Omaveloxolone

The FDA lifted the partial clinical hold after reviewing Phase II dose escalation data. Image credit: Shutterstock/ Ismail Rajo. Larimar Therapeutics ’ clinical programme investigating nomlabofusp (CTI-1601) for the treatment of a neurodegenerative movement disorder is back on track after the US Food and Administration (FDA) lifted a partial clinical hold. Larimar is testing nomlabofusp in an open-label extension study for the treatment of patients with Friedreich’s ataxia – a genetic condition that causes progressive nervous system damage and movement impairment. The FDA lifted the partial clinical hold following a review of Phase II dose exploration data (NCT05579691), according to a 20 May press release by the US company. The FDA imposed a full clinical hold on the programme back in 2021 due to safety concerns with higher doses of the drug causing deaths in preclinical studies with non-human primates. In 2022, the FDA lifted the full clinical hold but kept a partial clinical hold, limiting trials to the 25mg dose. Shares in the biotech rose following the FDA’s decision, up 10% when the market opened on 21 May compared to the pre-announcement market close. See Also: FDA grants approval for two Eylea biosimilars for eye conditions FDA grants breakthrough therapy status for Nuvalent’s NVL-655 Nomlabofusp is a recombinant fusion protein that delivers human frataxin to cells. Patients with Friedreich’s ataxia are not able to make enough of this protein that is thought to play an important role in energy production in mitochondria. Larimar initially evaluated nomlabofusp at 25mg, dosing the first patient in March this year. After analysing frataxin pharmacodynamics at this dose, the biotech is planning to hike up the drug’s dose up to 50mg, for which it now has FDA clearance. Larimar’s CEO Carole Ben-Maimon said in a statement that interim data from the open-label extension study is expected in Q4 this year. The partial clinical hold was a blemish on the record for a drug that garnered multiple FDA awards, including rare paediatric disease designation, fast track designation and orphan drug designation. Nonetheless, Larimar touted positive results from the original Phase II study in February 2024. Data demonstrated that the nomlabofusp treatment over four weeks led to increases in frataxin levels in the skin and cells that line the cheeks and lips. Patients in the 50mg cohort had frataxin levels 33% higher than the control group’s average after two weeks. However, one participant in the 25mg cohort reported a severe adverse event for an allergic reaction and withdrew from the trial. The ongoing open-label extension study will add data on long-term safety and tolerability, pharmacokinetics, and peripheral tissue frataxin levels. Reata Pharmaceuticals won the race for the first approved therapy for Friedreich’s ataxia in the US, with its Skyclarys (omaveloxolone) receiving FDA approval in February last year. The oral capsules, which likely work by activating a pathway important in oxidative stress response, are approved to treat the disease in patients aged 16 years and above. Biogen assimilated the therapy into its portfolio following the acquisition of Reata in September 2023.

Biogen said Wednesday that sales of its Eisai-partnered Alzheimer's disease treatment Leqembi (lecanemab) gained traction in the first quarter, reaching $19 million and nearly tripling the sales it generated in the previous three-month period.According to Biogen, a "significant increase" in new patient starts was seen in March, with CEO Christopher Viehbacher noting that "momentum [is] building at a steady pace" for the product. "As the launch progresses and infrastructure develops, we continue to believe in the potential longer-term commercial opportunity in Alzheimer's disease," he added.However, the performance was still far below the $30 million Jefferies analysts had forecast, although other analysts had set a more modest $11-million benchmark. Still, investors were pleased, bumping up company shares by nearly 7% before the bell.Pace of patient startsBiogen said the number of patients on the anti-amyloid antibody had increased by nearly 2.5-fold from roughly 2000 patients at the end of 2023. Eisai had previously aimed to treat 10,000 patients by March, but earlier this year indicated that it would likely miss that target. For more, see Physician Views In-Depth: Neurologists provide five key insights into Leqembi's laggardly launch.While the latest numbers are encouraging, Jefferies analyst Michael Yee said the growth in patient starts needs to pick up steam from here.Leqembi's uptake has been hindered by requirements such as extra diagnostic testing, twice-monthly infusions and regular brain scans. Viehbacher said challenges include the "amount of effort it takes to actually be able to initiate even the first patient…I do think we're now on a positive track. I think we're seeing momentum," although he does not expect the launch to be linear.Bright spot with new launchesBiogen came into 2024 riding on the momentum of three other key product launches last year. Aside from Leqembi, the company also secured regulatory nods for Zurzuvae, a drug for post-partum depression (PPD) co-developed with Sage Therapeutics, and for Qalsody (tofersen), which is used to treat an ultra-rare genetic form of ALS. It also expanded in the rare disease space with a drug for Friedreich's ataxia, marketed as Skyclarys (omaveloxolone), when it acquired Reata Pharmaceuticals for $7.3 billion.Zurzuvae notched approximately $12 million in quarterly revenue, whereas analysts had expected just $5 million for the drug, which missed out on the larger opportunity in major depressive disorder when the FDA approved it only for the PPD indication. See Spotlight On: Zurzuvae approval a real downer for Sage, Biogen.Meanwhile, Skyclarys brought in $78 million, which edged passed consensus by about $6 million, while Qalsody generated $4.6 million in sales.Older drugs continue to struggleHowever, Biogen's mainstay MS drugs and spinal muscular atrophy treatment Spinraza continue to grapple with increased competition. Sales of its once-blockbuster MS drug Tecfidera came in at $254.3 million, although that was still about $18 million higher than consensus, while the MS franchise saw sales dip 4% to $1.1 billion during the quarter. Spinraza had sales of $341.3 million, falling below estimates.Overall sales for the first quarter were down 7% at $2.3 billion, which was in line with consensus, while net income reached $393.4 million, up from $387.9 million for the same period last year. Viehbacher indicated that the company's cost-cutting measures are also taking hold and helped offset competition for its older drugs. Last July, Biogen said it would eliminate 1000 jobs, or more than 11% of its workforce, in a bid to rein in operating expenses by an additional $700 million by 2025.Biogen on Wednesday also reaffirmed its outlook for adjusted earnings this year of $15 to $16 a share. The CEO said Biogen has no upcoming plans for major acquisitions, but is instead interested in licensing drugs and collaborating with other companies to expand its portfolio beyond neuroscience and into rare diseases and immunology.