Sunitinib Malate

- PEAK trial first ever study to demonstrate statistical significance over an active comparator in GIST patients, with bezuclastinib plus sunitinib combination demonstrating mPFS of 16.5 months and ORR of 46% in patients who had received prior treatment with imatinib - Cogent is expected to initiate the RTOR process immediately; completion of the PEAK NDA submission expected in April 2026 WALTHAM, Mass. and BOULDER, Colo., Jan. 20, 2026 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has agreed to accept its New Drug Application (NDA) for bezuclastinib in combination with sunitinib for patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib under the Real-Time Oncology Review (RTOR) program. “This milestone reflects the FDA’s recognition of the significant unmet need facing patients with imatinib resistant GIST,” said Andrew Robbins, Cogent’s President and Chief Executive Officer. “Based on positive results from the PEAK trial, the bezuclastinib combination has the potential to be the first new approval in this patient population in over 20 years. We look forward to the continued, close collaboration with the FDA as we advance bezuclastinib toward commercialization.” As announced in November 2025, the bezuclastinib combination in the PEAK trial demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. Additionally, the bezuclastinib combination demonstrated an unprecedented overall response rate (ORR) in imatinib-resistant patients, with 46% of patients treated with the bezuclastinib combination achieving an objective response compared to 26% of patients treated with sunitinib. At the time of this analysis, data for overall survival remains immature. The bezuclastinib combination was generally well tolerated, and no unique risks were observed with the novel combination when compared to the known safety profile of sunitinib. The FDA’s RTOR program allows an applicant to pre-submit components of its NDA to allow the FDA to review clinical trial data before the complete filing is submitted and aims to provide a more efficient review process to ensure safe and effective treatments are available to patients as early as possible. Cogent is expected to initiate the RTOR process immediately with completion of the NDA submission expected in April 2026. Full results from the PEAK trial will be presented at a major medical meeting during the first half of 2026. Additionally, Cogent expects to initiate in mid-2026 a Phase 2 trial investigating the benefit of the bezuclastinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib. About Cogent Biosciences, Inc.Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases. The most advanced clinical program, bezuclastinib, is a selective tyrosine kinase inhibitor that is designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. KIT D816V is responsible for driving systemic mastocytosis, a serious disease caused by unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with strong dependence on oncogenic KIT signaling. In addition to bezuclastinib, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases initially targeting mutations in FGFR2/3, ErbB2, PI3Kα, KRAS and JAK2. Cogent Biosciences is based in Waltham, MA and Boulder, CO. Visit our website for more information at www.cogentbio.com. Follow Cogent Biosciences on social media: X (formerly known as Twitter) and LinkedIn. Information that may be important to investors will be routinely posted on our website and X. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding: the company’s expectation that it will initiate the RTOR process immediately and complete its NDA submission for bezuclastinib in GIST in April 2026; the potential for the bezuclastinib combination to be the first approved therapy for GIST patients in over 20 years; plans to present the full results from the PEAK trial at a major medical meeting during the first half of 2026 and the expectation to initiate in mid-2026 a Phase 2 trial investigating the benefit of the bezuclastinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib. The use of words such as, but not limited to, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar words expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results, the rate of enrollment in our clinical trials and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. We may not actually achieve the forecasts or milestones disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption "Risk Factors" in Cogent's most recent Annual Report on Form 10-K and/or Quarterly Report on Form 10-Q filed with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date hereof. Contact: Christi WaarichSenior Director, Investor Relationschristi.waarich@cogentbio.com617-830-1653

Exelixis CEO Michael Morrissey, Ph.D., said he\'s carefully evaluating whether the company would expand outside the U.S.\n Exelixis may only have one drug on the market right now, but with phase 3 results for a potential new blockbuster on the horizon and a growing pipeline, the Cabometyx maker is aiming big. Specifically, the drugmaker intends to become a top 5 solid tumor company by U.S. sales.“It’s all about building franchises,” Exelixis CEO Michael Morrissey, Ph.D., said in an interview with Fierce on the sidelines of the J.P. Morgan Healthcare Conference in San Francisco. “It’s all about having the mindset that you’re not going to do one compound for one indication, because the economy of scale doesn’t work,” he added.Since its first FDA approval more than 13 years ago in medullary thyroid cancer, cabozantinib—sold as Cabometyx and Cometriq—has been Exelixis’ sole approved drug, driving $2.1 billion in U.S. revenues in 2025, according to a preliminary calculation the company unveiled at JPM. Cabozantinib alone has put the California company at No. 10 on the U.S. solid tumor rankings by sales.Morrissey described cabozantinib as a “franchise molecule,” having recently added its sixth and seventh indications in pancreatic and extra-pancreatic neuroendocrine tumors (NETs), respectively.Now, Exelixis hopes to repeat the success with zanzalintinib, which the company considers “the foundation of future oncology franchises” with the potential to reach $5 billion in peak annual sales. The company has just filed the next-generation TKI with the FDA for use alongside Roche’s Tecentriq in previously treated metastatic colorectal cancer (mCRC). Several other phase 3 programs are either underway or being planned for earlier stages of disease and in other tumor types. Zanzalintinib’s first phase 3 readout was not exactly the most exciting outcome that doctors were hoping for. The phase 3 Stellar-303 study linked zanzalintinib to a 20% reduction in the risk of death compared with Bayer’s Stivarga in patients with previously treated mCRC, meeting one of its dual primary endpoints. Patients’ median overall survival was improved by 1.5 months to 10.9 months with the investigational therapy. That limited magnitude of improvement, combined with an increase in grade 3 or higher adverse events, raised a question around whether the combo could be the new standard of care.The answer to that question is partly dependent on the final analysis of the trial’s other primary endpoint, OS in a subgroup of patients without liver metastases, which Exelixis expects to read out mid-2026.Despite the modest showing in the study, Morrissey noted that doctors have wanted to use an immunotherapy-based regimen for late-line colorectal cancer, and that Stellar-303 offers them their first hope after multiple prior pivotal trial failures within the PD-1/L1 class.As for the non-liver metastases population, Morrissey noted that data were getting close to statistical significance at the last analysis. He acknowledged that showing an OS improvement in the subgroup is important “from a marketing point of view” when educating doctors about zanzalintinib’s overall profile, but added that “it’s not a must-have.”In any case, the bigger fight is not in late-line mCRC. Instead, earlier treatment settings and expansions into other tumor types will be key to zanzalintinib’s success.“We’re moving up,” Morrissey said, referring to the company’s efforts earlier in the treatment sequence.Exelixis recently reached a collaboration with diagnostic company Natera, which will provide a ctDNA assay to identify patients with resected stage 2/3 CRC who have completed definitive therapy but still have molecular residual disease (MRD) to enroll in the phase 3 Stellar-316 trial. This is a high-risk population who likely will progress to metastatic disease within about six months to nine months after surgery and adjuvant chemotherapy, Morrissey noted.Using its ctDNA assay to detect MRD, Natera recently helped Roche turn a previous failure in a broad group of muscle-invasive bladder cancer patients into a phase 3 win in a selective MRD-positive trial population.Partnering with Natera could also save Exelixis some effort when selecting trial sites.“They’ve been screening patients for years,” Morrissey said. “So they know the institutions that use it well, they know the investigators that use it a lot, and they know what patients are already MRD-positive. So it’s the ideal setup to be able to lock and load.”CRC is only piece of the zanzalintinib puzzle. Another major opportunity for the TKI lies in kidney disease, for which Exelixis has formed a clinical development partnership with Merck & Co. In December, Litespark-033, the first Merck-sponsored pivotal trial under the collaboration, kicked off to evaluate zanzalintinib and Merck’s Welireg. A second phase 3 is being planned.In another major milestone expected this year, the phase 3 Stellar-304 study pitting zanzalintinib and Bristol Myers Squibb’s Opdivo against Pfizer’s Sutent in previously untreated patients with advanced non-clear cell renal cell carcinoma is expected to read out mid-2026. Collaborating with the competitionExelixis already knows how to work with Big Pharma through Cabometyx, which Morrissey said highlights the idea of collaborating with the competition. Opdivo was once Cabometyx’s biggest competition in kidney cancer until the CheckMate-9ER trial showed the power of combining the two agents. Results from that trial helped drive a 2.5-fold increase in Cabometyx’s revenue in the ensuing five years, Morrissey said.Exelixis’ franchise-in-a-molecule plan for zanzalintinib includes other tumor types, although Morrissey is letting data determine the biggest indication.“Our job is to do the right selection and the right prioritization and then execute at the highest level, and then the data will tell us where to go,” he said.Even with blockbuster sales pegged to their names, the two TKIs might not be enough to get Exelixis into the “top 5” spot, especially considering that cabozantinib is expected to face generics at the beginning of 2031.“We probably need more,” Morrissey said.The CEO, for now, also wouldn’t pick a potential third pillar of the company following zanzalintinib. Exelixis’ earlier-stage pipeline includes antibody-drug conjugates targeting DLL3, 5T4 and tissue factor; a PD-L1xNKG2A bispecific; and molecular glue and degraders targeting KRAS.On the commercial side, Exelixis has been building out its gastrointestinal cancer sales force following Cabometyx’s NET approvals in March 2025. The hope is that the same sales force can cover zanzalintinib in CRC, too, although Morrissey recognized that the timing of the back-to-back launches may be tight.“Those are good problems to have if you have to scale the organization, because you’re so successful clinically,” Morrissey said.But seemingly running counter to a potential business expansion and its lofty corporate aspirations, Exelixis recently launched a reorganization that includes winding down a site in Pennsylvania and 130 layoffs. Morrissey described the overhaul as the final move in his push to implement a back-to-office policy after the pandemic. RELATED: UPDATED: Exelixis to lay off 130 employees, shut down Pennsylvania site in post-pandemic downsizing “It concentrates all of our critical mass and all of our brain power in Alameda, [California],” Morrissey said.While the satellite operation outside of Philadelphia and its big remote workforce “worked okay,” it “wasn’t nearly as effective” as people working together in the same office, the CEO observed.Exelixis first unveiled the goal to become a “top 5 solid tumor oncology company” during an R&D event in December. During his interview with Fierce, Morrissey put a “U.S.” parameter around that goal because the company has so far relied on partners to handle ex-U.S. markets, including Ipsen in Europe and Takeda in Japan. As the company grows, will it look to build a global footprint?“We’re looking at that very carefully right now,” Morrissey said. “We’re really analyzing all the options that we have relative to ex-U.S. We have deep expertise, and our commercial organization in the U.S. competes with Big Pharma every single day very effectively. So, we’re obviously going to really retrench in the U.S., and then look carefully at how we operationalize the rest of the world.”