Acucela Inc., of Seattle, and Youhealth Eyetech Inc., of San Diego, have agreed on an exclusive option from Youhealth for lanosterol technology licensed from the Regents of the University of California, San Diego, for the non-surgical pharmacologic treatment of cataracts. Lanosterol, a naturally occurring compound found in the human body, has shown promise for this condition in preclinical studies. It has been identified as a key molecule in the prevention of lens protein aggregation and lens opacification. Acucela will evaluate lanosterol as a treatment for mild cataracts with an option to fully develop and commercialize the compound globally with the exception of Greater China including China, Taiwan and Hong Kong. The use of lanosterol in other ophthalmic conditions such as presbyopia may be investigated by Acucela in the future. Youhealth’s parent company, Guangzhou Kang Rui Biological Pharmaceutical Technology Co. Ltd., of Guangzhou, China, will be responsible for lanosterol’s clinical development and commercialization in Greater China including China, Taiwan and Hong Kong.
Biotron Ltd., of Sydney, disclosed positive outcomes from its phase II study of its first-in-class antiviral drug BIT225. The trial was designed to assess the safety and antiviral activity of three months of dosing with BIT225 in patients infected with hepatitis C virus (HCV). The study also provided key information on a new capsule form of BIT225, information that is critical for determining dosage in further studies with the drug, the company said. Key outcomes from analysis of the 30-subject HCV genotype 1 (G1) cohort found BIT225 was safe and well-tolerated with none of the HCV G1 patients withdrawing due to BIT225-related adverse events. HCV G1 patients treated with BIT225 and interferon/ribavirin are significantly more likely to clear virus within 24 weeks of commencing treatment than those treated with the combo alone. Twelve weeks after stopping BIT225 treatment, 82 percent of HCV G1 patients treated with BIT225 plus interferon/ribavirin were clear of virus, compared to 60 percent of those treated with only interferon/ribavirin. BIT225 is a small-molecule viral assembly inhibitor that targets p7.
Bone Medical Ltd., of Perth, Australia, is acquiring Botanix Pharmaceuticals Inc., of Philadelphia. As part of the transaction, Bone Medical will undertake a consolidation of its capital on a one-for-three and one-third basis and plans to raise $3 million through the issue of 150 million shares at 2 cents each. Botanix’s lead product, BTX1503, is a topically applied gel for acne. Upon completion of the planned offer, Botanix will be funded to complete initial clinical studies over the next 12 months including a pilot study on patients, through to commencement of a planned phase II efficacy study in the U.S.
Celltrion Inc., of Incheon, South Korea, reported data from real-world studies involving nearly 600 inflammatory disease patients, showing comparable efficacy and safety following a switch to biosimilar infliximab from the originator drug, branded Remicade (Johnson & Johnson). Those data were reported at the Congress of European Crohn’s and Colitis Organisation meeting in Amsterdam. Celltrion markets Remsima, a biosimilar of infliximab that has gained approval in a number of countries, though still awaits a decision in the U.S.
Researchers from the Japanese Chiba University and the University of California at Davis have identified an enzyme that could be a target for antidepressant drug development. The enzyme, soluble epoxide hydrolase (sEH), plays a role in pro-inflammatory signaling and is implicated in the onset of anorexia nervosa, but its role in depression had not been studied. In their work, the authors found that the sEH inhibitor TPPU had antidepressant effects in inflammation-induced depression models in mice. Moreover, sEH knockout mice were resilient to stress-induced depression, while postmortem analysis of she levels in the brains of patients with depression (but also with other neuropsychiatric disorders) was elevated. The findings appeared in the March 14, 2016, online issue of the Proceedings of the National Academy of Sciences.
Huawei, of Shenzhen, China, an information and communications technology firm, and Wuxi Apptec, of Shanghai, said they signed a framework agreement to support the China Precision Medicine Initiative through the creation of a precision medicine cloud platform.
Contravir Pharmaceuticals Inc., of Edison, N.J., started screening healthy volunteers for enrollment into the phase I portion of the company’s phase I/IIa multiple ascending dose clinical study of CMX157, a lipid conjugate of tenofovir for treating hepatitis B virus infection. The phase I portion of the study will enroll 50 healthy volunteers assigned to one of five sequential, ascending CMX157 dosing cohorts. Participants will receive two weeks of a once-daily dose of either 5 mg, 10 mg, 25 mg, 50 mg or 100 mg of CMX157 or placebo. An independent review will be conducted by a data safety and monitoring board between the 25 mg and 50 mg cohorts. Following the review. Contravir plans to start the phase IIa portion of the study in parallel with the continuing phase I portion. The phase IIa will enroll 60 treatment-naïve patients with chronic HBV infection to compare CMX157 to tenofovir DF (marketed by Gilead Sciences Inc. as Viread). It will also feature a sequential dose escalation period, with 10 patients per cohort receiving four weeks of a once-daily dose of either 5 mg, 10 mg, 25 mg, 50 mg or 100 mg of CMX157, and two patients per cohort receiving 300 mg of Viread, the standard dose of Viread. In order to facilitate enrollment, the study is being conducted by several leading academic medical centers in Thailand, a country in which the incidence of HBV is very high. Contravir believes that CMX157 may provide better efficacy and improved safety at a lower dose than Viread.
Shanghai-based Hutchison China Meditech Ltd. subsidiary Hutchison Medipharma Ltd. has started SANET-p, a phase III registration trial of sulfatinib (HMPL-012) in China in patients with pancreatic neuroendocrine tumors. The first patient was dosed on March 18. The randomized, double-blind, placebo-controlled registration study will treat patients with low or intermediate grade advanced NET whose disease has progressed, locally advanced or distant metastasized and those for whom there is no effective therapy. Patients are being randomized at a 2:1 ratio to receive either 300 mg of sulfatinib orally once per day, or placebo, on an every 28-day treatment cycle. The primary objective of the study is to evaluate the progression-free survival of sulfatinib as compared to that of placebo, with secondary endpoints including objective response rate, disease control rate, time to response, duration of response, overall survival, safety and tolerability. About 195 patients are expected to be enrolled in the SANET-p study from more than 20 centers across China, with top-line results expected in 2018. (See BioWorld Today, Dec. 23, 2015.)
Hutchison China Meditech Ltd., of Shanghai, raised $101 million from its IPO of 7.5 million American depositary shares at $13.50 each. It listed shares on Nasdaq under the symbol HCM. The company has a multipronged business that includes 19 active studies for seven clinical-stage therapies for oncology and immunological diseases, licensing deals with big pharma and diversified commercial operations for prescription and OTC drugs. The company’s shares closed Thursday down 10 cents at $13.40. (See BioWorld Today, Feb. 17, 2016.)
Huya Bioscience International LLC, of San Diego, has signed an exclusive license with Fudan University for the ex-China rights to a series of novel immuno-oncology drug candidates discovered by Fudan researchers. The agreement includes a panel of inhibitors of indoleamine 2,3-dioxygenase. The IDO pathway is one of several immune response checkpoints thought to play an important role in suppressing T-cell function, allowing tumors to avoid immune surveillance. Huya plans to advance the products into clinical testing in parallel with the ongoing development of HBI-8000, the histone deacetylase inhibitor it recently licensed to Tokyo-based Eisai Co. Ltd. (See BioWorld Today, Feb. 3, 2016.)
Medivate Partners, of Seoul, South Korea, said it closed a $12 million investment in Sillajen, an oncolytic immunotherapeutic firm, which plans to use the capital to complete an on-going phase III study of its lead product, Pexa-Vec (JX-594), the first engineered oncolytic virus with demonstrated intravenous delivery to tumors and induction of cancer-targeting antibodies.
Mesoblast Ltd., of Melbourne, Australia, said it exclusively licensed technology developed at Harvard Medical School that can modify mesenchymal lineage adult stem cells (MLCs) to enhance their natural homing properties to sites of excessive inflammation. Called ex vivo fucosylation, that technology has modified MLCs capable of inducing durable reversal of type 1 diabetes in a preclinical study, the company reported. Results showed the cell targeting technology increased by threefold the number of MLCs reaching the inflamed pancreas in autoimmune diabetic mice following intravenous infusion, compared with unmodified MLCs.
Novogen Ltd., of Sydney, said it received a cash refund of $2.8 million from the Australian Taxation Office under the R&D Tax Incentive Program for the financial year ending June 30, 2015. Novogen said the funding will support advancing Cantrixil (TRXE-002-1), its lead superbenzopyran candidate, into phase I testing this year.
Orexigen Therapeutics Inc., of San Diego, and Takeda Pharmaceuticals USA Inc., a wholly owned subsidiary of Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, agreed to terminate their amended and restated collaboration agreement for the weight management drug, Contrave (naltrexone HCl/bupropion HCl), inked in August 2015 after a fallout over Orexigen’s decision to terminate the drug’s cardiovascular outcomes trial. The transaction is subject to receipt of clearance under the Hart-Scott-Rodino Antitrust Improvement Act. Takeda agreed to continue commercialization of Contrave in the U.S. during a 180-day transition period and said the transaction is not expected to change its consolidated results forecast for fiscal year 2015. Separately, Orexigen said it signed an agreement with Valeant Pharmaceuticals International Inc., of Laval, Quebec, to commercialize the drug, branded Mysimba outside the U.S., in Central and Eastern Europe. The distribution agreement between Valeant Holdings Ireland and Orexigen’s wholly owned subsidiary, Orexigen Therapeutics Ireland Ltd., included 12 EU countries where Mysimba was approved for marketing and extends to several non-EU countries where Valeant will apply for marketing authorization. Orexigen expects Valeant to launch the product in the EU countries in the second half of 2016. Orexigen will retain regulatory affairs responsibilities in the EU countries and will supply Mysimba tablets to Valeant at an agreed transfer price. Other terms were not disclosed. (See BioWorld Today, May 14, 2015, and Aug. 7, 2015.)
Propanc Health Group Corp., of Melbourne, Australia, said it received $1.2 million from an institutional investor to fast track development of proenzyme related oncology products into clinical trials initially for pancreatic and ovarian cancers, followed by colorectal cancer. Key activities anticipated include completing a 28-day animal safety toxicology study, development of bioanalytical methods for animals and humans to assess movement of the drug within the body, and preparation of GMP manufacture of lead product PRP for patient trials.
Rockwell Medical Inc., of Wixom, Mich., said its Triferic (soluble ferric pyrophosphate) submission to the CFDA was accepted. Earlier this year, Rockwell signed exclusive licensing and manufacturing supply agreements with Wanbang Biopharmaceutical Co. Ltd., a subsidiary of Shanghai Fosun Pharmaceutical Group Co. Ltd., of Shanghai, for the rights to commercialize the iron replacement and hemoglobin maintenance drug, Triferic – approved by the FDA last year – and the vitamin D injection, Calcitriol, to treat end stage renal disease patients. Wanbang also gained the exclusive right to develop and commercialize Triferic for new therapeutic indications for the Chinese market. Wanbang is responsible for clinical, regulatory and marketing expenses for Triferic and Calcitriol in China as well as development and regulatory costs for new Triferic indications. (See BioWorld Today, Jan. 27, 2015.)
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, and Frazier Healthcare Partners, of Menlo Park, Calif., formed Outpost Medicine, a biopharmaceutical company focused on the development of new treatments of urologic and gynecologic diseases and disorders. Takeda granted an exclusive license to Outpost for the worldwide development and commercialization rights to OP-233 (formerly TAK-233), a clinical-stage product candidate being studied for the treatment of stress urinary incontinence. In connection with the license, Outpost completed a $41 million financing led by Frazier Healthcare Partners and including Adams Street Partners, Novo A/S, and Vivo Capital. Deal terms were not disclosed. In other news, Takeda said interim findings from the GEMINI long-term safety study were presented during the 2016 European Crohn’s and Colitis Organization Annual Scientific Meeting in Amsterdam, The Netherlands. The data showed that patients with moderately to severely active ulcerative colitis reported clinical improvements with about three years of treatment with vedolizumab. The drug is a humanized monoclonal antibody that is designed to specifically antagonize the alpha4beta7 integrin, inhibiting the binding of alpha4beta7 to intestinal mucosal address in cell adhesion molecule 1 and fibronectin, but not vascular cell adhesion molecule 1.
Theratechnologies Inc., of Montreal, and Taimed Biologics Inc., of Taipei, Taiwan, said they inked a 12-year collaboration to market and distribute ibalizumab, a CD4-directed HIV entry inhibitor, in the U.S. and Canada. Under the terms, Theratechnologies will make a $2 million payment, including $1 million in cash at signing and $1 million through the issuance of 957,169 common shares. A further $8.5 million will become due at commercial launch, paid as $2 million in common shares, with price to be determined upon FDA approval, with $1 million in common shares at a price to be determined upon commercial launch. The remaining $5.5 million will be payable in quarterly installments based on a predetermined percentage of net sales during that quarter. Once sales have reached an aggregate amount of $20 million over four consecutive quarters, Theratechnologies will make a $7 million milestone payment. It will make additional sales-related milestones, in the form of a $10 million payment once annual sales of ibalizumab reach $200 million; $40 million when annual sales reach $500 million and $100 million upon annual sales reaching $1 billion. Taimed also is entitled to development milestones. The monoclonal antibody currently is in phase III testing, with more than two-thirds of the patients required for the 24-week trial enrolled to date. Pending positive data, ibalizumab will be evaluated under the FDA’s priority review process, which is expected to be completed within six months of the application, or during the first half of 2017.
Valneva SE, of Lyon, France, said it has signed a $42 million contract with the U.S. Department of Defense for the supply of its Japanese encephalitis vaccine Ixiaro. Under the terms of the agreement, Valneva will supply Ixiaro doses to the Defense Logistics Agency over a two-year period, including a base year and an option year. First deliveries are expected to commence in the coming weeks, said Valneva.
Viralytics Ltd., of Sydney, reported positive data from the CANON1 clinical trial of its lead drug candidate, Cavatak, at the 31st Annual European Association of Urology (EAU) Congress held in Munich, Germany. The phase I study is expected to enroll approximately 25 non-muscle invasive bladder cancer (NMIBC) patients. In the first stage of the trial, nine patients were treated by intravesicular administration of monotherapy Cavatak. In the second stage, to date, five patients have received a subtherapeutic dose of the chemotherapy, mitomycin C, plus Cavatak intravesically prior to routine surgical removal of the tumor tissue. Some anti-cancer activity, including viral induced tumor inflammation, has been demonstrated in both the monotherapy and combination therapy arms of the study. A complete response has been observed in one out of the three patients in the highest dose cohort of the monotherapy.
Zambon SpA, of Milan, Italy, concluded a strategic agreement with US Worldmeds for the U.S. commercialization of Xadago (safinamide) to treat Parkinson’s disease. In 2012, Zambon gained global marketing rights to safinamide outside Asia and Japan from developer Newron Pharmaceuticals SpA, also of Milan. Zambon is commercializing safinamide, branded Xadago, in the EU and Switzerland, following approval by regulatory authorities last year. The new drug application for safinamide is under review by the FDA with a PDUFA date of March 29. Under terms of the U.S. sublicense, US Worldmeds agreed to pay Zambon undisclosed up-front, regulatory and commercial milestone payments and royalties on product sales. Newron also is entitled to a milestone payment upon FDA approval and a share of up-front, milestone and royalty payments made by US Worldmeds. Zambon said US Worldmeds plans to dedicate more than 60 sales representatives to the U.S. launch of Xadago. (See BioWorld Today, April 6, 2012, and Dec. 30, 2014.)