Jazz Pharmaceuticals Plc

A new kind of sleeplessness medicine developed by Takeda met its goals in late-stage testing, positioning the company to capitalize on what Wall Street analysts believe could be a multibillion-dollar market opportunity.The drug, formerly known as TAK-861 but now called oveporexton, was evaluated in two Phase 3 studies in a main type of narcolepsy. According to Takeda, oveporexton hit every main and secondary trial endpoint, helping improve patient-reported scores on measures of wakefulness, excessive daytime sleepiness, muscle weakness, and other symptoms after 12 weeks of treatment.The company didnt provide specifics, but said in a Monday statement that drug recipients had statistically significant and clinically meaningful improvements on those assessments, and achieved near normal ranges across studied symptoms.Oveporextons safety profile was consistent with whats been observed in previous clinical tests, Takeda said. The most common adverse events reported were insomnia as well as the sudden urge to urinate. No serious treatment-related side effects were reported, and more than 95% of study participants who completed the trials enrolled in a long-term extension study.The findings build on the transformative results we saw in our Phase 2b study, with most participants reaching normative ranges and reporting clinically meaningful improvement across a broad range of symptoms at the end of the 12-week treatment period, Andy Plump, Takedas R&D chief, said in the companys statement.Takeda will file an approval application in the U.S. and elsewhere in its 2025 fiscal year and plans to present detailed results at an upcoming medical meeting.The results position Takeda as the first company to seek approval of a narcolepsy drug that works by stimulating proteins called orexin receptors. These proteins have an array of effects on the brain, among them a role in maintaining wakefulness. They have long been linked to narcolepsy Type 1, a type of chronic sleeplessness that involves muscle weakness. Theyve attracted the interest of several drug companies, among them Jazz Pharmaceuticals, Alkermes, Johnson & Johnson and Centessa Pharmaceuticals, all of which have brought orexin medicines into clinical testing for either sleep disorders or other conditions.There have been some setbacks. Takeda shelved an earlier orexin-targeting medicine due to liver toxicity, while Jazz stopped an early-stage trial after reporting eye and heart-related side effects.Takedas success in late-stage testing cement[s] [its] lead over its competitors, wrote Jefferies analyst Stephen Barker in a Monday client note. Its drug could achieve $3 billion in peak yearly sales in narcolepsy Type 1 alone, he added.Barker also predicted that Takedas broader portfolio of orexin drugs, which includes medicines in testing for Type 2 narcolepsy and idiopathic hyperinsomnia, may achieve more than double that total. The company, which restructured in 2024, is counting on these and other drugs in late-stage development to help improve growth in the years ahead.Takedas closest competitor in narcolepsy Type 1, Alkermes ALKS 2680, is currently in Phase 2 testing. '

The European Commission (EC) has granted conditional approval to Jazz Pharmaceuticals’ Ziihera (zanidatamab) to treat a subset of advanced biliary tract cancer (BTC) patients. The human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody has been authorised for use as a monotherapy in adults with unresectable locally advanced or metastatic HER2-positive (IHC 3+) BTC who have received at least one prior line of systemic therapy. The EC’s decision was supported by results from the phase 2b HERIZON-BTC-01 trial, which included a cohort of 80 BTC patients with centrally confirmed HER2-positive tumours (cohort one). The study met its primary endpoint of confirmed objective response rate (cORR) in cohort one, with Ziihera demonstrating a cORR of 41.3%, including two complete responses, at a median follow-up of 21.9 months. The median duration of response (DoR) was 14.9 months, and the median overall survival (OS) was 15.5 months. Data from a pre-specified subgroup analysis in 62 patients with IHC 3+ tumours also showed that Ziihera demonstrated a cORR of 51.6%, with a median DoR of 14.9 months and median OS of 18.1 months. BTCs, including gallbladder cancer and cholangiocarcinoma, are a rare and aggressive group of cancers that are often diagnosed at an advanced stage, when curative surgery is no longer an option. Approximately 26% of BTC patients globally have HER2-positive disease, which is associated with poorer outcomes compared to HER2-negative disease. Robert Iannone, executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals, said the conditional approval “represents significant progress” for patients with advanced HER2-positive BTC. “Ziihera is the first HER2-targeted therapy authorised in the EU specifically for this population, and the EC’s decision reflects both the strength of the HERIZON-BTC-01 data and the urgency for innovation in rare gastrointestinal cancers,” Iannone said. In line with EC’s conditional approvals pathway, continued authorisation for Ziihera in this indication is contingent on the verification of clinical benefit in the ongoing phase 3 HERIZON-BTC-302 trial, which is evaluating the drug in combination with standard-of-care therapy in the first-line setting for patients with HER2-positive BTC.

SINGAPORE, July 7, 2025 /PRNewswire/ -- Nuevocor, a Singapore-headquartered biotechnology company developing cures for cardiomyopathies driven by aberrant mechanobiology, today announced the appointment of Dr. Thierry Abribat to its Board of Directors. Dr. Thierry Abribat is a seasoned biotech executive and serial entrepreneur with a proven track record of founding, leading, and successfully exiting three biotechnology companies, each focused on developing innovative therapies for patients with high unmet medical needs. He most recently orchestrated the $1 billion acquisition of Amolyt Pharma by AstraZeneca, underscoring his ability to scale and strategically position biotech companies for high-value exits. His other ventures include Alizé Pharma, acquired by Millendo Therapeutics in 2017 and Alizé Pharma 2, acquired by Jazz Pharmaceuticals in 2016. Dr. Abribat also held senior leadership roles in drug development, business development, and general management. He served as Chief Scientific Officer at Theratechnologies and as Chief Operating Officer at OPi SA, a specialty pharmaceutical company focused on rare diseases, which was acquired by EUSA Pharma in 2007. Dr. Abribat holds a Doctor of Veterinary Medicine degree and a PhD in neuroendocrinology from the National Polytechnic Institute of Toulouse. "I am excited to join Nuevocor at such a pivotal time in its journey toward becoming a clinical-stage company. Nuevocor's innovative mechanobiology-based approach has the potential to transform treatment for patients suffering from devastating genetic cardiomyopathies. I look forward to supporting the team as part of this Europe-Singapore partnership, as they advance this groundbreaking science into the clinic," said Dr. Thierry Abribat. "We are honored to welcome Dr. Thierry Abribat to Nuevocor's Board of Directors. Thierry is a highly accomplished biotech entrepreneur with a remarkable track record of building and scaling companies, and translating science into successful therapeutics. As we transition to a clinical-stage company and expand our presence in Europe, his strategic insights, deep operational expertise, and solution-oriented leadership will be invaluable. It's a privilege for us to work alongside him in advancing a potentially transformative therapy for patients suffering from debilitating cardiomyopathies," said Dr. Andreas Wallnöfer, Chairman of the Board. About NVC-001 NVC-001 is a first-in-class, AAV-based gene therapy for the treatment of LMNA-related dilated cardiomyopathy (LMNA DCM), one of the most aggressive forms of DCM with a rapid progression toward end-stage heart failure and malignant ventricular arrhythmias associated with increased risk of sudden cardiac death. NVC-001 is designed to reduce forces to the nucleus to restore nuclear envelope integrity, a hallmark of LMNA DCM, and treat disease. In preclinical studies, treatment with NVC-001 demonstrated significant benefits, including survival and cardiac function. Nuevocor plans to initiate a Phase 1/2 clinical trial in early 2026, with sites in the U.S. and Europe. About Nuevocor Nuevocor is biotechnology company developing novel therapies for genetic cardiomyopathies driven by aberrant mechanobiology, headquartered in Singapore with an office in the U.S. and expansion to Europe. Our unique approach, enabled by our proprietary PrOSIA mechanobiology platform, surpasses the limitations of traditional gene replacement therapy – which treats individual gene mutations – to treating defects in shared disease pathways across multiple cardiomyopathies by addressing the functional root cause of disease. Nuevocor is first-in-disease by addressing genetic cardiomyopathies that are not amendable to gene replacement therapy and have no effective treatment options. () SOURCE Nuevocor WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED