Vatiquinone

Taylor Tieden for BioSpace Arguably the FDA’s most anticipated decision this month is for a subcutaneous induction formulation of Biogen and Eisai’s Alzheimer’s drug Leqembi, which, according to Eisai, could “help reduce the burden on healthcare professionals and patients.” August is shaping up to be another busy and back-heavy month for the FDA, with at least eight target action dates lined up, including for some drugs that could open up completely new treatment pathways for rare diseases. Read below for more. Insmed Builds Up to First Bronchiectasis Approval New Jersey’s Insmed is proposing its oral DPP1 blocker brensocatib for the treatment of bronchiectasis, a chronic lung disease involving inflamed airways. The FDA is expected to decide on the matter on or before Aug. 12. If approved, brensocatib will be the first drug for bronchiectasis, and will open up the inhibition of DPP1 as a therapeutic mechanism. Insmed’s application is backed by the Phase III ASPEN study. Toplined in May 2024 , ASPEN showed that 10-mg brensocatib cut the annualized pulmonary exacerbation rate by 21.1% versus placebo. A 25 mg dose, meanwhile, led to a 19.4% drop. Brensocatib also hit the study’s secondary outcomes, including lung function, time to first exacerbation event and risk of exacerbations over 52 weeks. Tonix’s Fibromyalgia Drug Nears Verdict By Aug. 15, the FDA will release its decision on Tonix Pharmaceuticals’ non-opioid candidate TNX-102 SL, being proposed for the management of fibromyalgia. If it gets the go-ahead, TNX-102 SL would be the first therapy approved for this indication in more than 15 years, according to a March press release from the company. Designed to be taken orally via a tablet under the tongue, TNX-102 SL is a novel formulation of cyclobenzaprine hydrochloride, a non-opioid drug. Data from two pivotal trials, published in April, showed that the drug candidate can significantly reduce pain and elicit other clinical improvements, while also being tolerable. PTC Pushes Vatiquinone Forward in Friedrich’s Ataxia Despite Phase III Fail Fresh off the approval of its phenylketonuria therapy Sephience, PTC Therapeutics is approaching another decision date, this time for vatiquinone, an investigational drug for Friedreich’s ataxia. The FDA’s verdict is due Aug. 19. Vatiquinone is an investigational small-molecule drug that selectively blocks the 15-lipoxygenase enzyme, which regulates pathways involved in oxidative stress that are dysfunctional in Friedreich’s ataxia. The registration-directed late-stage MOVE-FA trial fell short of its primary endpoint, unable to significantly slow disease progression versus placebo. This study, nonetheless, formed part of PTC’s application for vatiquinone. Two long-term studies in pediatric and adult patients also support vatiquinone’s application. Ionis Targets HAE Prophylaxis Nod for Donidalorsen By Aug. 21, the FDA will release its decision on Ionis Pharmaceuticals’ proposal to use the RNA-targeting therapy donidalorsen to prevent attacks in patients with hereditary angioedema (HAE), a rare genetic disease that manifests as recurring episodes of severe swelling throughout the body. Ionis is backing its application with data from the Phase III OASIS-HAE and OASISplus trials. Data from the first trial, published in May 2024 , showed that monthly treatment with donidalorsen could cut HAE attacks by 81% versus placebo over a 25-week observation period. The second study, meanwhile, demonstrated that switching to donidalorsen from other prophylactics reduced the monthly HAE attack rate by 62%, as per a July readout . Donidalorsen works by reducing levels of the prekallikrein protein, which is involved in the inflammatory cascade. Outlook Eyes AMD Approval for New Avastin Formulation Outlook Therapeutics is advancing ONS-5010, an eye drop formulation of the anti-VEGF antibody bevacizumab—approved as an intravenous infusion under the brand name Avastin for various cancers—for the treatment of wet age-related macular degeneration. The FDA is scheduled to release its decision on Aug. 27. To support its regulatory application, Outlook conducted the Phase III NORSE EIGHT study, which in January showed that ONS-5010 could match Roche’s Lucentis in terms of improving visual acuity at 8 weeks. Retinal thickness, an indicator of anatomic response, was likewise comparable between patients treated with ONS-5010 and Lucentis. The FDA previously rejected ONS-5010, flagging in August 2023 a lack of evidence to support the drug’s efficacy. If approved, Outlook plans to market ONS-5010 under the brand name Lytenava. Precigen Anticipates First Approval in Recurrent Respiratory Papillomatosis Maryland’s Precigen is advancing the gene therapy PRGN-2012, also called zopapogene imadenovec, for the treatment of recurrent respiratory papillomatosis (RRP). The FDA is expected to render a verdict on Aug. 27. RRP is a rare and lifelong neoplastic lung disease affecting the upper and lower respiratory tract after HPV infection. Patients suffer from benign tumors growing in their air passages, causing voice hoarseness, difficulty swallowing and breathing problems. RRP can be fatal, according to Precigen, and there are currently no cures. PRGN-2012 induces the immune system to target cells infected with HPV 6 or HPV 11. Data from a pivotal Phase I/II study showed that the gene therapy can elicit more than a 50% complete response in RRP patients, while more than 85% of patients saw reductions in surgical interventions. If approved, PRGN-2012 will be the first treatment for RRP. Sanofi Targets Rare Disease Approval for Rilzabrutinib The FDA is currently reviewing Sanofi’s application for BTK inhibitor rilzabrutinib for the treatment of immune thrombocytopenia (ITP). A regulatory decision is expected on Aug. 29. ITP is a rare autoimmune disorder that manifests as low platelet counts arising from both heightened destruction and reduced production of platelets. Common symptoms include bruising and bleeding that, in more severe instances, can become life-threatening, such as in the case of intracranial hemorrhage. Rilzabrutinib, an orally available BTK blocker, helps address ITP by tamping down several inflammatory and immune pathways. Phase III data presented at the 2024 meeting of the American Society of Hematology last December showed that 65% of treated patients demonstrated platelet response , defined as a doubling of platelets from baseline and hitting a certain count threshold. This response was durable for at least eight of the last 12 weeks of the study in 23% of patients. Rilzabrutinib likewise resulted in bleeding improvements and lowered the need for rescue therapy versus placebo. Biogen, Eisai’s Subcutaneous Leqembi Could Reduce Treatment Burden The FDA will cap off the month with arguably its most closely watched regulatory action: A verdict , due on Aug. 31, for Biogen and Eisai’s proposed subcutaneous maintenance formulation of their anti-amyloid Alzheimer’s therapy Leqembi. If approved, the partners expect an under-the-skin delivery method could minimize the need for hospital visits and, in turn, “help reduce the burden on healthcare professionals and patients,” according to a February 2025 company presentation from Eisai. The companies are also planning a regulatory filing for a subcutaneous induction formulation of Leqembi next year. Data from the open-label extension phase of the Phase III Clarity AD study supported the subcutaneous application. Results in October 2023 showed that weekly injections under the skin were able to remove 14% more amyloid plaques than biweekly infusions. Subcutaneous Leqembi also appeared to be as safe as the intravenous formulation, with comparable rates of amyloid-related imaging abnormalities.