Omaveloxolone in Friedreich Ataxia Highlighted

6 months ago

• A long-term safety analysis of omaveloxolone, the first FDA-approved treatment for Friedreich ataxia, shows a positive safety profile, offering reassurance for its use. • Updated Restless Legs Syndrome (RLS) guidelines for 2024 include major changes that clinicians should be aware of, impacting diagnosis and treatment strategies. • Advancements in blood biomarker tests for Alzheimer's disease are improving early diagnosis and treatment intervention, enhancing patient care. • Shared decision-making is crucial when selecting FDA-approved therapies for NMOSD, considering patient characteristics, safety, administration, and cost-effectiveness.

Omaveloxolone Shows Positive Long-Term Safety Profile in Friedreich Ataxia

6 months ago

• Long-term analysis of omaveloxolone demonstrates a favorable safety profile for treating Friedreich ataxia, with most adverse events being manageable. • The most common treatment-emergent adverse events (TEAEs) include gastrointestinal issues and elevated aminotransferase levels, typically occurring within the first 12 weeks. • Gradual dose titration of omaveloxolone over several months helps manage metabolic effects, allowing most patients to reach the full dose without significant complications. • Omaveloxolone, approved in early 2023, remains the first and only FDA-approved treatment for Friedreich ataxia, impacting approximately 1 in 40,000 people globally.

Sage Therapeutics' Dalzanemdor Fails in Huntington's Disease Trial, Pipeline Thins

6 months ago

• Sage Therapeutics' dalzanemdor failed in the Phase II DIMENSION trial for Huntington's disease, leading to the discontinuation of its development. • This setback follows previous failures of dalzanemdor in Parkinson's and Alzheimer's, further impacting Sage's clinical pipeline. • Sage is now heavily reliant on Zurzuvae for postpartum depression, with strategic reorganizations aimed at supporting its commercial launch. • Analysts express concerns about Sage's path to profitability despite cost-cutting measures and the initial success of Zurzuvae.

SKYCLARYS PASS Registry to Monitor Long-Term Safety of Omaveloxolone in Friedreich Ataxia

6 months ago

• The SKYCLARYS PASS registry (NCT06623890) will evaluate the long-term safety and patient experience with omaveloxolone (Skyclarys) in Friedreich ataxia (FA) patients. • The observational, multi-country cohort registry will enroll approximately 300 omaveloxolone-naïve patients from the UNIFIED natural history study for up to 5 years. • Primary outcomes focus on long-term safety, including drug-induced liver injury (DILI) and congestive heart failure (CHF), with interim analyses reported to regulatory agencies.

Biogen's Growth Prospects Face Challenges Amidst Leqembi's Slow Uptake

6 months ago

• Needham downgraded Biogen, projecting flat revenue until 2026 due to slow sales growth of Alzheimer's drug Leqembi and the time needed for new products to offset declines. • Leqembi's global sales reached $67 million in Q3, with $39 million from the U.S., but physician feedback suggests a continued gradual increase in sales, tempering market expectations. • Skyclarys' Q4 sales are projected to reach $116 million, with $608 million expected for 2025, while Zurzuvae's Q3 sales were reported at $22 million, contributing to Biogen's revenue diversification. • Despite a positive recommendation for Leqembi in the EU, Needham anticipates Biogen's revenues to remain flat or slightly decline until 2026, leading to a downgrade of the stock.

Biogen's Revenue Growth Stalled: Analyst Downgrade Cites Slow Leqembi Uptake

6 months ago

• Needham downgraded Biogen, projecting flat revenue until 2026 due to limited growth catalysts and slow sales of Alzheimer's drug Leqembi. • Leqembi's Q3 global sales reached $67 million, with $39 million in the U.S., but physician feedback suggests a continued gradual uptake. • Skyclarys is projected to reach $116 million in Q4 sales and $608 million in 2025, while Zurzuvae's Q3 sales were $22 million. • Despite EMA's positive opinion on lecanemab, Biogen faces challenges in meeting market expectations and offsetting declines in existing business.

Omaveloxolone's Safety Profile in Friedreich Ataxia: TEAE Analysis from MOXIe Trial

6 months ago

• Analysis of the MOXIe trial reveals that most treatment-emergent adverse events (TEAEs) associated with omaveloxolone occurred within the first 12 weeks of treatment. • Elevated aminotransferase levels (AST/ALT) and gastrointestinal disorders were the most frequently observed TEAEs in patients treated with omaveloxolone. • The study suggests that the transient increase in aminotransferases may be linked to beneficial metabolic adaptations driven by Nrf2 activation. • Findings emphasize the importance of dosing compliance and managing patient expectations regarding potential side effects during omaveloxolone treatment.

Biogen Raises 2024 Earnings Outlook as New Drug Portfolio Shows Promise

7 months ago

• Biogen has increased its 2024 earnings per share guidance to $16.10-$16.60, driven by strong performance from new products including Leqembi, which generated $67 million in Q3 revenue. • The company's transformation under CEO Chris Viehbacher continues with strategic focus on cost savings of $800 million by 2025 and pipeline diversification beyond neuroscience. • Despite declining multiple sclerosis drug sales, Biogen's new product launches, including Zurzuvae and Skyclarys, are helping offset revenue losses in core business segments.

PTC Therapeutics Plans FDA Submission for Vatiquinone in Friedreich's Ataxia

7 months ago

• PTC Therapeutics plans to submit a New Drug Application (NDA) to the FDA for vatiquinone to treat Friedreich's Ataxia by the end of the year. • The submission is supported by positive data from the Phase 2/3 MOVE-FA trial and extension studies, showing a slower decline in mFARS scores compared to untreated patients. • Vatiquinone demonstrated a strong safety profile and potential benefits, particularly in children and adolescents, where there are currently limited approved therapies. • The planned submission has been welcomed by the Friedreich's Ataxia Research Alliance, citing the high unmet need, especially in the pediatric population.

Larimar Therapeutics' Nomlabofusp Shows Promise in Friedreich's Ataxia Treatment

7 months ago

• 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.

UCB and Biogen's Dapirolizumab Pegol Shows Promise in Phase 3 Lupus Trial

8 months ago

• UCB and Biogen's dapirolizumab pegol met the primary endpoint in a Phase 3 trial for moderate-to-severe systemic lupus erythematosus (SLE). • The drug demonstrated significant clinical improvement in disease activity compared to placebo when added to standard of care. • A second Phase 3 trial, PHOENYCS FLY, is planned to further evaluate the efficacy and safety of dapirolizumab pegol in SLE patients. • Dapirolizumab pegol targets the CD40L pathway, offering a novel approach to reduce B cell activation and autoantibody production in SLE.

Health Canada Grants Priority Review to Biogen's Omaveloxolone for Friedreich's Ataxia

8 months ago

• Health Canada has accepted Biogen's New Drug Submission for omaveloxolone under priority review for treating Friedreich's ataxia in patients aged 16 and older. • Omaveloxolone, a once-daily oral medication, could become the first disease-specific treatment option available for this rare and debilitating neuromuscular condition. • The submission is supported by data from the MOXIe Part 2 trial, which demonstrated significant improvements in mFARS scores compared to placebo at 48 weeks. • A regulatory decision from Health Canada regarding the approval of omaveloxolone is anticipated in early 2025, offering hope for Canadian patients with Friedreich's ataxia.

Evaluating mFARS in Pediatric Friedreich's Ataxia: Insights from the FACHILD Study

a year ago

The FACHILD study focuses on evaluating the modified Friedreich Ataxia Rating Scale (mFARS) in pediatric patients with Friedreich's ataxia (FRDA), a rare hereditary condition. The study highlights the rapid disease progression in children, the challenges in assessing disease status using mFARS, and the need for pediatric trials to explore therapeutic interventions. It also discusses the impact of the COVID-19 pandemic on data collection and the importance of FARS E (Upright Stability) as a predictor of disease progression.

Real-World Evidence Transforms Drug Development: FDA Embraces New Paradigm in Clinical Research

6 years ago

• Real-world evidence (RWE) is revolutionizing drug development by providing crucial data from patient experiences outside traditional clinical trials, helping bring safer and more effective treatments to market. • The FDA has demonstrated strong commitment to RWE adoption, exemplified by Pfizer's Ibrance receiving expanded indication for male breast cancer based solely on real-world data. • RWE is being utilized across pharmaceutical functions, from informing clinical trial design to supporting market access decisions and value-based care initiatives worldwide.

Drug Development Updates