Senior Medical Director

Axsome Therapeutics and Revolution Medicines have delivered strong returns this year, with shares climbing 34% and 135% respectively in the first half. Both biotechnology companies attracted investors seeking growth opportunities beyond artificial intelligence. Axsome specialises in central nervous system disorders and has three marketed products. In the recent quarter, Auvelity sales rose 59% to $153 million, whilst Sunosi revenue increased 34% to $33 million. The company recently secured approval for Auvelity in treating Alzheimer's agitation and has five phase 3 programmes underway. Revolution Medicines, whilst earlier-stage with no commercialised products yet, has candidates in registrational trials for pancreatic cancer and non-small cell lung cancer treatment. The company recently announced positive phase 3 results for its lead oncology candidate.

Verastem Oncology's pancreatic cancer combo shows 86% OS rate. Verastem's data in pancreatic cancer follows Revolution's impressive daraxonrasib readout at ASCO 2026. Verastem Oncology's pancreatic cancer combination has shown an 86% overall survival (OS) rate in a Phase Ib/IIa trial. In the RAMP 205 study (NCT05669482), 29 patients were enrolled into the recommended Phase II dose cohort, which evaluated Avmapki Fakzynja (avutometinib plus defactinib) in combination with gemcitabine and nab-paclitaxel in first-line metastatic pancreatic ductal adenocarcinoma (PDAC). In the Phase Ib/IIa study, patients were treated with avutometinib 2.4mg twice weekly, defactinib 200 mg twice daily for three weeks on and one week off, and gemcitabine (800 mg/m2) plus nab-paclitaxel (125 mg/m2) administered on days one, eight and 15 of each 28-day cycle. At diagnosis, 90% of patients presented with metastatic disease. As of the 5 June 2026 data cutoff, with a median follow-up of 9.8 months, the combination demonstrated encouraging clinical activity, including an 86% OS rate at six months, with this data continuing to mature. The progression-free survival (PFS) rate at the same six-month time point was 68%, and the confirmed objective response rate (ORR) was 52%. The majority (83%) of patients experienced tumour shrinkage. Nine patients remain on treatment at the recommended Phase II dose. Adverse events (AEs) remained generally consistent with the previously reported safety and tolerability profile, with no new safety signals observed. Dr John Hayslip, CMO of Verastem Oncology, said: "The updated data from the RAMP 205 trial provide important clinical insights into the potential impact of combined RAF/MEK and FAK inhibition as a therapeutic option for pancreatic cancer. While pancreatic cancer remains one of the most challenging cancers to treat, the early survival trends and deep responses observed in this study indicate that Avmapki Fakzynja is combinable with standard-of-care chemotherapy and may help overcome resistance mechanisms inherent in pancreatic cancer and support further exploration of strategies designed to address oncogenic signalling and mechanisms of treatment resistance." The RAMP 205 trial was designed to evaluate whether simultaneous inhibition of KRAS-driven signalling and FAK-mediated resistance pathways, in combination with standard-of-care chemotherapy, could improve outcomes for patients living with metastatic pancreatic cancer. KRAS is mutated in more than 90% of pancreatic cancers, making it a key driver of tumour growth. Pancreatic cancer is the third leading cause of cancer-related death in the US and the seventh leading cause of cancer-associated mortality worldwide. Metastatic pancreatic cancer, or stage IV disease, occurs when the cancer spreads beyond the pancreas to distant organs. Pharma's giant step in pancreatic cancer drug development. Pancreatic cancer has historically been difficult to treat. This, however, could be nearing an end. Data presented at the American Society of Clinical Oncology (ASCO) meeting 2026 showed that Revolution Medicine's oral RAS(ON) inhibitor, daraxonrasib, achieved a median OS rate of 13.2 months, almost double that of chemotherapy (6.7 months). In Revolution's Phase III RASolute 302, which was presented at the ASCO plenary session, investigators also touted that the survival benefit was matched by a meaningful improvement in progression-free survival (PFS) of 7.2 months vs 3.6 months. Response rates nearly tripled with an objective response rate (ORR) of 31.6% compared to 11.2% in the chemotherapy arm. The field is ecstatic about the prospect of a targeted therapy meaningfully extending OS at a rate that is higher than many frontline chemotherapy regimens achieve. In conversation with Clinical Trials Arena, Jess Paulus, real-world data (RWD) and pharmacoepidemiologist leader at Ontada, who attended ASCO 2026, said: "The readout from the Phase III pancreatic trial is a once-in-a-lifetime kind of experience. It has been nearly 30 years of research from first discovery to where that trial is right now. That kind of readout doesn't happen every ASCO." While Dr Deborah Patt, executive VP of policy and strategy for Texas Oncology, added: "I have been an oncologist for 20 years, and we've really not had such groundbreaking changes in pancreatic cancer. We need to continue to improve, but it is the first real breakthrough in 20 years. I'm incredibly excited about it." GlobalData anticipates sales for daraxonrasib to surpass $1bn by 2029, reaching $4.3bn by 2032. GlobalData is the parent company of Clinical Trials Arena. Give your business an edge with its leading industry insights.

ASCO 2026: A revolution in pancreatic cancer. June 1, 2026 The overall survival data for Revolution Medicines Inc.'s phase III RASolute 302 study in previously treated, metastatic pancreatic ductal adenocarcinoma induced a mid-presentation standing ovation during the plenary session at the 2026 American Society of Clinical Oncology Annual Meeting. The pan-RAS inhibitor daraxonrasib reduced the risk of death by 60% both in patients with RAS G12 mutation as well as the overall population, which included patients with other mutations as well as wild type RAS. "It is an absolutely beautiful curve," the discussant said of the Kaplan-Meier curve during their review of the presentation. Hanmi, Haisco win billion-dollar partnerships with Eli Lilly. Hanmi Pharmaceutical Co. Ltd. secured a $1.26 billion deal with Eli Lilly and Co. to out-license ex-Korea rights to sonefpeglutide (HM-15912), a Lapscovery-based GLP-2 analog in development for multiple indications, including an ongoing phase II study of short bowel syndrome. It was one of two billion-dollar Asian company deals signed by Lilly on June 1, with the second transaction involving Haisco Pharmaceutical Group Co. Ltd., of Beijing. Haisco's licensing and research collaboration to develop medicines across multiple therapeutic areas is worth $87 million in up-front and near-term payments, as well as up to $2.967 billion in milestone payments, plus single-digit tiered royalties on any product sales. Drugmakers go WEE1 at ASCO 2026. At the 2026 American Society of Clinical Oncology Annual Meeting, multiple companies presented data for their drugs targeting WEE1, a checkpoint for the transition from G2 into mitosis. Aprea Therapeutics Inc. presented phase 1 data for its WEE1 inhibitor APR-1051 in patients with advanced solid tumors showing two patients with endometrial cancers achieved partial responses in the dose-escalation study. Likewise, Zentalis Pharmaceuticals Inc. had tantalizing early results from a phase Ib study showing azenosertib plus paclitaxel in patients with platinum-resistant ovarian cancer produced an overall response rate of 39% with a median progression-free survival of 7.3 months. Oculis shifts focus on phase III DME disappointment. Oculis SA is shifting focus to two other pipeline programs after its most advanced candidate, OCS-01, failed to hit the primary endpoint in phase III testing, delaying the possibility of a topical option for treating diabetic macular edema (DME). An eye-drop formulation of high concentration dexamethasone, OCS-01 had previously shown promising results in stage 1 of the Diamond-1 phase III trial, including statistically significant improvement in mean BCVA at the three-month mark. However, a higher-than-expected placebo response prevented OCS-01 from hitting the visual acuity improvement goal at the 52-week timepoint. Shares of Oculis (NASDAQ:OCS), which dropped 23% the afternoon of May 29 when the news was announced, were trading down 34% this morning. Phase II interim peek yields upbeat findings for Turn in AD. Turn Therapeutics Inc.'s positive data from an interim analysis included the first 50 subjects in the phase II trial in atopic dermatitis (AD) with topical IL-36 inhibitor GX-03. The randomized, double-blind, vehicle-controlled study is evaluating GX-03, an extended-release formulation of polyhexanide, widely used in Europe as a broad-spectrum disinfectant and antiseptic, in adults with moderate to severe disease. Heeding advice from the firm's data monitoring committee as well as independent statisticians, and clinical/regulatory advisors - including strategy overseer Stephen Hahn, former head of the U.S. FDA - Turn used the completed cohort of patients as an integrated first-stage analysis. The idea is to better evaluate responder dynamics, endpoint sensitivity, and inflammatory burden behavior as a way of optimizing the trial's second stage. Shares of the Westlake Village, Calif.-based firm (NASDAQ:TTRX) were trading at $6.16, up 17%, or 91 cents. Edgewise sells muscular dystrophy assets to Servier for $2.65B. Edgewise Therapeutics Inc. is pulling in $1.55 billion up front by selling its muscular dystrophy business, including its fast skeletal myosin inhibitor, sevasemten, to Servier SA. The deal is potentially worth up to $2.65 billion when including potential $1.1 billion in milestone payments. Once Suresnes, France-based Servier takes over the business, Edgewise, of Boulder, Colo., will become solely a cardiovascular disease-focused company, with a pipeline consisting of EDG-7500 for hypertrophic cardiopyopathy, EDG-15400 for heart failure with preserved ejection fraction, and EDG-003 for an undisclosed target. ASCO 2026: PD-(L)1 x VEGF bispecifics fight it out. Multiple companies are chasing Akeso and Summit Therapeutics Inc. in the battle to potentially dethrone Keytruda (pembrolizumab, Merck & Co. Inc.) as the top cancer immunotherapy with bispecific antibodies targeting PD-(L)1 and VEGF. Data from clinical studies testing drugs targeting the duo from Biontech and 3SBio - recently licensed to Pfizer Inc. - were presented in rapid oral abstract sessions at the 2026 American Society of Clinical Oncology Annual Meeting. With the most advanced drug in the class, Akeso and Summit got a spot in the plenary session although the data from the Chinese study was met with a bit of skepticism over the applicability of the results to the global population of lung cancer patients. Policy to have bigger role in US grants? The Trump administration's efforts to ensure U.S. federal grants align with its policies may soon be coming to fruition. The White House Office of Management and Budget (OMB) released a proposed rulemaking Friday to revise its Guidance for Federal Financial Assistance. The overarching goal, according to the OMB, is to improve transparency, accountability and oversight for how federal tax dollars are used across the government in awarding and managing grants, cooperative agreements and other forms of federal financial assistance. With one of the stated purposes being to ensure that such grants and awards align with the president's policies, the proposed changes echo executive orders President Donald Trump issued last year and earlier this year while increasing concerns of further politicizing the grant process. ASCO 2026: Grail shifts focus to secondary endpoints in UK study. New data from Grail Inc.'s randomized study of its Galleri multicancer early detection test could not alter the fact that the U.K. trial missed the primary endpoint of reducing the number of cancers diagnosed at stage III and stage IV. But the company is now suggesting that discounting the initial screen, when most late-stage cancers were detected, would be more representative of a real-life steady state screening program. EASL 2026: Emerging liver disease targets to watch. At the recently concluded European Association for the Study of the Liver meeting, presentations underscored how increasingly granular insights into liver pathobiology are driving the rapid identification of new druggable targets across diverse indications. One such example was presented during the poster session by researchers from CIC Biogune, whose work builds on emerging evidence that disrupted magnesium homeostasis may drive cellular stress, epithelial dysfunction and fibrogenic activation - key processes underlying chronic cholestatic disorders such as primary biliary cholangitis and primary sclerosing cholangitis. Also in the news. Abbvie, Abiomed, Agios, Allogene, Altimmune, Astrazeneca, Braeburn, Bristol, Cercare, Coredio, Cumberland, Cumulus, Dizal, Draig, Edap, Getinge, Idb, Immunogenesis, Intuitive, Johnson, Longbio, Merck, Moleculin, Nestle, Neuropace, Optimi, OSE, Otsuka, Outlook, Panther, Pfizer, Phanes, Regeneron, Replimune, Samsung Bioepis, Sciclone, Sequana, Siemens, Skia, Spectral, Tempest, Tempus, Zydus To read more on related topics, click on one of the words below.