The SPEARHEAD-1 trial included 44 patients and demonstrated significant efficacy, with a median duration of response of 6 months. Adaptimmune plans to roll out the therapy through at least 30 treatment centers within two years. An integrated support program, AdaptimmuneAssist, will help ensure a seamless and personalized experience for patients.
Brandi Felser, CEO of the Sarcoma Foundation of America, remarked, “For decades, therapeutic options for people diagnosed with synovial sarcoma have been limited. Today, there is a renewed sense of hope for this patient community.”
Sandra D’Angelo, MD, from Memorial Sloan Kettering Cancer Center, added, “TECELRA (afami-cel), which uses each patient’s own immune cells to recognize and attack their cancer cells in a one-time infusion treatment, is significantly different than the current standards of care for advanced synovial sarcoma.”
Biomarker tests for human leukocyte antigens (HLA) type and melanoma-associated antigen A4 (MAGE-A4) tumor expression are required before treatment with TECELRA. Adaptimmune has partnered with Agilent Technologies and Thermo Fisher Scientific to provide companion diagnostic products necessary for identifying eligible patients. The treatment comes with significant safety considerations, including potential serious side effects like cytokine release syndrome (CRS) and immune effector cell–associated neurotoxicity syndrome (ICANS).
GSK Bags Sought-after JEMPERLI Okay in Endometrial Cancer
The FDA has granted approval for GSK’s PD-1 inhibitor, JEMPERLI (dostarlimab), for the treatment of all adult patients with primary advanced or recurrent endometrial cancer, marking a significant milestone in the company’s growth strategy for the drug.
This approval is based on data from the RUBY trial, which demonstrated that JEMPERLI, when combined with carboplatin and paclitaxel chemotherapy, reduced the risk of death by 31% compared to chemotherapy alone over two and a half years. According to GSK, JEMPERLI is the only immuno-oncology agent to show a survival benefit in this patient group, bolstering its position against MSD’s Keytruda (pembrolizumab) in the endometrial cancer market.
Previously, JEMPERLI was approved for front-line use in endometrial cancer for patients with mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) tumors based on progression-free survival data from RUBY. Keytruda was also approved for first-line endometrial cancer therapy without label restrictions based on similar data. The new approval for JEMPERLI levels the playing field between the two drugs and highlights the significance of the overall survival data from RUBY Part 1 in their competition for market share.
Luke Miels, GSK’s chief commercial officer, emphasized JEMPERLI’s central role in the company’s immuno-oncology development and its potential to drive future growth beyond dMMR-driven tumors. With more than 70% of endometrial cancer tumors being mismatch repair proficient (MMRp)/microsatellite stable (MSS), the new approval opens up the majority of the market for GSK. An EU approval for this indication is expected in the first half of 2025.
The broader label in first-line endometrial cancer is crucial for JEMPERLI’s projected annual sales to reach blockbuster status. The drug generated £188 million ($240 million) in the first half of this year, a significant increase from £36 million in the same period of 2023, with peak sales projected between £1 billion and £2 billion.
Future growth for JEMPERLI could also come from phase III trials exploring its use in rectal, lung, and head and neck cancers. This includes a new study combining JEMPERLI with GSK’s TIGIT antibody belrestotug as a first-line therapy for PDL1-high non-small cell lung cancer (NSCLC).
Further developments in endometrial cancer include RUBY Part 2, which investigates the combination of JEMPERLI, chemotherapy, and GSK’s PARP inhibitor ZEJULA (niraparib). Results have shown improved progression-free survival with this triplet therapy compared to JEMPERLI and chemotherapy alone.
AstraZeneca is also a competitor in this market, having received FDA approval in June for its PD-L1 inhibitor IMFINZI (durvalumab) in combination with chemotherapy for front-line endometrial cancer treatment, restricted to dMMR tumors. The approval was based on the DUO-E study, which also tested a triple combination including AstraZeneca’s PARP inhibitor LYNPARZA (olaparib), though this was not included in the FDA’s decision.
MBX Biosciences Raises $63.5 Million in Series C Financing to Advance its Precision Endocrine Peptide™ (PEP™) Platform
MBX Biosciences, Inc., has successfully closed a $63.5 million Series C financing round. This funding round was led by Deep Track Capital and included new investors Driehaus Capital Management and T. Rowe Price Associates, Inc., as well as existing investors Frazier Life Sciences, OrbiMed, and Wellington Management. This influx of capital is expected to support the company’s research and development efforts through 2026, covering critical milestones and data read-outs.
The new funding will accelerate MBX’s clinical programs, including a Phase II trial of MBX 2109, a parathyroid hormone peptide prodrug aimed at treating hypoparathyroidism. Topline results from this trial are anticipated in the third quarter of 2025. Additionally, MBX 1416, a candidate for post-bariatric hypoglycemia, is currently undergoing a Phase I single and multiple ascending dose trial, with topline data expected in late 2024. The company is also advancing its portfolio in obesity and metabolic diseases, including MBX 4291, a long-acting GLP-1/GIP receptor co-agonist prodrug currently in IND-enabling studies.
Gerard Smith, M.D., Managing Director at Deep Track Capital, expressed enthusiasm about MBX’s innovative approach and their ability to transform scientific discoveries into effective therapies. The Series C financing underscores the confidence investors have in MBX’s team and their groundbreaking work in the endocrine and metabolic drug space.
FDA Halts Actinium’s Radiotherapy Blood Cancer Plans, Prompting a Shift to Partnering
Actinium Pharmaceuticals has faced a major hurdle with the FDA rejecting its application for approval of Iomab-B, a targeted radiotherapy for acute myeloid leukemia. The setback comes despite promising results from the Phase III SIERRA trial, which showed Iomab-B achieving a higher rate of complete remission compared to standard care. However, the FDA has requested an additional head-to-head randomized trial specifically designed to demonstrate a survival benefit, which Actinium had not anticipated.
The SIERRA trial showed a median overall survival (OS) of 6.4 months for patients receiving Iomab-B versus 3.2 months for those in the control group. Despite this, the analysis was complicated by a significant portion of control patients crossing over to the Iomab-B group, leading to a request for further validation without such crossovers. This additional trial will be necessary to meet the FDA’s requirements for approval.
In light of the FDA’s demands, Actinium has decided to seek a partner for Iomab-B rather than advancing it independently. The company will now shift its focus to other development programs, including Actimab-A, Iomab-ACT, and several preclinical candidates. This decision has sharply impacted Actinium’s stock, which fell 69% in premarket trading. The company, ending March with $84 million in cash, will need to navigate these challenges while exploring new opportunities in its pipeline.
Otsuka Pays Up to $1.1B to Buy Jnana, Adds Drug Discovery Capabilities
Otsuka Pharmaceutical Co., Ltd. has entered into a definitive agreement to acquire Jnana Therapeutics Inc. for up to $1.125 billion. The deal involves an initial payment of $800 million to Jnana’s shareholders, with an additional $325 million contingent upon achieving development and regulatory milestones. The acquisition is expected to be completed by the third quarter of fiscal 2024, pending customary closing conditions.
Jnana Therapeutics is renowned for its RAPID chemoproteomics platform, which is designed to discover drugs for challenging targets that are difficult to address with traditional methods. The platform has enabled Jnana to identify innovative compounds and pursue drug discovery for rare diseases and autoimmune conditions. Notably, Jnana has developed JNT-517, an allosteric small molecule inhibitor that shows promise as a first-in-class oral treatment for phenylketonuria. This compound has demonstrated effectiveness and safety in early-stage clinical trials, potentially benefiting a large segment of the PKU population that currently lacks effective treatment options.
In addition to its work in PKU, Jnana is focusing on autoimmune diseases, targeting challenging proteins such as interferon regulatory factor 3 (IRF3), a key transcription factor involved in the production of interferon.
Otsuka’s President and Representative Director, Makoto Inoue, highlighted the strategic value of the acquisition: “I am gratified that Otsuka has entered into an agreement with Jnana. The addition of Jnana’s drug discovery technology and small molecule pipeline in PKU and autoimmune diseases will strengthen our R&D in the Boston area of the U.S., one of the most important bioclusters in the world, and in a combined form will have a synergistic effect on Otsuka Pharmaceutical’s global expansion.”
Joanne Kotz, Ph.D., CEO and co-founder of Jnana Therapeutics, expressed enthusiasm about the merger: “This transaction acknowledges our achievements with the RAPID platform and JNT-517 for PKU. We look forward to advancing JNT-517 into a registrational study in 2025 and further developing our pipeline of oral medicines for autoimmune diseases with Otsuka.”
Following the acquisition, Jnana will continue its operations in Boston as a wholly owned subsidiary of Otsuka. The deal is subject to approval by Jnana’s shareholders and compliance with U.S. antitrust regulations.
