Point Therapeutics, Inc.

Courtesy of Getty Images BioXcel Therapeutics’ foray into oncology is looking hopeful. Its subsidiary, OnkosXcel, announced positive top-line results from a Phase II trial in patients with small cell neuroendocrine metastatic castration-resistant (SCNC) prostate cancer Wednesday morning. BXCL701 is an innate immune activator being studied in combination with Merck’s Keytruda. OnkosXcel spun out of BioXcel last April as a wholly owned subsidiary to focus on developing its oncology asset. The drug was acquired from Point Therapeutics years after it failed in a Phase III trial as a monotherapy for NSCLC. Immunotherapies often fail to help cancers that appear “cold” or uninflamed to the immune system. BioXcel’s oral therapy is designed to turn cold tumors hot, lighting them up for the immune system to recognize and attack. SCNC is a rare but growing population that emerges in approximately 20% of metastatic castration-resistant prostate cancer patients. The difficult-to-treat indication currently has no FDA-approved therapies for the nearly 11,000 patients whose cancer progressed to SCNC just last year. Experts believe numbers will only increase as checkpoint inhibitor use grows. The trial included 28 patients on a twice-daily dose for two weeks alongside three weeks of intravenously administered Keytruda. Primary and secondary endpoints included a composite response rate, duration of response, progression-free and overall survival and biomarker evaluation. Patients in the trial had previously failed to respond to platinum therapy. In an earlier release of interim trial data, the treatment regimen elicited a response in four out of the twelve radiographically evaluable patients. There was also evidence of a durable response rate in excess of nine months as of the last report. “Keytruda has a negligible response rate in this group [MS stable and TMB low population], so we are highly encouraged to see those responses,” Vincent O’Neill, Chief Research & Development Officer, OnkosXcel Therapeutics, told BioSpace. “Bear in mind that these are elderly men who are pretty beaten up, having already had platinum chemo. We now have a duration of response in excess of nine months in a patient population that literally would have weeks or at best months to live." O’Neill believes regulators will want evidence of individual contribution, so the next step will be a randomization of the Keytruda combo versus a noncomparative randomization of BXCL701 alone. “We know, however, from all the preclinical work we've done, it really is the synergy between the checkpoint and 701. So we'll be having those conversations with the regulators. What type of data set would be required for us to get this drug to market?” O’Neill said. One of the drug’s biggest differentiators from other innate immune activators is its oral delivery. TLRs and STING agents are intertumoral, injected directly into the tumor, yet a large percentage of patients don’t have an injectable lesion available. The asset also inhibits a novel target – DPP89 - for inflammasome activation. BXCL701 has received Orphan Drug Designation from the FDA for four other indications and is being tested in a basket trial in combination with Keytruda for other types of advanced “hot” solid tumors.

HOUSTON and SAN DIEGO, June 24, 2022 (GLOBE NEWSWIRE) -- Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a clinical-stage precision oncology company designing and developing novel targeted therapies for cancer treatment, and The University of Texas MD Anderson Cancer Center today announced a strategic research and development alliance to expand the evaluation of two of Turning Point's investigational small molecules focusing on precision medicine targets well known in oncology. The initial focus of the alliance will be Turning Point’s lead drug candidate, repotrectinib, a next-generation kinase inhibitor targeting the ROS1 and NTRK oncogenic drivers of non-small cell lung cancer and advanced solid tumors that is currently being studied in a registrational Phase 1/2 study (TRIDENT-1). Alliance studies also will include elzovantinib (TPX-0022), a kinase inhibitor targeting MET, CSF1R and SRC, which is currently being studied in a Phase 1 trial of patients with advanced solid tumors harboring genetic alterations in MET (SHIELD-1). The planned focus of the alliance will include monotherapy and potential combinations with other agents – including chemotherapy, immunotherapies and other targeted agents. "This agreement embodies our commitment to further advancing our innovative macrocyclic programs and complementing our development efforts through collaborative alliances with those who share our vision for breakthrough science," said Homa Yeganegi, Senior Vice President and Global Product Development Lead at Turning Point Therapeutics. "We look forward to working with MD Anderson to strengthen our scientific and clinical understanding of our multi-targeted compounds in several tumor types with the goal of speeding delivery of new cancer treatments to patients." The alliance brings together MD Anderson's clinical trial infrastructure and expertise with Turning Point's differentiated targeted oncology pipeline. Under the terms of the alliance’s agreement, which spans a five-year period, collaborative preclinical and clinical studies will be conducted in several solid tumors, including non-small cell lung cancers, gastrointestinal malignancies and endocrine cancers. “There is a major unmet need to develop effective next-generation targeted therapies for cancer patients with oncogene-driven solid tumors, particularly those with mutations that render them resistant to our current therapies,” said John V. Heymach, M.D., Ph.D., Chair of Thoracic/Head & Neck Medical Oncology at MD Anderson. "Our alliance with Turning Point represents an important opportunity to work toward advancing new treatment options for patients using novel inhibitors that target multiple driver mutations with the most characterized resistance patterns in common cancers." The collaborative studies will be overseen by a joint steering committee. Turning Point will provide funding, study materials and other ongoing support throughout the term of the alliance. About MD AndersonThe University of Texas MD Anderson Cancer Center in Houston ranks as one of the world's most respected centers focused on cancer patient care, research, education and prevention. The institution’s sole mission is to end cancer for patients and their families around the world. MD Anderson is one of only 52 comprehensive cancer centers designated by the National Cancer Institute (NCI). MD Anderson is No. 1 for cancer in U.S. News & World Report’s “Best Hospitals” rankings. It has been named one of the nation’s top two hospitals for cancer since the rankings began in 1990. MD Anderson receives a cancer center support grant from the NCI of the National Institutes of Health (P30 CA016672). About Turning Point Therapeutics Inc.Turning Point Therapeutics is a clinical-stage precision oncology company with a pipeline of investigational drugs designed to address key limitations of existing cancer therapies. The company’s lead drug candidate, repotrectinib, is a next-generation kinase inhibitor targeting the ROS1 and TRK oncogenic drivers of non-small cell lung cancer and advanced solid tumors. Repotrectinib, which is being studied in a registrational Phase 2 study in adults and a Phase 1/2 study in pediatric patients, has shown antitumor activity and durable responses among kinase inhibitor treatment-naïve and pre-treated patients. The company’s pipeline of drug candidates also includes elzovantinib, targeting MET, CSF1R and SRC, which is being studied in a Phase 1 trial of patients with advanced or metastatic solid tumors harboring genetic alterations in MET; TPX-0046, targeting RET, which is being studied in a Phase 1/2 trial of patients with advanced or metastatic solid tumors harboring genetic alterations in RET; TPX-0131, a next-generation ALK inhibitor, which is being studied in a Phase 1/2 trial of previously treated patients with ALK-positive advanced or metastatic non-small cell lung cancer; and TPX-4589 (LM-302), a novel ADC targeting Claudin18.2 which is being studied in a Phase 1 study in gastrointestinal cancers. The company is driven to develop therapies that mark a turning point for patients in their cancer treatment. For more information, visit www.tptherapeutics.com. Forward Looking StatementsStatements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “plans,” “will,” “believes,” “anticipates,” “expects,” “intends,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include statements regarding, among other things, Turning Point Therapeutics’ ability to advance its macrocyclic programs and speed delivery of new cancer treatments to patients. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon Turning Point Therapeutics’ current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Turning Point Therapeutics’ business in general, risks and uncertainties related to the impact of the COVID-19 pandemic to Turning Point Therapeutics’ business and the other risks described in Turning Point Therapeutics’ filings with the Securities and Exchange Commission (SEC), including its quarterly report on Form 10-Q filed with the SEC on May 10, 2022. All forward-looking statements contained in this press release speak only as of the date on which they were made. Turning Point Therapeutics undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Contact: Adam D. Levy, Ph.D., MBA ir@tptherapeutics.com858-867-6366 Clayton Boldt, Ph.D.CRBoldt@MDAnderson.org713-792-9518

If you want an idea of the kind of biotech M&A deals to expect in H2, look at the recent spate of buyouts. Endpoints News talked with a pharma investment banker, an experienced dealmaker, a finance professor at MIT and a top analyst to find out what’s center stage these days in the M&A scene, as the bear market mauls biotech stocks and erases follow-on opportunities. The trend of acquiring late-stage biotech companies is expected to continue into the second half of the year, sources told Endpoints. But while there are signs of hope for a quickened pace of M&A — Bristol Myers Squibb’s announcement it would acquire clinical-stage precision oncology company Turning Point Therapeutics for a 122% premium over the previous closing price made some question if the bear market may finally be over — there aren’t any guarantees.