Spinocerebellar Ataxias

iStock, Alona Horkova Biohaven will use the money to bankroll commercial preparations for the spinocerebellar ataxia drug candidate troriluzole, which is currently under FDA review with a decision expected in the third quarter. Biohaven has secured a potential $600 million non-dilutive infusion from private investment firm Oberland Capital, which will help fund its clinical and commercialization efforts, the company announced on Monday. Oberland will hand over $250 million once the deal closes, which Biohaven expects to occur “on or before April 30, 2025.” An additional $150 million tranche will be paid at the biopharma’s option and only after its spinocerebellar ataxia (SCA) drug troriluzole secures FDA approval, plus other specified conditions. The final $200 million balance will come once the parties reach a mutual agreement on certain “permitted strategic acquisitions and related costs and expenses.” In return for its investment, Oberland will be able to receive a regulatory milestone payment—which Biohaven can make quarterly through December 31, 2030—plus tiered single-digit royalties on net sales of troriluzole for up to 10 years. According to Biohaven, Monday’s funding arrangement will help it prepare for the commercial roll-out of troriluzole, in anticipation of an FDA approval. The drug, an investigational modulator of the glutamate neurotransmitter, is currently undergoing regulatory review for SCA, with a decision expected in the third quarter . Troriluzole’s prospects seem mixed. In September 2024, topline pivotal data showed that the drug candidate could slow patients’ rate of decline by 50% to 70% versus untreated counterparts. Troriluzole also delayed SCA progression by 1.5 years to 2.2 years. On the other hand, in August 2023, the FDA refused to even review troriluzole’s drug application, flagging that a Phase III trial used to support that application had failed to meet its primary endpoint. Oberland’s funding will also support Biohaven’s efforts across its developmental portfolio—a capital infusion that will help the company regain its footing in the clinic after a series of stumbles. In March, Biohaven reported that its potassium channel agonist BHV-7000 failed a Phase II/III trial in bipolar mania, unable to elicit significant improvements in manic symptoms in more than 250 patients with bipolar I disorder. In November last year, the myostatin and activin receptor blocker taldefgrobep alfa likewise flopped in the Phase III RESILIENT SMA study, failing to significantly boost motor function versus placebo in patients with spinal muscular atrophy. A few months earlier, in May 2024, Biohaven reported that its protein degrader BHV-1300 could reduce levels of autoantibody IgG in a Phase I healthy-volunteer trial. Investors were unimpressed by these data, however, sending the company crashing some 12% in its aftermath. At the time, analysts at William Blair said Biohaven’s findings fall “below the high bar” that many investors were hoping for.

Biohaven has secured funding to support its potential drug launch and pipeline growth. Credit: Olena Malik via Getty Images. Biohaven has secured a funding deal worth up to $600m from Oberland Capital Management, providing the biotech with non-dilutive capital as it prepares for a potential US launch of troriluzole for spinocerebellar ataxia (SCA). The financing comes in the form of a note purchase agreement, allowing Oberland to acquire up to $600m of Biohaven’s senior secured notes in multiple tranches. The first tranche of $250m is scheduled to be funded by 30 April, according to the company statement issued on 28 April. The second tranche – worth $150m – is contingent on US Food and Drug Administration (FDA) approval of troriluzole and subject to additional unspecified conditions. A third tranche of $200m may be made available for strategic acquisitions and related expenses. Oberland Capital will be eligible for a regulatory approval milestone payment, payable quarterly through the end of 2030. Shares in Biohaven closed 9.6% higher at $21.74 on 28 April, compared to $19.84 at market open. The deal comes as Biohaven awaits a regulatory decision from the FDA on troriluzole. The candidate is a prodrug of riluzole, which is already approved for amyotrophic lateral sclerosis (ALS). Troriluzole is being evaluated for SCA , a rare neurodegenerative disorder with limited treatment options, characterised by a loss of coordination and balance. The FDA granted the application priority review in February 2025, with a final decision expected in Q3 2025. The company is positioning the new funding to accelerate commercial planning and advance its broader pipeline, which includes CNS-targeting therapies. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Biohaven’s chief financial officer Matt Buten said: “This agreement with Oberland Capital provides us with the support and flexibility to continue advancing our pipeline as we make launch preparations in parallel, and we are excited to accelerate our planning efforts in earnest.” If it wins approval, troriluzole would represent Biohaven’s first greenlit product since it spun out from its migraine business following a $11.6bn acquisition from Pfizer back in 2022. However, Biohaven’s path to approval for troriluzole has not been straightforward. The drug failed to meet its primary endpoint in a Phase III trial (NCT03701399) conducted in 2022. Despite the setback, Biohaven shared subsequent analyses revealing benefits for patients with a certain subset of the disease and showed a slowed disease progression. The FDA initially refused to review the application but later accepted a revised submission following the release of this ad hoc data. Troriluzole’s prospects in Europe are more uncertain. Earlier in April, the European Medicines Agency (EMA) questioned whether the drug’s efficacy meaningfully differed from that of riluzole and challenged its classification as a new active substance. According to the EMA, Biohaven said it would generate additional data before submitting a new application. The new Biohaven entity was formed after the divestiture of its migraine assets to Pfizer in 2022. That deal included the commercial rights to Nurtec ODT (rimegepant), which generated $1.26bn in global sales in 2024, as per Pfizer’s financials. In January 2025, Pfizer agreed to pay $59.7m to settle allegations related to illegal payments made by the former Biohaven entity to healthcare providers before its acquisition. The US Department of Justice alleged that Biohaven paid kickbacks to physicians to boost prescriptions of Nurtec, resulting in false claims submitted to Medicare and other federal healthcare programmes between March 2020 and September 2022.

Under a new deal, Oberland Capital Management is in line to collect royalties on ultrarare brain disease drug troriluzole if it\'s approved. \n Oberland Capital Management is pumping some new life into Biohaven, striking a unique investment deal worth up to $600 million as the biotech awaits a key FDA approval decision that could propel it back into the commercial arena.The fresh funding comes in the form of a note purchase agreement, through which Oberland can buy up to $600 million of Biohaven’s senior secured notes (a type of corporate debt) split into specific tranches. The first portion of the investment, $250 million, is to be funded on or before April 30, while another $150 million is tied to the FDA’s potential approval of Biohaven’s spinocerebellar ataxia (SCA) prospect troriluzole. That latter tranche is an option exercisable by Biohaven and is \"subject to the satisfaction of certain additional conditions,\" the company said in an April 28 press release.Besides those two group of funds, up to $200 million could go toward “permitted” strategic acquisitions by Biohaven or related expenses, the company said. That final tranche would be contingent on a mutual agreement between the parties.In exchange, Oberland is in line to realize a piece of troriluzole\'s long-term commercial potential. The investor is slated to receive a milestone payment upon potential regulatory approval of the drug plus tiered single-digit royalty payments on global net sales for up to 10 years from the investment closing date.“This agreement with Oberland Capital provides us with the support and flexibility to continue advancing our pipeline as we make launch preparations in parallel, and we are excited to accelerate our planning efforts in earnest,” Biohaven Chief Financial Officer Matt Buten said in a company statement. Biohaven\'s share price jumped about 10% on Monday in the wake of the deal. It\'s been something of an uphill battle for Biohaven since it split off from its migraine franchise, which went to Pfizer as part of a $11.6 billion M&A deal back in 2022.Troriluzole, a prodrug of older amyotrophic lateral sclerosis (ALS) treatment riluzole, is up for a second chance with the FDA after an analysis last year showed that the asset slowed progression of the ultrarare disease SCA. The regulator has accepted the company’s drug application and granted a priority review, so the agency\'s approval decision is expected in the third quarter of this year.Even getting to the FDA’s desk represents a change of fate for Biohaven. The agency had initially refused to evaluate the drug in 2023, citing a failed phase 3 trial. Now, the company is already gearing up for a commercial launch within the year, it said in a March update.  Overseas, troriluzole’s future is more unclear as the biotech withdrew its bid for approval with the European Medicines Agency (EMA), the regulator’s Committee for Medicinal Products for Human Use recently revealed in its April meeting highlights.European regulators listed two main concerns with troriluzole in SCA, namely centered on the drug’s efficacy data and its application status as a new, active substance considering its prodrug origin. The company’s ultimate withdrawal letter noted that it plans to generate additional data before submitting a new application, according to the EMA.Prodrugs are inactive compounds that become active once metabolized by the body. In many cases, they\'re derivatives of older or existing medicines. Still, the EMA application pull “comes as a surprise” considering the FDA’s application acceptance, William Blair analysts said in an April 25 note to clients. Biohaven\'s share price fell more than 12% on Friday in the wake of the news. Nonetheless, the William Blair analysts view the FDA as “likely to show more regulatory flexibility” compared to its European counterpart.Beyond troriluzole, Biohaven’s pipeline is stuffed with prospects across its five key platforms that cover neuroscience, oncology, immunology and inflammation. The company boasts one of the “most diverse” pipelines among its peers, William Blair analysts noted. However, recent setbacks have left a mark on the biotech\'s R&D efforts. For example, the company is leaning on impending major depressive disorder and epilepsy results for its BHV-7000 after hitting a wall in bipolar disorder with a failed 2/3 study earlier this year. Elsewhere, Biohaven\'s spinal muscular atrophy prospect taldefgrobep fell short in a phase 3 trial last year, but the company still planned to explore a possible regulatory path forward.