orphan drug designation Archives - Medical Design Sourcing

Rilzabrutinib on track for regulatory filing after ITP trial win

Sanofi revealed that its investigational BTK inhibitor rilzabrutinib notched a significant win in the LUNA 3 phase 3 study, hitting the primary endpoint of durable platelet response in adults with persistent or chronic immune thrombocytopenia (ITP). The study showed a significantly higher proportion of rilzabrutinib-treated patients achieved the platelet response goal compared to placebo in this difficult-to-treat ITP population.

“The LUNA 3 results reinforce rilzabrutinib’s potential to provide clinically meaningful benefits for people living with severe immune diseases like ITP,” said Houman Ashrafian, Sanofi’s head of R&D. The company expects to file regulatory submissions for rilzabrutinib in the U.S. and EU by year-end.

Rilzabrutinib received Fast Track Designation from FDA for ITP in November 2020 and also previously won Orphan Drug Designation.

The small molecule became part of Sanofi’s pipeline in 2020 via its $3.6 bil…

Insilico’s AI-discovered INS018_055 graduates to phase 2

Roughly a year ago, Insilico Medicine announced that it had dosed the first patient in a phase 1 study of INS018_055, an AI-discovered, first-in-class small molecule inhibitor. Now, the company has progressed to the next stage, launching a phase 2 study for the drug candidate.

Insilico, a founding member of NVIDIA Inception, developed its AI drug-discovery platform on NVIDIA GPUs. Inception is a complimentary program that provides startups with technical training and AI platform support.

The discovery of INS018_055 was the result of combining multiple technologies. Insilico’s proprietary Pharma.AI platform incorporates AI models trained on massive amounts of data. Insilico’s target identification platform, PandaOmics, discovered a novel target. Then Insilico’s generative chemistry platform, Chemistry42, designed the molecule’s structure. In addition, these AI systems rely on machine learning techniques like deep generative models, reinforcement lear…

Amylyx Pharmaceuticals launches phase 2 trial for AMX0035 Wolfram syndrome therapy

Cambridge, Massachusetts–based Amylyx has dosed the first participant in its phase 2 HELIOS clinical trial of AMX0035 focusing on Wolfram syndrome. The condition is an ultra-rare genetic disorder involving the endocrine system. Symptoms of Wolfram syndrome can include diabetes insipidus, diabetes mellitus, optic atrophy and hearing loss.

In September, Amylyx won FDA approval for AMX0035 (sodium phenylbutyrate and taurursodiol), marketed as Relyvrio, for adults with amyotrophic lateral sclerosis (ALS).

Both sodium phenylbutyrate and taurursodiol are smal molecules demonstrating neuroprotective effects in preclinical studies.

Trial design

The HELIOS is an exploratory open-label proof of biology study. Amylyx anticipates topline results from HELIOS in 2024.

The HELIOS trial is an exploratory open-label, single-group proof of biology study, with Amylyx anticipating topline results in 2024. The trial aims to assess the safety and efficacy of AMX0035 in…