Mizuho Medy Co., Ltd.

BioXcel is taking the next step in an effort to update the label of its psychiatric drug Igalmi. A Phase 3 trial studying Igalmi enrolled schizophrenia or bipolar disorder patients who suffer episodes of agitation. The company said early Wednesday morning that the drug succeeded in showing it can be taken on an as-needed basis at home. The primary endpoint was safety and tolerability, and patients were allowed to remain on their other antipsychotic drugs. (Igalmi is a film placed under the tongue akin to a Listerine strip.) Igalmi’s previous approval — in 2022 for the same indication — only allows it to be taken under the supervision of a doctor. After the 2022 approval, the company sought to update the label to remove the need for medical supervision, BioXcel’s CEO Vimal Mehta told Endpoints News . The FDA asked for a study to see if it was safe to do so, citing concerns about patients experiencing orthostatic hypotension or dizziness upon standing up. Sometimes patients aren’t able to make it to a doctor’s office before their agitation escalates, Mehta added. “These patients have been through the agitation before; they have an aura like migraine, they know that it is coming,” he said. “They also know that they need to arrest the agitation or do something about it.” BioXcel also measured the number of agitation episodes resolved on treatment compared to placebo, although it did not power this comparison for statistical significance. In the study, 66% of patients taking Igalmi saw their agitation fully resolve compared to 25% of those who took placebo. The study lasted 12 weeks. With the results in hand, BioXcel plans to file for the label expansion in the first quarter of next year. BioXcel collected data from 2,628 episodes in 215 patients, 55% of whom had schizophrenia and 45% of whom had bipolar disorder. BioXcel is facing a cash crunch, according to a note from Mizuho analyst Graig Suvannavejh earlier this month. The company’s full-year revenues are expected to be around $1 million, while two debt tranches of at least $100 million each are due in 2026 and 2027. Regardless of the study’s outcome, Suvannavejh expects BioXcel will need to raise cash — and soon. The company would need either an equity raise “in significant excess” of its market cap (roughly $70 million) or a partnership with another company. A successful Phase 3, Suvannavejh wrote, would be key to convincing investors that BioXcel is well-positioned to do so. Mehta declined to comment on whether BioXcel would seek a new raise based on Wednesday’s data, but said the results provide “a lot of optionality” for the company.

Insmed appears to have a future blockbuster on its hands, with Mizuho analysts writing Monday that they expect Brinsupri to reach peak sales of $6.6 billion in NCFBE. With a landmark U.S. approval  Tuesday, Insmed is making waves with a new class of drug that’s also the first to be green lit in the chronic lung disease bronchiectasis.The FDA has signed off on Insmed’s brensocatib to treat non-cystic fibrosis bronchiectasis (NCFBE) in adults and kids ages 12 and older. The daily oral drug, approved in both 10 and 25 mg tablets, is already available by prescription through a specialty pharmacy network, Insmed noted in a press release.Prior to Brinsupri's approval, Insmed boasted a single commercial medicine in Arikayce, which was approved in 2018 to treat a narrow group of patients with lung disease caused by the mycobacterium avium complex (MAC) family of bacteria. Now, the New Jersey drugmaker appears to have a future blockbuster on its hands, with Mizuho analysts writing Monday that they expect Brinsupri to reach peak sales of $6.6 billion in NCFBE.Insmed itself believes the drug can generate $5 billion at peak in the indication, according to an investor presentation (PDF) published Tuesday.Aside from bringing the first dedicated treatment for NCFBE to the fore, Brinsupri's FDA approval also marks the introduction of the dipeptidyl peptidase 1 (DPP1) inhibitor class. As a DPP1 inhibitor, Insmed's drug is designed to inhibit an enzyme that activates neutrophil serine proteases (NSPs) in white blood cells. Though NSPs normally help mediate inflammation and destroy pathogens, in certain diseases, they’re activated to excess, leading to inflammation and the destruction of organs.Key opinion leaders (KOLs) who spoke to Mizuho have been “incredibly bullish” about the prospects of Brinsupri, the analysts wrote in their Monday note to clients. The KOLs pointed to the drug’s impressive efficacy and safety data, continuing unmet need in the sizable bronchiectasis market and the general enthusiasm around the drug that’s been expressed by both patients and doctors, according to the analysts.It's estimated that around 500,000 people in the U.S. are diagnosed with NCFB, Insmed noted in its release. The chronic condition causes the walls of the body’s airways to widen and thicken from inflammation and infection. The FDA based its approval of Brinsupri on data from Insmed’s phase 3 ASPEN trial, as well as the company's midstage WILLOW study. The results from the phase 3 trial wowed investors and industry watchers when Insmed unveiled them last May, with the company’s shares now trading up roughly 4 times compared to where they were before the 2024 data drop.In the trial of more than 1,700 NCFBE patients, participants who received a 10 mg dose of Brinsupri charted a 21% reduction in the annual rate of pulmonary exacerbations compared to those on placebo, while patients who received a 25 mg dose of Insmed’s drug experienced a 19% reduction.The drug also won out on multiple key endpoints by helping extend the time before patients’ first pulmonary exacerbation and boosting the odds of patients remaining exacerbation free over an entire year.It wasn’t a perfect showing, however, as the study failed to meet the secondary goal of change in baseline of post-bronchodilator forced expiratory volume in one second at Week 52 for the low dose. Brinsupri also failed to reduce the annualized rate of severe pulmonary exacerbations on either dose.Insmed will price Brinsupri at $88,000 per year before discounts, Mizuho analysts wrote in a new note following a company conference call Tuesday. That sticker price is in line with the range the company had most recently communicated. It will likely be about a month before Insmed starts logging sales from Brinsupri, the analysts added. In turn, sales of the drug in 2025’s third quarter may be “relatively small,” the Mizuho team said in its previous note.With the FDA nod, Insmed’s sales force is expected to “begin engaging with U.S. pulmonologists about Brinsupri immediately,” the company said in its approval slide deck. Insmed isn’t about to be caught flatfooted, as the company has left “no stone unturned to have the strongest launch possible for one of the most anticipated drug launches of the year,” analysts at Leerink Partners wrote in a note to clients last week.The company’s force has been on the ground since last October and has subsequently already engaged with some 27,000 healthcare professionals in the U.S., which the Leerink team noted is “essentially all the pulmonologists in the country.”To further ensure a smooth launch, Insmed has built out a patient support team dubbed inLighten, the analysts pointed out. Wall Street, for its part, has forecasted Brinsupri sales between $40 million and $60 million during the drug’s first year on the market.  Elsewhere, Insmed has submitted additional applications for Brinsupri in the EU and the U.K. The company said last week that it is preparing to file for approval in Japan in the back half of the year as well. Insmed anticipates launching in those territories in 2026 should brensocatib win the requisite approvals in each region. Editor's note: This story was updated with additional details on Insmed's launch preparations. 

Astellas reported a 22% sequential increase in quarterly sales of geographic atrophy drug Izervay and is projecting booming sales increases for the rest of the year and beyond. After reporting a 15% sequential decline in Izervay sales for the opening quarter of this year, Astellas has reversed the momentum for its geographic atrophy (GA) treatment and is projecting the upward trend to continue this year.In an investor event on Wednesday—three weeks ahead of its quarterly report—Astellas revealed a 22% sequential increase in Izervay sales for the period ending on June 30. The injected C5 inhibitor generated revenue of 15.9 billion Japanese yen ($110 million) in the most recent quarter, up from 13.8 billion Japanese yen ($91 million) in the first quarter.Astellas sees Izervay’s growth continuing to track upward—in the “high 20s or above"—for the next three quarters and is reiterating its fiscal year sales guidance of 105 billion Japanese yen ($750 million).In the longer term, Astellas is now tabbing peak sales for Izervay to reach between 200 billion and 400 billion Japanese yen ($1.3 billion to $2.6 billion) in the U.S. alone. Those figures match the company’s previous projection for peak global sales.Some analysts, however, aren’t buying into Astellas’ rosy expectations just yet. Despite the increase in quarterly sales, the figures came up far short of consensus, which were based largely on the company’s annual sales projection, according to analysts from ISI Evercore.“We wouldn’t be surprised to see Astellas miss their aggressive FY25 guidance,” they wrote in a note to investors.Meanwhile, analysts at Mizuho Securities have doubts about the growth of the GA market as portrayed by the company. Astellas said on Wednesday that, by 2029, it expects to treat at least 35% of the GA patient population in the U.S., crediting “broader upstream awareness and more patient referrals to retina specialists.”“Our biggest concern lies with an overall GA market where growth is potentially stalling,” Mizuho analyst Graig Suvannavejh, Ph.D., wrote in a note to investors, citing recent discussions with key opinion leaders (KOLs). “Despite Astellas' vision of future growth for Izervay in 2025 and beyond, we question what it will take for the CI class to meaningfully grow over time.” It's uncertain what this all means for Astellas’ rival in the GA market, Apellis. Two months ago, the Massachusetts company revealed sales of $130 million in the first quarter for Syfovre, which were down 22% sequentially.The company’s CEO, Cedric Francois, M.D., Ph.D., blamed inventory fluctuations and funding shortfalls in copay assistance programs for the sales decline.Eyes will be on Apellis when it reports its second quarter sales to see if Syfovre gets a similar sequential bump, which would be “supportive of a growing market,” Mizuho wrote.“On the other hand, given KOL feedback we've been hearing of a relatively slow-growing/flat market, there's potential of Izervay gaining at Syfovre's expense,” the analysts added. Astellas has had its own set of problems hindering the launch of Izervay. Early this year, the company took an impairment charge of 115 billion Japanese yen ($760 million) because of regulatory setbacks, including an FDA rejection for Izervay to broaden its label.In February, Astellas received a much-needed boost when the FDA lifted a 12-month restriction on the use of Izervay. With that and the sales upturn, the company said on Wednesday that it expects to hold 55% to 60% of the market share in the U.S. by 2029.