Opna Bio SA

SAN DIEGO--( BUSINESS WIRE )--Opna Bio, a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics, announced interim data from its lead clinical program, OPN-2853, in patients with advanced myelofibrosis showing encouraging levels of spleen reduction, with minimal toxicities and adverse events. Preclinical data from a second program, OPN-6602, showed significant tumor regression in multiple myeloma models as a single agent and in combination with other therapeutics. The presentations took place this weekend at the American Society of Hematology (ASH) annual meeting, December 7-10, 2024, in San Diego. OPN-2853 Shows Spleen Reduction in Patients with Advanced Myelofibrosis OPN-2853, a small molecule bromodomain and extra-terminal motif (BET) inhibitor, is being evaluated in an ongoing Phase 1 investigator-led study in patients with advanced myelofibrosis who are no longer responding to ruxolitinib alone. Myelofibrosis is a type of blood cancer that causes fibrosis in the bone marrow, anemia and enlarged spleen, amongst other symptoms. The study is testing three dose levels of OPN-2853 (20 mg, 40 mg and 80 mg), given orally once daily, in combination with ruxolitinib. As of February 2024, the cut-off date, 16 patients had been enrolled at different dose levels, across multiple sites in the United Kingdom, coordinated by the Cancer Research UK (CRUK) Clinical Trials Unit at the University of Birmingham. In 12 evaluable patients, the median spleen size was reduced from baseline with spleens no longer palpable in 50% of evaluable patients. The combination dose has been well tolerated, and the majority of patients have completed eight cycles of combination treatment. “We are very encouraged by these data to date, which demonstrate that daily dosing of OPN-2853 in combination with ruxolitinib was well tolerated, and showed spleen reduction in patients with myelofibrosis who have very limited options once they have progressed,” said principal investigator Adam Mead, PhD, MRCP, FRCP, professor of hematology at the University of Oxford and CRUK senior cancer research fellow. “We are enthusiastic about the OPN-2853 and ruxolitinib combination and expect to have a recommended Phase 2 dose in early 2025.” Opna Bio is planning further clinical development with OPN-2853. OPN-6602 Combinations Demonstrate Complete Tumor Regression in Preclinical Multiple Myeloma Models OPN-6602, an oral, small molecule inhibitor of the E1A binding protein (EP300) and CREB-binding protein (CBP), is currently being tested in a Phase 1 trial in patients with multiple myeloma (MM) . MM is a type of blood cancer derived from malignant plasma cells in the bone marrow. In MM mouse xenograft models, OPN-6602 showed 71% tumor suppression as a single agent, and 100% tumor regression when combined with dexamethasone, pomalidomide or mezigdomide with a sustained duration of response. RNA sequencing of treated tumors showed that OPN-6602 downregulated key MM driver and signature genes, and has the potential to overcome resistance mechanisms to standard-of-care regimens. “We are very encouraged by the strength of the OPN-2853 clinical and OPN-6602 preclinical data, which highlights the potential of their unique pharmacokinetic profiles and validates our ‘safety by design’ approach. Both drug candidates have a high Cmax and short half-life, which allows for continuous daily dosing and effective target engagement with rapid clearance to mitigate toxicities,” said Gideon Bollag, PhD, co-founder and chief scientific officer of Opna Bio. “This confers a significant advantage to these drugs when used as single agents and a potentially synergistic effect when used in combination with other therapies.” About Opna Bio Opna Bio is a clinical-stage biopharmaceutical company focused on the discovery and development of novel oncology therapeutics. The company’s broad portfolio targets multiple drivers of cancer, including a novel oncology discovery program focused on the fragile-X multifunctional RNA-binding protein (FMRP) and a diversified pipeline of promising oncology assets. The Opna team has a proven track record of scientific expertise and commercial value creation, having advanced multiple FDA-approved drugs to market. Opna’s lead clinical compounds include OPN-2853, a potentially best-in-class BET bromodomain inhibitor, being evaluated in patients with myelofibrosis in combination with ruxolitinib, and OPN-6602, a dual EP300/CBP inhibitor, currently being studied in a first-in-human Phase 1 clinical trial in multiple myeloma patients. For more information, please visit opnabio.com .

→ Kristen Mulholland will take over for the retiring Peter Fasolo as chief human resources officer at J&J on Oct. 1. “Peter has led J&J’s talent strategy for the past 16 years including remarkable leadership through the global pandemic and the separation of our consumer health business,” CEO Joaquin Duato said in a statement . “He leaves a legacy of strong leaders, deep bench strength and an inclusive, caring and patient-focused culture.” Fasolo is a Bristol Myers Squibb HR vet who left J&J to become chief talent officer for KKR , only to return in 2010. Mulholland started at J&J in 2005 and has spent nearly five years as head of human resources, global total rewards & enterprise HR functions. → Just do it: Amgen technology chief David Reese said in a LinkedIn post that the pharma giant has appointed Sean Bruich as SVP of artificial intelligence and data. Bruich moves into the pharma world after an 11-year run with Nike , where he vaulted to global VP, insights, data science & analytics. This is the second high-level AI hire by a major pharma in as many weeks — Pfizer just welcomed Berta Rodriguez-Hervas as its new chief AI and analytics officer. → In its biggest personnel news since Doug Williams ’ resignation , Sana Biotechnology has poached Dhaval Patel from UCB as the Seattle biotech awaits data readouts for its CD19-targeted allogeneic CAR-T therapy in a host of indications. Patel had a 10-year career with Novartis before he held the CSO post at UCB for nearly seven years, and he’ll have the same responsibilities at Sana under CEO Steve Harr . Sana said in its second-quarter update that the goal is to reveal data for SC291 in B cell malignancies — and such autoimmune diseases as lupus nephritis, extrarenal lupus and ANCA-associated vasculitis — sometime this year. “His expertise in immunology and autoimmune diseases make[s] him ideally suited to help advance our clinical pipeline,” Harr said in a statement . → Brian Di Donato , the CFO and head of strategy at Immunocore since May 2020 , is stepping down “at the end of the year” to take the CEO job at an unnamed San Diego biotech. Di Donato was the finance leader for Achillion when Alexion scooped it up for $930 million, and he’s also a board member at Jim Wilson joint iECURE , where ex-Achillion chief Joseph Truitt is CEO. → Telix Pharmaceuticals has separated its business into four separate entities: Telix Therapeutics , Telix Precision Medicine (diagnostics), Telix Lightpoint (medtech, from its acquisition of Lightpoint Medical ) and Telix Manufacturing Solutions . With these changes in place, Telix has tapped Richard Valeix as CEO of Telix Therapeutics, Kevin Richardson as Telix Precision Medicine’s chief executive, and Raphael Ortiz as CEO of Telix International . Christian Behrenbruch ’s title has been slightly modified to group CEO. Telix had hoped to join the Nasdaq in June with an IPO around the $200 million mark, but the Melbourne biotech made a gametime decision to pull those plans . → Cambridge, MA-based neuro player Cognito Therapeutics has promoted Christian Howell to CEO. Brent Vaughan had been running Cognito since 2020 after co-founding and leading the autism diagnostic specialist Cognoa , and he hired Howell as his chief commercial officer last October. Howell had a long career with Medtronic and then joined Aetion , where he was elevated to SVP and general manager of the emerging channels group (medical device, diagnostics, digital health, and government) in April 2023. → Elsewhere, Endo is also losing its president and CEO Blaise Coleman . Board member Scott Hirsch will serve in the interim as the company searches for a permanent successor. Hirsch joined Endo’s board in April, around the same time that the company sued the FDA over its approval of a generic version of Endo’s drug Adrenalin . Hirsch is the former CEO of Solta Medical and also held the roles of president of Ortho Dermatologics and CBO of Bausch Health / Bausch + Lomb . → Thomas Bock has handed in his resignation as CEO of Notable Labs , and CSO Joseph Wagner will take over the top spot on an interim basis. This isn’t uncharted territory for Wagner: He’s the former CEO of Oncocyte (2011-15) and Canadian I/O biotech BriaCell Therapeutics (2015-16). Notable Labs merged with VBL Therapeutics in February 2023 and will start enrolling patients in its Phase 2 study of the ex- Boehringer Ingelheim drug volasertib for acute myeloid leukemia “in the coming months,” the company said in July. → Delfi Diagnostics marked the beginning of the year with a new CEO it hired away from Illumina . Now, the competitor to Grail has brought on Thomas Russo as finance chief. Russo served in the same role at Icosavax and Assembly Biosciences . During his time at Icosavax, Russo helped with the company’s growth until its $838 million upfront acquisition by AstraZeneca . → Marea Therapeutics , the Third Rock upstart led by ex- Graphite Bio CEO Josh Lehrer , has selected Ian Clements as CFO. Clements resigned from the same post at Livmarli maker Mirum Pharmaceuticals in May 2023 after four years overall with the company, and he’s the ex-VP of investor relations for Dermira . Marea launched in June with $190 million in “combined Series A and B” funding, testing an ex-Novartis monoclonal antibody that targets ANGPTL4 in adults with metabolic dysfunction. → With ex- Synlogic CEO Aoife Brennan at the controls, a seemingly rejuvenated Eliem Therapeutics has named Brett Kaplan as COO and Nishi Rampal as SVP, clinical development. Kaplan had been president and chief financial & corporate development officer for epigenetic editing upstart Chroma Medicine , and he was also the finance chief at Prevail until Eli Lilly bought the gene therapy biotech for $1 billion. An Alexion vet, Rampal also handled the role of global development and strategy lead for the neuromyelitis optica spectrum disorder (NMOSD) treatment Uplizna as an executive director with Horizon /Amgen. Eliem’s hunt for strategic alternatives ended with the acquisition of Tenet Medicines and its anti-CD19 antibody budoprutug , once known as TNT119 . → Backed by RA Capital and Forbion , Navigator Medicines disclosed its $100 million Series A this week and the recruitment of Dana McClintock as CMO. McClintock spent six years in the same position at Alladapt Immunotherapeutics and was global head of the Office of I2O Innovation for Genentech . Navigator also said that Forbion general partner Wouter Joustra and RA Capital managing director Andrew Levin have made their way to the board of directors. → Alex Bastian has been named VP, commercial strategy at Swiss-based Memo Therapeutics , reuniting with CEO Erik van den Berg . Toward the end of van den Berg’s tenure as CEO of AM-Pharma , Bastian was the Dutch biotech’s VP, commercial & patient access. He also worked for Galapagos as VP, head of value & market access. → Structure Therapeutics , one of many biotechs developing a GLP-1 drug for obesity, has elected ex- Shire CEO Angus Russell to the board of directors. The 68-year-old Russell has chaired the board at Daxxify maker Revance Therapeutics since 2014 and has a board seat at Lineage Cell Therapeutics . → Ex- Vividion CEO Jeff Hatfield has joined the board of directors at Gate Bioscience , a California drug developer that launched last November with a $60 million Series A from several blue-chip investors. Hatfield chairs the board at Vividion and Rome Therapeutics . → Opna Bio has made room for Inozyme co-founder and former CEO Axel Bolte on the board of directors. The South San Francisco biotech also announced that it had dosed the first patient in a Phase 1 trial of its multiple myeloma candidate OPN-6602 and appointed Myeloma Investment Fund managing director Stephanie Oestreich as a board observer.

More than 1,000 clinical trials for acute myeloid leukemia drugs have been launched globally since 2019, a new report said. Europe has launched the most clinical trials for cystic fibrosis (CF) treatments, while North America leads in those for acute myeloid leukemia (AML), according to a pair of new global trial landscape reports announced April 10 by contract research organization Novotech. Global venture funding for R&D on treatments for both diseases topped $1 billion each over the past five years.  CF is caused by an inherited mutation in the gene CFTR that damages mucus-producing cells in the lungs, pancreas and other organs. Though there is currently no cure, the past four decades have seen significant progress on treatments. Before the 1980s, half of CF patients survived into their 20s, the Novotech report noted. By 2025, 75% of patients with CF are expected to live to adulthood. Drugmakers since 2018 have launched 450 clinical trials for CF drugs worldwide, with 37% in Europe and 35% in North America, the report said. The field includes Vertex Pharmaceuticals, the creator of a powerful but pricey drug trio called Trikafta that in 2019 became the first FDA-approved triple combination therapy for the most common genetic mutation in CF. The company has several investigational CF treatments currently in late-stage clinical trials on top of four therapies already on the market.  Worldwide venture capital funding for CF research totaled just over $1 billion between 2019 and 2023, the report said, with the U.S. leading at $919 million followed by the Netherlands, Israel and Italy. ReCode Therapeutics, Adaptive Phage Therapeutics, Metagenomi and Carmine collected the most VC dollars. ReCode, Metagenomi and Carmine are all working on genetic medicines, while Adaptive Phage (recently acquired by BiomX) is studying whether viruses called bacteriophages can clear dangerous bacteria from the lungs of those with CF.  Another Novotech report highlights the landscape for AML, a type of blood cancer that begins in the blood stem cells in the bone marrow. It’s the second most prevalent blood cancer in adults and children but has the lowest survival rate. Fewer than half of patients live beyond five years of diagnosis. Pharma companies have attempted to improve the odds by launching more than 1,000 trials for AML treatments globally since 2019, according to Novotech. North American trials account for about 40% of those trials, with another third in the Asia-Pacific region and 22% in Europe. Venture funding for AML R&D from U.S. firms totaled about $1.8 billion between 2019 and 2023, with another $1.2 billion in China over the same period. The top-funded companies for AML drugs within the same time span were Opna Bio, Shorla Oncology and OncoVerity.  Novotech runs clinical trials for clients in the APAC region, the U.S. and Europe. The CRO is based in Australia and has more than 3,000 staff worldwide, according to the company.