Pridopidine

Prilenia and Ferrer Form €500 Million Partnership to Commercialize Pridopidine for Huntington's Disease in Europe

25 days ago

• Prilenia Therapeutics and Ferrer have signed a collaboration and license agreement worth up to €500 million for pridopidine, a selective sigma-1 receptor agonist targeting Huntington's disease and ALS. • The deal includes an €80 million upfront payment plus €45 million in near-term milestones, with Ferrer gaining commercialization rights across Europe, Middle East, North Africa, and other select markets. • Pridopidine is currently under review by the European Medicines Agency for Huntington's disease treatment, with a regulatory opinion expected in the second half of 2025.

Pridopidine Shows Promising Long-Term Benefits for Huntington's Disease Patients Not on Antidopaminergic Medications

a month ago

• Pridopidine demonstrated significant benefits on clinical progression, cognition, and motor function in Huntington's disease patients not taking antidopaminergic medications during the PROOF-HD phase 3 trial. • The investigational therapy showed sustained efficacy for up to two years, with meaningful improvements in composite UHDRS scores, slowing disease decline compared to placebo. • Pridopidine's safety and tolerability profile was comparable to placebo, offering potential hope in a disease landscape with no current curative options.

Pridopidine Shows Promise for ALS Subgroups in Healey Platform Trial

a month ago

• Pridopidine demonstrated safety and potential efficacy in secondary endpoints for ALS patients, particularly improving motor speech performance, despite not meeting the primary endpoint in the Healey Platform Trial. • The drug showed greater impact in a prespecified subgroup of patients with definite ALS and shorter disease duration (≤18 months), suggesting targeted efficacy in rapidly progressing cases. • The HEALEY platform trial approach reduces placebo assignments, cuts time and costs, and has evaluated seven drugs since 2020, with Pridopidine now advancing to Phase 3 based on promising signals.

Biotech Deal Landscape: February-March 2025 Sees Surge in Partnerships Across Multiple Therapeutic Areas

3 months ago

• The first quarter of 2025 witnessed significant biotech partnership activity, with Eli Lilly, AstraZeneca, and Novo Nordisk emerging as top collaborators in deals worth billions across small molecules, antibodies, and RNA therapeutics. • February 2025 featured notable acquisitions including Novartis's $2.15 billion buyout of Anthos Therapeutics, while March saw AstraZeneca acquire Belgian biotech EsoBiotec and Bristol Myers Squibb purchase 2seventy bio for $286 million. • Obesity therapeutics gained significant traction in March 2025, with AbbVie entering the field through a $350 million upfront deal with Gubra for an amylin analog, while Roche partnered with Zealand Pharma on petrelintide in a deal worth up to $5.25 billion.

Annexon's 2025 Outlook: Key Milestones for Neuroinflammatory Disease Therapies

4 months ago

• Annexon anticipates a Biologics License Application (BLA) submission for ANX005 in the first half of 2025, potentially transforming Guillain-Barré Syndrome (GBS) treatment. • Enrollment in the Phase 3 ARCHER II trial for ANX007 in geographic atrophy (GA) is expected to be completed in the latter half of 2025. • Clinical proof-of-concept data for ANX1502, an oral C1s inhibitor targeting autoimmune conditions, is anticipated in the first quarter of 2025. • Annexon's current cash reserves are projected to sustain operations into the second half of 2026, supporting the achievement of critical developmental milestones.

Denali Therapeutics' DNL343 Fails to Meet Primary Endpoint in HEALEY ALS Platform Trial

5 months ago

• Denali Therapeutics' DNL343, an eIF2B agonist, did not meet the primary endpoint of slowing ALS disease progression in a Phase 2/3 trial. • The HEALEY ALS Platform Trial's Regimen G assessed DNL343's impact on disease severity and survival over 24 weeks compared to placebo. • While DNL343 was safe and well-tolerated, key secondary endpoints like muscle strength and respiratory function showed no significant difference. • Further analyses, including biomarker assessments, are planned for 2025 to explore potential subgroup benefits and long-term effects.

PrimeC Shows Promise in ALS Treatment: 53% Extension in Complication-Free Survival in Phase 2b Trial

5 months ago

NeuroSense's PrimeC demonstrated significant benefits in ALS treatment during a Phase 2b clinical trial, extending complication-free survival by 53% compared to placebo. The therapy also showed improvements in patients' quality of life, leading to discussions with U.S. regulators about Phase 3 trials and potential early approval in Canada.

HEALEY ALS Platform Trial: Streamlining Drug Development for Amyotrophic Lateral Sclerosis

6 months ago

• The HEALEY ALS Platform Trial is pioneering a new approach to accelerate the development of effective treatments for amyotrophic lateral sclerosis (ALS). • By testing multiple drugs simultaneously using a shared infrastructure, the platform trial significantly reduces the time and cost associated with traditional clinical trials. • Initial results from the platform have led to two drugs, CNM-Au8 and pridopidine, advancing to Phase 3 testing based on promising trends in secondary outcome measures and biomarker data. • The collaborative effort unites patients, clinicians, scientists, and industry partners, fostering innovation and improving access to care across a network of over 70 sites.

Huntington's Disease Therapeutics: Promising Advances Amidst Setbacks

6 months ago

• Several companies are advancing potential Huntington's disease treatments, including Wave Life Sciences with WVE-003 and uniQure with AMT-130. • PTC Therapeutics' PTC518 received FDA Fast Track designation after demonstrating reduced mutant huntingtin protein levels in Phase 2 trials. • Prilenia Therapeutics' pridopidine is under EMA review, potentially offering the first treatment to impact Huntington's disease progression. • Despite recent trial failures, the Huntington's disease therapeutic field remains optimistic due to genetic-level targeting advancements.

Huntington's Disease: Disease-Modifying Therapies on the Horizon After Decades of Setbacks

6 months ago

• Several Huntington's disease therapies are nearing regulatory approval, potentially becoming the first disease-modifying treatments for the condition. • Prilenia Therapeutics' pridopidine has an application accepted by the EMA, while Wave Life Sciences and uniQure are in discussions with regulators for accelerated approval pathways. • Wave Life Sciences' WVE-003 demonstrated a significant reduction in mutant HTT protein levels and slowed caudate atrophy in clinical trials. • uniQure's AMT-130 gene therapy showed significant slowing of disease progression and reduction in neurofilament light chain levels in clinical studies.

Huntington's Disease: Promising Therapies Offer Hope Amidst Clinical Challenges

6 months ago

• Prilenia Therapeutics' pridopidine, a sigma-1 receptor agonist, shows potential efficacy in Huntington's patients not taking anti-dopaminergic or chorea medications, awaiting European regulatory review. • Sage Therapeutics' dalzanemdor, a novel NMDA receptor modulator, is under evaluation for cognitive impairment in Huntington's, with Phase II DIMENSION study results anticipated. • uniQure's AMT-130, a microRNA gene therapy, demonstrates dose-dependent slowing of disease progression in a Phase I/II trial, targeting mutant HTT protein production. • Wave Life Sciences' WVE-003, an allele-specific silencing therapy, significantly reduces mutant HTT protein levels, potentially supporting accelerated approval despite partnership changes.

Huntington's Disease Research Sees Renewed Hope with Promising Trial Results

7 months ago

• A phase 1/2 gene therapy trial demonstrated an 80% slowing of disease progression in Huntington's disease (HD) patients, as measured by the composite Unified Huntington's Disease Rating Scale. • Wave Life Sciences' ASO, WVE-003, showed a statistically significant 46% reduction in mutant huntingtin protein, prompting plans for accelerated FDA approval. • Numerous companies are actively developing HD therapeutics, employing diverse approaches based on an expanding understanding of the disease, signaling a dynamic future for HD treatment. • Researchers are exploring innovative strategies like CRISPR-based genome editing and neural progenitor transplantation to counteract HD pathology, expanding therapeutic horizons.

Prilenia's Pridopidine for Huntington's Disease: Hope vs. Hype in Clinical Trials

8 months ago

• Pridopidine has undergone multiple clinical trials for Huntington's disease (HD), with all major endpoints consistently failing to be met, raising concerns about its efficacy. • Recent interpretations of the PROOF-HD trial results by Prilenia Therapeutics have been controversial, emphasizing subgroup benefits while downplaying overall negative outcomes. • The EMA has accepted Prilenia's application for marketing authorization review, but this step is routine and doesn't guarantee approval, warranting caution against undue hype. • Concerns arise regarding potential influence on treatment decisions, particularly regarding neuroleptic use, urging transparent and balanced reporting of clinical trial results.

Prilenia's Pridopidine Under EMA Review for Huntington's Disease Treatment

8 months ago

• The EMA is reviewing Prilenia Therapeutics' application for pridopidine, an oral therapy for Huntington's disease, based on clinical data suggesting it may slow disease progression. • Pridopidine targets the sigma-1 receptor, aiming to modulate cellular pathways crucial for nerve cell function, potentially easing symptoms and slowing the progression of Huntington's disease. • While a Phase 3 trial showed mixed results, subgroup analyses indicated benefits in patients not taking certain medications, supporting the application for approval. • If approved, pridopidine could be available in Europe by the second half of 2025, offering a new therapeutic approach to slow disease progression for Huntington's patients.

ALS Clinical Trial Landscape: Investigational Therapies Targeting Sporadic ALS

2 years ago

• Several clinical trials are underway for sporadic ALS, targeting TDP-43 pathology, STMN2 expression, and neuroinflammation. • Biogen's BIIB-105, AbbVie/Calico's ABBV-CLS-7262, Denali's DNL-343, and QurAlis' QRL-201 are among the therapies currently being evaluated in clinical trials. • Prilenia's Pridopidine and Clene's CNM-Au8 have completed Phase 2 trials, showing some positive trends in motor function and survival benefits, respectively. • The FDA is set to make a decision on Brainstorm Cell's NurOwn, a mesenchymal stem cell therapy, with an advisory committee meeting scheduled for September 27th.

New Drug Development Pipeline Shows Promise for ALS Treatment with 53 Candidates Under Investigation

2 years ago

• A comprehensive review of ALS drug development reveals 53 new drug candidates in clinical trials between 2020-2022, with 13 compounds advancing to Phase 3 trials and showing promising therapeutic potential. • Five drugs demonstrated particularly strong efficacy: high-dose methylcobalamin, masitinib, AMX0035, CNM-Au8, and tofersen, with AMX0035 receiving FDA approval as the third treatment for ALS. • The success of clinical trials is influenced by patient population homogeneity, observation duration, and analysis strategies, highlighting the importance of refined trial design in ALS drug development.

Drug Development Updates