Servier Pharmaceuticals LLC

Oncomine Dx Target Test to Identify Patients Eligible for Servier’s VORANIGO® (vorasidenib) tablet, the only FDA-approved treatment for Grade 2 IDH-mutant glioma CARLSBAD, Calif.--(BUSINESS WIRE)--Thermo Fisher Scientific, the world leader in serving science, has received approval from the U.S. Food and Drug Administration (FDA) for its Ion Torrent™ Oncomine™ Dx Target Test as a companion diagnostic (CDx) to identify patients eligible for treatment with Servier Pharmaceuticals, LLC’s VORANIGO® (vorasidenib) tablets. VORANIGO is an isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor indicated for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery including biopsy, sub-total resection or gross total resection. As the first targeted therapy for Grade 2 IDH-mutant glioma, VORANIGO provides a new care path for patients with extremely limited treatment options. Gliomas are the most common malignant primary brain tumor in adults, representing approximately 81% of primary malignant brain tumors. Of those, approximately 20% harbor an IDH mutation and testing for these mutations is essential for accurate treatment decisions. Further, the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology recommend IDH mutation testing in all patients with a glioma, noting its impact on diagnosis and prognosis of gliomas. IDH mutation status has been a key factor in the NCCN treatment guidelines for years. With the FDA approval of VORANIGO, determining the IDH mutation status of patients with glioma will be even more critical. “VORANIGO is the first and only targeted therapy for patients living with grade 2 IDH mutant glioma, a relentless and incurable type of brain cancer that hasn’t seen treatment advances in nearly 25 years,” said David K. Lee, CEO of Servier Pharmaceuticals. “As more targeted therapies become available to patients, identifying key driver mutations is essential to help the right patients find the right treatment, at the right time.” In addition to the approval for IDH-mutant diffuse glioma, the Oncomine Dx Target Test, has also previously received approvals for indications in non-small cell lung cancer (NSCLC), cholangiocarcinoma (CCA), medullary thyroid cancer (MTC) and thyroid cancer (TC). As a distributable companion diagnostic, the test simultaneously delivers biomarker results for multiple targeted therapies from one sample, helping quickly match patients with the right targeted therapies. “As the healthcare system works to realize the impact of precision medicine, patients must have access to the proper testing that helps unlock targeted treatment options based on their unique genomic pro This access is the driving motivation behind the extensive work we do with pharma partners to help connect the right patients to new therapies as soon as they are approved,” said Kathy Davy, president, clinical next-generation sequencing at Thermo Fisher Scientific. “The work we do every day reflects our Mission, and combining our CDx technology with Servier’s breakthrough therapy will help dramatically impact care for patients with aggressive brain tumors.” Today’s approval expands clinical indications for the Oncomine Dx Target Test, which is currently approved and reimbursed by government and commercial insurers in 19 countries, including the U.S., Japan, South Korea and countries across Europe and the Middle East, covering more than 550 million lives globally. Following this approval, the two organizations will continue to collaborate on an additional companion diagnostics with the Ion Torrent™ Oncomine Dx Express Test.* Available on the Ion Torrent Genexus™ Dx System*, the Oncomine Dx Express Test can return results in as little as a single day, dramatically accelerating the pace with which patients can be matched with optimal treatments based on their genomic results. *The Oncomine Dx Express Test and the Genexus Dx System are currently available only in countries that accept the CE mark. About Thermo Fisher Scientific Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue over $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD. For more information, please visit . Contacts Media Contact Information: Amanda Strickland Phone: (910) 622-8752 E-mail: amanda.strickland@thermofisher.com Jessika Parry Phone: 419-266-4016 Email: jparry@greenoughagency.com

The Ion Torrent Oncomine Dx Target Test is the first FDA-approved next generation sequencing-based diagnostic. Credits: JHVEPhoto / Shutterstock. Thermo Fisher Scientific has announced that its in vitro diagnostic test, Oncomine, has won US Food and Drug Administration (FDA) approval for use as a companion diagnostic in grade 2 IDH-mutant glioma. As per the 21 October press announcement, Oncomine is now approved to be used to identify patients who are eligible for treatment with Servier Pharmaceuticals’ Voranigo (vorasidenib) tablets. Voranigo, which gained FDA approval in August 2024, is the first and only targeted therapy available for patients with grade 2 IDH-mutant glioma. The French drugmaker gained control over the asset in 2020 after purchasing Agios’ oncology pipeline for $1.8bn in 2020. Agios has since sold its royalty stake in Voranigo to Royalty Pharma for $905m. Under the terms of the agreement, Royalty Pharma will gain a 12% royalty on annual US sales up to $1bn and a 12% royalty on net US sales over $1bn. As a once-daily oral inhibitor of IDH1 and IDH2 enzymes, Voranigo is indicated for use in grade 2 IDH-mutant glioma patients following surgery, including biopsy or gross total resection. The Ion Torrent Oncomine Dx Target Test is the first FDA-approved sequencing-based diagnostic. Oncomine received its first approval as a companion diagnostic in 2017 and subsequently gained approval in August 2022 as a companion diagnostic for identifying non-small cell lung cancer (NSCLC) patients eligible for treatment with Daiichi Sankyo ’s Enhertu (fam-trastuzumab deruxtecan-nxki). See Also: FDA approves Astellas’ VYLOY for gastric cancer treatment Intercept’s liver disease drug Ocaliva faces FDA approval delay Oncomine is also approved for use in cholangiocarcinoma (CCA), medullary thyroid cancer (MTC) and thyroid cancer (TC). Daiichi and AstraZeneca ’s Enhertu has seen several wins this year. The drug gained conditional approval in China earlier this month and a breakthrough therapy designation for a specific type of breast cancer in August 2024. Additionally, in April, the treatment was granted accelerated approval for the treatment of unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-positive [immunohistochemistry (IHC) 3+] solid tumours. According to GlobalData’s consensus forecasts, Voranigo is expected to generate sales of $37m in 2030. Enhertu is projected to generate total sales of $13bn in 2030. GlobalData is the parent company of Pharmaceutical Technology .

Whether a brain cancer drug enters the body through a needle in the arm or swallowed by mouth, the blood-brain barrier stands as a formidable last obstacle on the therapy’s journey to its destination. NeOnc Technologies navigates around this protective barrier using drugs inhaled through the nose. With a lead program in mid-stage clinical testing, NeOnc is now preparing to take itself public. Though some recent signs point to improving IPO conditions, the IPO window has been mostly closed to biotech companies the past two years, which NeOnc knows firsthand. After laying groundwork for a traditional IPO earlier this year, the Westlake Village, California-based firm withdrew its registration statement amid lukewarm investor interest. NeOnc is changing course. Just as its drug takes an alternate path to the brain, the company is pursuing an alternate way of going public: a direct listing. Executives say they expect to file new securities paperwork this month. NeOnc is seeking investor buy-in for its novel approach to treating aggressive brain cancers, such as glioblastoma. The standard of care includes surgery, chemotherapy, and radiation. If the cancer advances, the standard drug treatment is temozolomide, brand name Temodar, a Merck chemotherapy in oral and injectable formulations that was initially approved in 1999. According to NeOnc CEO Thomas Chen, a neurosurgeon and professor of neurological surgery at the University of Southern California’s Keck School of Medicine, the challenge for the Merck drug is getting enough of it through the blood-brain barrier to reach tumors. presented by Health Tech What’s Keeping Healthcare CIOs Up at Night: How to Improve Patient Satisfaction by Handling Calls More Efficiently Health systems have spent billions on portals while investments in modernizing the voice channel — the dominant preference of healthcare consumers — have taken a backseat. By Stephanie Baum There’s precedent for intranasal drug delivery to hit central nervous system targets. Some migraine drugs work this way. NeOnc’s drug, NEO100, leverages cranial nerves in the brain, Chen said. While these nerves play a role in our senses, including smell and taste, they can also transport drugs across the blood brain barrier. “We want our drug to get to the target, but instead of trying to open up or break through the blood-brain barrier, we’re trying to bypass it,” Chen said. The Idea for Intranasal Drug Delivery to the Brain The active pharmaceutical ingredient in NEO100 is perillyl alcohol (POH), a naturally occurring compound isolated from essential oils of certain plants. While POH is an ingredient in common consumer items such as cosmetics and cleaning products, lab tests have also shown it interferes with the replication of dividing cancer cells, Chen said. However, academic research testing oral POH found not enough of the compound reached the bloodstream to confer a therapeutic effect. Increasing the dose led to intolerable gastrointestinal side effects. sponsored content 5 Health Reasons to Ditch Concrete Jungles for Countryside In an increasingly urbanized world, the allure of countryside living is growing stronger, especially when it comes to health and well-being. By Impact Brands Chen said the idea for delivering POH through the nose came from a NeOnc co-founder, a neurosurgeon who had success testing intranasal delivery of the compound in Brazil. NeOnc’s intellectual property is technology licensed from USC that enables the company to make ultra-pure, pharmaceutical-grade POH. The drug is administered with a commercially available nebulizer. In NeOnc’s Phase 1 dose-escalation study testing NEO100 in 12 glioblastoma patients, published results showed the drug was safe and well tolerated. This trial was not powered to demonstrate efficacy, but Chen noted three patients lived more than three years and one is still alive. For context, glioblastoma patients whose disease has returned after one or more rounds of standard treatment live an average of six months. A genomic analysis of tumors found better responses in those with a specific genetic signature, a mutation to IDH1 enzymes. IDH1 has validation with the recent FDA approval of the first systemic brain cancer therapy for that target, a pill from Servier Pharmaceuticals. NeOnc’s ongoing Phase 2a test is recruiting patients with IDH1 mutations. When NeOnc set preliminary IPO terms this past winter, it aimed to raise about $75 million. NeOnc’s largest shareholder is AFH, a biotech-focused family office. Amir Heshmatpour, managing director of AFH and executive chairman of NeOnc’s board of directors, said institutional investors did not warm to NeOnc, telling him they prefer companies be in late-stage testing or at least have Phase 2 data. “Institutional investors have pulled back in the last five years,” Heshmatpour said. “They want you to de-risk the lead asset as fast as possible with as little money as possible.” A Rare Path to the Public Markets NeOnc withdrew its registration statement in June, then raised $18.5 million, a combination of equity financing and the conversion of debt into equity. The company also stated its intention to go public via a direct listing. Whereas an IPO raises new capital by issuing new shares sold to the public, a direct listing involves no new shares and raises no new money, said Taylor Wirth, a partner at Barnes & Thornburg who specializes in securities transactions. Instead, company insiders sell their shares to the public without involving underwriters, explained Wirth, who has no connection to NeOnc. With no new shares offered, existing shareholders experience no dilution. Going public this way is also cheaper than an IPO because a company avoids paying underwriting fees. Music-streaming company Spotify, which went public in 2018, might be the most prominent direct listing example. The direct listing trend peaked in 2021, when six U.S. companies went public this way, according to PitchBook. From 2018 to mid-2023, the most recent figures available from PitchBook, 14 U.S. companies went public via a direct listing — none in the life sciences. There’s been at least one biotech direct listing this year. FibroBiologics, a regenerative medicine company in early-stage clinical development, completed its direct listing in January. Direct listings are particularly rare for biotech, Wirth said. One reason is that drug development is R&D intensive and companies in this sector need substantial sums of new money to finance that work. The benefits of a direct listing are clearer for well-capitalized companies that do not need extra cash, Wirth said. “I think the original thinking around the direct listing is you could cut out the middle man, cut out the underwriter, and democratize the process of going public,” he said. “But people found services of underwriters are extremely helpful marketing shares, placing them with investors. Going public is a complex legal and business process. Underwriters are helpful navigating those waters.” The biotech IPO market is showing new signs of life. BioAge Labs, a metabolic diseases-focused company collaborating with Eli Lilly, filed its registration statement last week. Two clinical-stage developers of bispecific antibody drugs, Zenas Biopharma and Bicara Therapeutics, have each set financial terms for IPOs expected to price this week. If NeOnc is able to join those companies on the public markets, it aims to continue work on other programs that use NEO100 as a way to deliver other therapies into the brain. NEO212 is comprised of NEO100 conjugated to Merck’s glioblastoma drug. The company also has preclinical research testing NEO100 as a way to deliver the Parkinson’s disease drug levadopa to the brain. But NeOnc’s near-term focus is glioblastoma. Chen expects the Phase 2a test of NEO100 will reach its targeted enrollment of 25 patients by the end of 2024; preliminary data could come in mid-2025. Image: Jolygon, Getty Images