Axim Biotechnologies Inc., of New York, said its acquiree, Sapphire Biotech Inc., of San Diego, signed an agreement with Skysong Innovations LLC, the intellectual property management company for Arizona State University (ASU), to exclusively license rights to pharmaceutical compositions and methods for the treatment of cancer. The technology was developed in the laboratory of Sapphire co-founder, Douglas Lake, associate professor at ASU with an adjunct appointment at Mayo Clinic. He discovered that quiescin sulfhydryl oxidase 1 (QSOX1) promotes cancer invasion by remodeling the extracellular matrix that serves as an adhesive surface to tumor cells. The compound, SBI-183, selectively targets QSOX1 and, in animal models, it inhibits tumor growth, invasion and suppresses metastasis of tumors by inactivating QSOX1.
Basilea Pharmaceutica Ltd., of Basel, Switzerland, said it extended its clinical supply agreement with Roche Holding AG, also of Basel, to explore a combination of the company’s panFGFR kinase inhibitor, Derazantinib, and Roche’s Tecentriq (atezolizumab) in patients with gastric cancer. The initial agreement that covered urothelial cancer was concluded in January 2019. Basilea expects to start a biomarker-driven multicohort phase I/II study (FIDES-03) in advanced gastric cancer patients with FGFR genetic aberrations in the third quarter. The study will assess the efficacy and safety of derazantinib as monotherapy and combination therapy in the second-line setting. Basilea will be the sponsor of the study and Roche will provide clinical supply of atezolizumab, a PD-L1 checkpoint inhibitor.
Bavarian Nordic A/S, of Copenhagen, said the sale of its priority review voucher (PRV) has received antitrust clearance. On Dec. 17, the company signed an agreement to sell its PRV for $95 million to an undisclosed purchaser. It was awarded the voucher in September last year upon approval of Jynneos vaccine for the prevention of smallpox and monkeypox.
Biocartis Group NV, of Mechelen, Belgium, said it entered a master collaboration agreement (MCA) with Astrazeneca plc, of Cambridge, U.K., that enables the collaborative development and commercialization of Idylla-based molecular tests in support of Astrazeneca’s pharmaceutical products. The agreement broadens the existing partnership between the companies that originally focused on demonstrating how the platform can overcome the complexity and long turnaround time of biomarker testing for lung cancer patients. A prospective study with the tissue-based Idylla EGFR mutation test, under the existing partnership, was initiated at more than 12 sites located in European countries. That ongoing study will be extended to additional countries within and outside Europe under the MCA.
Celavie Biosciences LLC, of Oxnard, Calif., presented five-year follow-up data on the first-in-human transplantation of undifferentiated stem cells into Parkinsonian patients showing no adverse effects, with improvement in motor function or arrest of disease progression in five of seven patients. Data were presented at the Phacilitate Leaders World and World Stem Cell Summit in Miami.
Genialis Inc., of San Ramon, Calif., which is focused on applied data science for the development of precision medicines, said it established a collaborative partnership with Waltham, Mass.-based Oncologie Inc. Their initial research will focus on the use of Genialis’ Expressions software platform and analytics IP with Oncologie’s clinical and translational expertise to refine patient stratification methodologies. Early work between the two companies has so far focused on modeling gene expression signatures predictive of treatment for gastric cancer. The patent-pending enhanced methodology will further Oncologie’s clinical development plans to expand beyond gastric cancer to other tumor microenvironment phenotypes, the companies said.
Inotrem SA, of Paris, entered a licensing agreement with Roche Diagnostics, a unit of Roche Holding AG, of Basel, Switzerland, for the commercialization of a companion diagnostic test for soluble TREM-1 plasma. The companies have been working on an in vitro test measuring the protein in septic shock patients since 2017. Inotrem is currently testing a TREM-1 inhibitor, nangibotide, in a phase IIb study in septic shock patients. Financial terms of the agreement weren't disclosed.
Ironwood Pharmaceuticals Inc., of Boston, and Allergan plc, of Dublin, settled their lawsuit against Teva Pharmaceuticals USA, a unit of Jerusalem-based Teva Pharmaceuticals Co. Ltd., over patents on their irritable bowel syndrome drug, Linzess (linaclotide). Teva will have a license to market its 145-mcg and 290-mcg generic versions of Linzess in the U.S. starting on March 31, 2029. The settlement doesn't cover the 72-mcg generic version of Linzess.
Ocuphire Pharma Inc., of Farmington Hills, Mich., licensed APX-3330 for all ophthalmic and diabetic indications from Indianapolis-based Apexian Pharmaceuticals Inc. APX-3330 inhibits reduction-oxidation effector factor-1, which regulates transcription factors that control inflammatory and angiogenesis pathways. Ocuphire plans to start a phase II study testing the drug in nonproliferative diabetic retinopathy and diabetic macular edema in 2020.
Pharmacyte Biotech Inc., of Silver Spring, Md., said both batches of the company's Cell-in-a-Box clinical trial product have passed the release tests required for submitting its IND to the FDA for a planned clinical trial in locally advanced, inoperable pancreatic cancer.
Pharmamar SA, of Madrid, and Jazz Pharmaceuticals plc, of Dublin, said the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act for the license of the U.S. rights to lurbinectedin expired on Jan. 21. Pharmamar will receive the $200 million up-front payment from Jazz associated with the agreement in the forthcoming days.
Proteostasis Therapeutics Inc., of Boston, presented an update from the ex vivo study of its cystic fibrosis transmembrane conductance regulator (CFTR) modulators at the Keystone Symposia on Tissue Organoids. The company has established organoid cultures from more than 370 cystic fibrosis patients with plans to reach 500 patients by the end of the first quarter of 2020. To date, 85% of the patients have genotypes that lead to CFTR protein synthesis, making the organoids amenable to testing with the company's CFTR modulators.
Controversy over the FDA's accelerated approval of Cambridge, Mass.-based Sarepta Therapeutics Inc.’s Vyondys 53 (golodirsen) in December sprang back to life Jan. 22 as the agency released the full text of an earlier complete response to the NDA. Such letters, generally not shared with the public by their recipients, contain a detailed view of agency objections to a particular application. At the time of the CRL for golodirsen, Sarepta said the agency had generally cited two concerns: the risk of infections related to I.V. infusion ports and renal toxicity seen in preclinical models of golodirsen and observed following administration of other antisense oligonucleotides. In fact, the letter went further, minimizing the reported effect size of golodirsen and suggesting that the noted infections put "vulnerable boys at ever greater risk of complications and death" than if they had not been treated with the medicine. The CRL, authored by CDER's director of the Office of Drug Evaluation I, Ellis Unger, also took Sarepta's team to task for failing to have begun a confirmatory trial for Exondys 51 (eteplirsen), which was at that point nearly three years overdue, relative to the FDA's expectations that it would be initiated soon after the drug's accelerated approval. (It has since begun.) Despite the concerns, following a September 2019 appeal by Sarepta, CDER's director of the Office of New Drugs, Peter Stein, re-evaluated the evidence around golodirsen, granting the company's appeal and outlining a path to approval. "I request that the company provide a written commitment, prior to approval of golodirsen, that if the results of the confirmatory study do not support a clinical benefit (i.e., no relevant analyses finds sufficient evidence of such a benefit), that they will voluntarily withdraw golodirsen from the market," he wrote in his November 2019 letter to the company. That study is expected to read out in 2023.
Sierra Oncology Inc., of Vancouver, British Columbia, said its board approved a reverse stock split at a ratio of 1-for-40, which will become effective when the market opens on Jan. 23. Shares of Sierra (NASDAQ:SRRA) closed at 35 cents Jan. 22.
Zhittya Genesis Medicine Inc., of Las Vegas, signed a $151.5 million international marketing partnership agreement with Regenerative Medicine of Latin America Inc. for the exclusive rights to market and sell all biological drugs developed by Zhittya during a 30-year time period. The payments include an initial up-front payment valued at $76.5 million with the additional $75 million to be amortized through future milestone payments. Zhittya said it is developing a family of biological drugs to treat diseases characterized by diminished blood flow, or perfusion, to specific tissues or organs. The firm is targeting a variety of disorders and diseases, including coronary heart disease, diabetic foot ulcers, stroke recovery, Parkinson’s disease, Alzheimer’s disease and 14 additional major medical disorders characterized by insufficient blood perfusion.