Shareholders of Achillion Pharmaceuticals Inc., of Blue Bell, Pa., approved the company’s acquisition by Alexion Pharmaceuticals Inc. The transaction is expected to close in the first half of 2020. The price tag is approximately $930 million, or $6.30 per share in cash for each share of Achillion common stock with potential nontradeable contingent value rights for Achillion shareholders if milestones are met, such as FDA approval of (AHC-4471) danicopan to treat paroxysmal hemoglobinuria and initiating the ACH-5228 phase III study to treat immune-related diseases.
Addex Therapeutics Ltd., of Geneva, Switzerland, filed an SEC registration statement for a proposed Nasdaq listing. The company is not proposing to register any new issuance of securities and its shares will continue to be traded on Zurich’s Six Swiss Exchange.
Akcea Therapeutics Inc., of Boston, will retain development and commercialization rights to AKCEA-APOCIII-LRx since Novartis AG passed on its option. Akcea plans to develop AKCEA-APOCIII-LR for familial chylomicronemia syndrome or other indications impacted by reducing APOCIII and triglycerides. Results from a phase II study of AKCEA-APOCIII-LRx are due in 2020. AKCEA-APOCIII-LRxAKCEA-APOCIII-LRx is a ligand conjugated antisense drug designed to reduce apolipoprotein C-III production in the liver.
Amag Pharmaceuticals Inc., of Waltham, Mass., awarded four independent research grants totaling about $300,000 to study preterm birth reduction and preeclampsia to Jaipal Singh, of the Indiana University School of Medicine, and Jennifer Sasser, associate professor at the University of Mississippi Medical Center, who are researching pharmacological and molecular validation of dimethylarginine dimethylaminohydrolase as a therapeutic target for preeclampsia; Rupsa Boelig, Walter Kraft and Leonard Edelstein, all of Thomas Jefferson University, who are researching aspirin for preventing preeclampsia; Wayne State University’s Jennifer Condon and Gil Mor to research targeting circulating GRP-78 to prevent preterm labor; Anum Minhas, Allison Hays, Arthur Vaught and Sammy Zakaria, all of Johns Hopkins Medicine, to research imaging methods to understand the association of preeclampsia and cardiovascular disease.
Arcus Biosciences Inc., of Hayward, Calif., and Genentech Inc. will evaluate combinations with AB-928, a dual antagonist of adenosine receptors A2aR and A2bR, in two studies: third-line metastatic colorectal cancer and first-line metastatic pancreatic cancer. Each company is supplying its anticancer agent to support the jointly funded studies. Additional financial terms were not disclosed.
Askbio Biopharmaceutical Inc., of Research Triangle Park, N.C., exercised its option to exclusively license, develop and commercialize Watertown, Mass.-based Selecta Biosciences Inc.’s immune tolerance platform for use in adeno-associated virus gene therapy for the treatment of Pompe disease. When used in combination with AAV gene therapy vectors, Selecta’s platform has been shown to inhibit the immune response to the vector. Selecta and Askbio previously announced their partnership in August to jointly develop, manufacture and commercialize targeted therapeutics for next-generation AAV gene therapies. Under the terms of this new license agreement, Selecta is eligible to receive up-front and milestone payments of over $240 million plus royalties on product sales.
Axogen Inc., of Alachua, Fla., received a grant from the Advanced Regenerative Manufacturing Institute to research and develop an automated bioprocessing system for human tissue scaffolds. The institute will reimburse up to $804,000 in costs during a period not exceeding 30 months, while Axogen retains intellectual property rights developed during the project.
Beyondspring Inc., of New York, published details on the mechanism of action of its lead asset, Plinabulin, showing it can successfully treat chemotherapy-induced neutropenia (CIN) caused by multiple chemotherapies. The details, in Cancer Chemotherapy and Pharmacology, show Plinabulin has positive effects on bone marrow cells, with a mechanism distinct from G-CSF-based therapies, the current standard of care. The paper, titled, “Plinabulin ameliorates neutropenia induced by multiple chemotherapies through a mechanism distinct from G‑CSF therapies,” reports on Plinabulin’s ability to reduce neutropenia induced by docetaxel, cyclophosphamide or doxorubicin chemotherapy, without affecting bone marrow or blood G-CSF levels. The results support Plinabulin’s clinical testing as a non-G-CSF-based treatment for CIN associated with chemotherapies of different mechanisms.
Catalyst Biosciences Inc., of South San Francisco., Calif., entered a global license and collaboration deal with Biogen Inc. to develop and commercialize pegylated CB-2782 (CB-2782-PEG) for treating geographic atrophy associated dry age-related macular degeneration (dry AMD). Biogen receives the exclusive worldwide license to develop and commercialize CB-2782-PEG and Catalyst’s other anti-C3 proteases for treating dry AMD. Catalyst will perform preclinical and manufacturing activities and Biogen is solely responsible for funding preclinical and manufacturing activities and performing IND-enabling activities, worldwide clinical development, and commercialization. Catalyst receives $15 million up front and could receive up to $340 million in clinical, regulatory, and commercial milestone payments plus future tiered royalties based on net sales.
Cellgenix GmbH, of Freiburg, Germany, launched Cellgenix GMP rh TGF-ß1 for cell and gene therapy applications based on CAP(R)Go technology developed by Cevec Pharmaceuticals GmbH, of Cologne, Germany. The product allows companies to switch from the preclinical rh TGF-ß1 to the GMP version as they move from preclinical to clinical development.
Circassia Pharmaceuticals plc, of Oxford, U.K., received a notice from its partner Beyond Air Inc., of Garden City, N.Y., that it's terminating Circassia's licensing agreement for LungFit PH in the USA and China based on material breach. Circassia refutes the allegations and plans to enforce its rights under the agreement.
Contrafect Corp., of Yonkers, N.Y., selected its next product candidate, CF-370, an engineered lysin for treatment of infections by Pseudomonas aeruginosa. In rabbit pneumonia models with multidrug-resistant P. aeruginosa, rabbits treated with CF-370 alone had longer survival, compared to vehicle control. The drug also reduced bacterial burden in lung, kidney and spleen with greater reductions seen in combination with meropenem.
Dicerna Pharmaceuticals Inc., of Lexington, Mass., and Roche Holding AG, of Basel, Switzerland, closed their licensing deal to develop treatments for chronic hepatitis B virus infection using Dicerna’s proprietary GalXC RNAi platform technology.
Evotec SE, of Hamburg, Germany, achieved the third milestone under its beta cell replacement therapy research alliance with Paris-based Sanofi SA, triggering a €3 million ($3.34 million) payment to Evotec.
Exelixis Inc., of Alameda, Calif., and Roche Holdings AG, of Basel, Switzerland, are collaborating to test Exelixis' tyrosine kinase inhibitor Cabometyx (cabozantinib) with Roche's PD-L1 immune checkpoint inhibitor Tecentriq (atezolizumab). The companies will co-fund three phase III studies in patients with advanced non-small-cell lung cancer, castration-resistant prostate cancer and renal cell carcinoma.
Generex Biotechnology Corp., of Miramar, Fla., said that at least one beneficiary of the Veneto asset acquisition violated the leak out provisions in the Veneto asset purchase agreement, which it believes caused irregular trading activities. Generex hasn't received certain assets from the acquisition that it paid for and is considering cost efficient solutions to these violations.
Genmab A/S, of Copenhagen, and Curevac AG, of Tubingen, Germany, are collaborating to develop mRNA-based antibody products using Curevac’s mRNA technology and Genmab’s antibody technologies. Genmab will pay Curevac $10 million up front and make a €20 million (US$22.25 million) equity investment in the company. Curevac will cover a portion of the development costs up to the IND application at which point Genmab will take over development and commercialization. Curevac is eligible for undisclosed milestone payments and tiered royalties. The partnership includes an option for Genmab to license up to three additional products using Curevac's mRNA technology, which would be funded by Genmab with Curevac eligible for development, regulatory and commercial milestone payments between $275 million and $368 million for each product, as well as tiered royalties in the range from mid-single digits up to low double digits on each product.
Health Sciences Acquisitions Corp., of New York, closed its acquisition of Immunovant Sciences Ltd., and changed its name to Immunovant Inc. The company will be focused on developing IMVT-1401, a monoclonal antibody targeting the neonatal Fc receptor, for the treatment of Graves’ ophthalmopathy, myasthenia gravis and warm autoimmune hemolytic anemia. On Dec. 19, 2019, shares of the company began trading on the Nasdaq under their new ticker symbol IMVT.
Medison Pharma Ltd., of Petach Tikva, Israel, received exclusive rights from Bridgebio Pharma Inc., of Boston, to distribute, market, sell and commercialize fosdenopterin (BBP-870/ORGN001) in Israel.
Fosdenopterin is a cPMP replacement therapy for treating patients with molybdenum cofactor deficiency type A. A rolling NDA submission with the FDA is underway. Pending approval, Medison is responsible for regulatory approval and commercializing fosdenopterin in Israel. Bridgebio is a subsidiary of Origin Biosciences Inc., which receives an up-front cash payment and will receive milestone payments after achieving certain milestone events and ongoing royalties on net sales of fosdenopterin in Israel. No financial details were released.
Oxurion NV, of Leuven, Belgium, said its pipeline will focus on clinical development of THR-149 and THR-687 as potential VEGF independent treatments for diabetic macular edema (DME). THR-149 is a plasma kallikrein inhibitor while THR-687 is a pan-RGD integrin inhibitor, both of which have the potential to treat a broad range of patients with diabetic eye disease. Commercialization of Jetrea, (ocriplasmin) – FDA-approved for symptomatic vitreomacular adhesion and vitreomacular traction – will be shifted to distributors and licensees in marketed territories, patient access to be maintained. The company said the move is designed to “… simplify our organizational structure, allowing us to focus our resources on what we believe are two of the most exciting potential new therapies for diabetic eye disease currently in the clinic.”
Onconova Therapeutics Inc., of Newtown, Pa., licensed rights to rigosertib, which targets the RAS effector pathways, in Australia and New Zealand to Specialised Therapeutics Asia Pte Ltd., of Singapore. Onconova is eligible for clinical, regulatory and sales-based milestone payments up to $30.4 million and tiered double-digit royalties on net sales.
Paratek Pharmaceuticals Inc., of Boston, was awarded a five-year contract from the Department of Health and Human Services, Office of the Assistant Secretary for Preparedness and Response and Biomedical Advanced Research and Development Authority to support the development, FDA approval and post-marketing requirements for Nuzyra (omadacycline) for the treatment of pulmonary anthrax. Under the contract, which is valued at up to $285 million and has an option to be extended to 10 years, the government has the option to buy up to 10,000 treatment courses of Nuzyra for the strategic national stockpile.
Protokinetix Inc., of Marietta, Ohio, reported preclinical data on its glycopeptide PKX-001 in a rat model of genetic retinal degeneration using transplanted photoreceptor precursor cells (PPCs). PPCs pretreated with PKX-001 had improved visual behavioral, measured with an optokinetic tracking test, and function, measured with an electroretinogram test, compared with PPCs without pretreatment. Pretreatment also resulted in improved survival at three months, 4.5 months and six months after transplant.
SK Holdings Co. Ltd., of Seoul, South Korea, met with its partner Astrazeneca plc, of Cambridge, U.K., to discuss plans to expand cooperation between the two companies. SK Holdings currently manufactures active pharmaceutical ingredients for Astrazeneca through a partnership worth approximately $100 million per year.
Ultragenyx Pharmaceutical Inc., of Novato, Calif., sold royalty rights that it's due from Tokyo-based Kyowa Kirin Co. Ltd. on the net sales of Crysvita (burosumab) in the EU, U.K. and Switzerland to Royalty Pharma. Ultragenyx will receive $320 million up front and the royalty rights will revert to the company when the aggregate payments are equal to or greater than 1.9 times the purchase price prior to Dec. 31, 2030, or 2.5 times the purchase price if the threshold is not met by the end of 2030.
Zafgen Inc., of Boston, and Chondrial Therapeutics Inc., of Bala Cynwyd, Pa., announced plans to merge. The resulting company, which will be called Larimar Therapeutics Inc., will develop Chondrial’s lead asset, CTI-1601, for the treatment of Friedreich’s ataxia. After the merger, Chondrial stockholders are expected to own approximately 60% of the combined company with Zafgen stockholders owning the rest, subject to the amount of cash Zafgen has at closing and other potential adjustments.