Jazz Pharmaceuticals Plc

Today, a brief rundown of news involving Biohaven and Catalent, as well as updates from Stealth Biotherapeutics, Jazz Pharmaceuticals and Celldex Therapeutics that you may have missed.The Food and Drug Administration no longer plans to hold a meeting with outside experts to help evaluate an approval application for a Biohaven medicine called troriluzole. According to a regulatory filing, the agency informed Biohaven of this change on Thursday, but still expects to issue an approval verdict sometime late in the year. Canceling the advisory committee meeting “likely indicates that the FDA feels it has sufficiently resolved any difficulties in interpreting data pertaining to the safety or efficacy of the drug though whether their conclusion is positive or negative is difficult to determine,“ wrote RBC Capital Markets analyst Leonid Timashev in a note to clients. Timashev also highlighted how the history of these cancellations is “modestly encouraging“ for Biohaven, since, in roughly two-thirds of cases, the FDA subsequently green lit the drugs under review. Jacob BellContract manufacturer Catalenthas laid off 350 employees working at its Baltimore gene therapy facility due to an unexpected shift in demand from a large customer, a company spokesperson confirmed to BioPharma Dive. Our gene therapy business continues to see strong growth and we look forward to working on behalf of customers to deliver novel therapies for patients with genetic diseases, the spokesperson added. Catalent is now owned by Novo Holdings, the parent company of Novo Nordisk. Ned PagliaruloThe FDAhas accepted a revised approval submission for a rare disease drug developed by Stealth BioTherapeutics. According to Stealth, the agency considers its new application a class 2 response to a prior rejection, meaning its treatment, a Barth syndrome therapy called elamipretide, would typically undergo a six-month review ending in February. The FDA, though, intends to make a decision by Sept. 26, Stealth said Thursday. The review of elamipretide has been flagged by conservative outlets as evidence of biotech innovation slowing down under the leadership of commissioner Martin Makary. Ben FidlerJazz Pharmaceuticals has bought rights to an experimental epilepsy medicine in a heavily back-loaded deal that could ultimately surpass $1 billion in value. Jazz gave Denmark-based Saniona almost $43 million upfront for an exclusive license to SAN2355, a small molecule drug designed to activate certain so-called potassium ion channels. While not yet in human testing, SAN2355 looks to be a highly promising asset with the potential to be the best in its drug class, said Robert Iannone, Jazzs head of research and development. TD Cowen analyst Joseph Thome wrote in a note to clients how the deal brings in a familiar, closely watched target that should fit in nicely with Jazz's current neuroscience portfolio. Thome added that Sanionas molecule is somewhat de-risked due to the clinical advancement of rival therapy from Xenon Pharmaceuticals. Jacob BellAn experimental drug from Celldex Therapeutics didnt alleviate signs and symptoms of the inflammatory condition eosinophilic esophagitis in a Phase 2 trial, the company said Wednesday. The drug, barzolvolimab, is currently in late-stage testing in people with skin hives called urticaria. Celldex had been hoping the drugs ability to deplete levels of mast cells immune cells implicated in multiple immune conditions might be beneficial in eosinophilic esophagitis too. While that didnt happen, barzolvolimab still had clean safety in the study, which bodes well for its ongoing Phase 3 trials in urticarias, wrote Leerink Partners analyst Thomas Smith. Ben Fidler '

iStock, Softulka The drug, for hereditary angioedema, is Ionis’ second wholly owned asset. The FDA on Thursday approved Ionis Pharmaceuticals’ donidalorsen for the prevention of disease attacks in patients with hereditary angioedema. The product will carry the brand name Dawnzera. Dawnzera is the first and so far only RNA-targeting therapy approved as a prophylactic treatment for hereditary angioedema (HAE), Ionis said in its Thursday news release . The product, which can be used in patients 12 years and above, will launch in the U.S. in the coming days. “We see Dawnzera’s pro superior to current prophylactic options, with less frequent dosing and at-home auto-injector dosing being further differentiators,” William Blair analysts told investors in a Thursday note, adding that Dawnzera marks Ionis’ second wholly owned asset. “We view Dawnzera as a highly competitive HAE prophylactic.” Data from the OASIS-HAE study backed Thursday’s approval. Results published in the New England Journal of Medicine in May 2024 showed that over 24 weeks of follow-up, the RNA-targeted therapy lowered the monthly HAE attack rate by 81% versus placebo. Dawnzera also demonstrated an approximately 90% reduction in moderate-to-severe HAE attacks over 24 weeks when measured from the second dose, and significantly improved patients’ quality of life. Ionis filed data from the Phase III OASISplus open-label extension study to bolster its application. According to Thursday’s release, Dawnzera treatment resulted in a 94% drop in the total mean attack rate from baseline at one year of follow-up. Meanwhile, a readout last month demonstrated that patients who switched to the RNA-targeting therapy from other prophylactics did so without experiencing spikes in attacks. In a survey, 84% of patients indicated that they preferred Dawnzera over their previous prophylactic. Administered subcutaneously every four weeks, Dawnzera is a conjugated antisense oligonucleotide that targets the prekallikrein mRNA, causing its destruction. This reduces overall levels of PKK, a protein that plays a role in swelling and pain attacks in HAE. Dawnzera does not come with a boxed warning, though its label has precautions for hypersensitivity reactions such as anaphylaxis. Dawnzera’s approval on Thursday continues a series of history-making approvals for the rare disease space this month. Leading the pack is Jazz Pharmaceuticals, which on Aug. 6 won the FDA’s go-ahead for dordaviprone, now marketed as Modeyso, for the treatment of diffuse midline glioma with an H3 K27M mutation in patients aged one year and up who have progressed after previous interventions. Modeyso is the first drug approved for this ultrarare brain tumor. A week later, Insmed’s brensocatib won approval for non-cystic fibrosis bronchiectasis for patients 12 and over. The drug, branded as Brinsupri, is the first therapy approved for this indication and is also the first DPP1 inhibitor to win the FDA’s blessing. Finally, Precigen on Aug. 14 bagged the industry’s first immunotherapy approval for recurrent respiratory papillomatosis with zopapogene imadenovec-drba, which will carry the brand name Papzimeos.

Saniona to receive US $42.5 million upfront; potential for development, regulatory and sales milestone payments in addition to royalties on future net sales Jazz obtains exclusive worldwide rights to develop and commercialize preclinical asset SAN2355 in epilepsy and other potential indications For U.S. media and investors only DUBLIN, Ireland and COPENHAGEN, Denmark I August 20, 2025 I Jazz Pharmaceuticals plc (Nasdaq: JAZZ ) and Saniona (Nasdaq OMX: SANION) today announce that the companies have entered into a global license agreement for Jazz to obtain exclusive worldwide rights to develop SAN2355, a highly differentiated, subtype-selective Kv7.2/Kv7.3 activator in preclinical development for epilepsy and other potential indications, designed to overcome the limitations of non-selective Kv7-targeting compounds. Jazz will lead and fund further development, regulatory submissions, and global commercialization activities. “Our collaboration with Jazz leverages their leading position in epilepsy and their strong track record of rapidly advancing clinical development programs and effectively commercializing novel therapies in neuroscience, positioning SAN2355 as a promising option for epilepsy and neurological conditions, while enabling us to advance several of our other valuable pipeline programs to key inflection points,” said Thomas Feldthus, CEO of Saniona. “Jazz’s commitment to develop life-changing medicines for people with serious diseases aligns with our mission to improve outcomes for those affected by neurological and psychiatric disorders, and both companies aim to expand treatment possibilities for patients in need.” “SAN2355 is a unique and highly promising preclinical asset that has the potential to become a best-in-class Kv7 activator for the treatment of epilepsy. We are pleased to enter this exclusive agreement with Saniona to lead the SAN2355 clinical development program,” said Robert Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. “This transaction further expands our early-stage neuroscience pipeline building on our existing expertise in the treatment of epilepsy and our R&D team remains committed to developing novel approaches to improving treatment options for people living with epilepsy and other neurologic conditions.” Transaction Terms Under the terms of the exclusive licensing agreement, Saniona will receive an upfront payment of US $42.5 million. Saniona is eligible to receive: up to US $192.5 million in development and regulatory milestones, including a US $7.5 million milestone payment upon initiation of the first Phase 1 clinical study; up to US $800 million in commercial milestone payments based on the achievement of significant and pre-specified annual net sales thresholds; and tiered royalties ranging from the mid-single digits to low-double digits on net sales of commercial products resulting from the development of SAN2355. About SAN2355 SAN2355 is a preclinical, selective small molecule activator of Kv7.2/Kv7.3 potassium channels, a mechanism validated for seizure suppression. Prior Kv7-targeting agents have demonstrated clinical efficacy, but dosing appears to be limited by adverse events associated with off-target activation. SAN2355 is uniquely selective for Kv7.2/Kv7.3, the Kv7-subtypes responsible for seizure suppression, and avoids activation of other Kv7-subtypes. This selectivity enables SAN2355 to deliver dosing to optimal efficacy and supports its potential as a best-in-class treatment for epilepsy. About Jazz Pharmaceuticals Jazz Pharmaceuticals plc (Nasdaq: JAZZ ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information. About Saniona Saniona is a clinical-stage biopharmaceutical company focused on discovering, developing, and delivering innovative treatments for neurological and psychiatric disorders. The company’s internal pipeline includes SAN2219 for epilepsy and SAN2465 for major depressive disorder. Saniona has established strategic collaborations with leading pharmaceutical companies, including Jazz Pharmaceuticals, which holds global rights to SAN2355 for epilepsy, Acadia Pharmaceuticals, which holds worldwide rights to ACP-711 for essential tremor, and with Medix, which holds rights to tesofensine for obesity in Mexico and Argentina, where a market authorization application is currently under review. In addition, Saniona has two clinical-stage programs available for partnering: Tesomet™, ready to advance to Phase 2b trials in rare eating disorders, and SAN903, ready to enter Phase 1 trials in inflammatory bowel disease. Saniona’s ion channel discovery platform is further validated through research collaborations with Boehringer Ingelheim, AstronauTx, and Cephagenix. Headquartered in Copenhagen, Saniona is listed on the Nasdaq Stockholm Main Market. For more information, visit www.saniona.com . SOURCE: Jazz Pharmaceuticals