On February 6, 2017 Aura Biosciences, a biotechnology company developing a new class of therapies to target and selectively destroy cancer cells using viral nanoparticle conjugates, reported that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug application (IND) for the company’s lead program, light-activated AU-011 in ocular melanoma (OM) (Press release, Aura Biosciences, FEB 6, 2017, View Source [SID1234517651]). This active IND enables Aura to begin initial clinical testing of AU-011, a unique targeted therapy that could transform the primary treatment of patients with OM, a rare and life-threatening disease.

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"Early detection of ocular melanoma, combined with the administration of AU-011 as a potential vision-sparing therapy, could transform the treatment of patients with this devastating disease," said Brian Marr, M.D., Director of the Ophthalmic Oncology Service at Columbia University Medical Center. Dr. Marr is the principal investigator for the AU-011 clinical trial and also is a member of Aura’s Clinical Advisory Board.

"Receiving IND clearance to enter the clinic for AU-011 is an important step in the development pathway for this novel class of drugs, and I’m thankful to our team of dedicated employees, as well as to our distinguished scientific and clinical advisors, for their contributions that have propelled us to this point," said Elisabet de los Pinos, Ph.D., founder and CEO of Aura. "With the advancement of AU-011, we are opening the door for innovation in a completely new therapeutic area where there are no FDA drugs approved today. Our hope is that AU-011 could be used to treat small primary melanomas early, with the potential to eliminate the tumor and preserve vision for patients."

The Phase 1b open-label, single ascending dose clinical trial currently enrolling is designed to evaluate the safety, immunogenicity and preliminary efficacy of two dose levels of AU-011 for the treatment of small-to-medium primary OM. Screening procedures for eligible patients are underway at five clinical trial sites across the country. For more information, visit www.clinicaltrials.gov or contact [email protected].
About ocular melanoma (OM)
Ocular melanoma (OM), also known as uveal or choroidal melanoma, develops in the uvea, or uveal tract, of the eye, and is an aggressive and rare eye cancer. No targeted therapies are currently available, and current treatments are associated with serious morbidities. The most common treatment today is placing an invasive radioactive plaque against the exterior of the eye near the tumor, which requires multiple surgeries and can lead to cataracts, retinopathy and loss of vision. The alternative is enucleation, the removal of the eye. OM metastasizes to the liver in about half of all cases (source: OMF), and only 15 percent of patients whose OM has metastasized survive beyond five years after diagnosis (source: ACS).

About light-activated AU-011
AU-011 is a first-in-class targeted therapy in development for the primary treatment of ocular melanoma (OM), also known as uveal or choroidal melanoma, a rare and life-threatening disease. The therapy consists of viral nanoparticle conjugates that bind selectively to cancer cells in the eye. AU-011 has a necrotic mechanism of action and is administered through an intravitreal injection into the eye. Upon activation with an ophthalmic laser, the drug rapidly and specifically destroys the membranes of tumor cells while sparing key eye structures, which may allow for the potential of preserving patients’ vision. AU-011 for OM has been granted orphan drug designation by the U.S. Food and Drug Administration and is currently in clinical testing.

On February 5, 2017 Abbisko Therapeutics, a biopharmaceutical company dedicated to discover and develop innovative medicines, reported the completion of a $28 million Series A financing from a leading group of healthcare investors, including Lilly Asia Ventures, Sinopharm Capital, Jianxin Capital, and TF Capital (Press release, Abbisko Therapeutics, FEB 5, 2017, View Source;article_id=43 [SID1234521011]).

The proceeds will be used to support Abbisko Therapeutics’ drug research and development effort, including the creation of a state-of-the-art R&D center and advancing a drug discovery pipeline with multiple programs in oncology and other disease areas.

"We are fortunate to have a number of the world’s premier healthcare investors participated in this round of financing." said Dr. Yao-chang Xu, founder and CEO of Abbisko. "We have a new but highly experienced drug research and development team. Investors agree with our strategy and philosophy, providing strong financing support to enable our quick start. We will seize the opportunities to become a critical force and leading company in the discovery of novel medicines in China and the world. We hope to bring revolutionary therapeutic solutions to patient as soon as possible".

"At present, China’s innovative drug discovery is in a golden period of time with vigorous development and progress. In a very rapid fashion, Abbisko has built up a top notch R&D team and established a strong pipeline." said Ai’min Wu, Partner and President of Sinopharm Capital. "Sinopharm Capital fully recognizes Abbisko’s expertise and strategy and believes in that, under the leadership of Dr. Yao-chang Xu and the management team, Abbisko has a great potential to become one of the leading biopharmaceutical companies in China and in the industry. Sinopharm Capital is pleased to participate the creation and development of Abbisko Therapeutics and will continue to dedicate in helping domestic biomedical companies to be competitive with international leading players.

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On February 5, 2017 Abbisko Therapeutics Co., Ltd. reported the successful completion of its series A round financing at $28 million (Press release, Abbisko Therapeutics, FEB 5, 2017, View Source;article_id=70 [SID1234556289]). This round was led by Lilly Asia Ventures and followed by Sinopharm Capital, Jianxin Capital, and TF Capital.

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The fund raised in this round will be used to support new drug research of Abbisko Therapeutics Co., Ltd. (hereinafter referred to as Abbisko), including setting up a new drug R&D center, building an innovative drug R&D team and establishing antitumor drug R&D pipelines.

Abbisko is a new innovative biopharmaceutical company established in Zhangjiang of Shanghai. The company’s founding members previously held significant R&D leadership and management positions in several Fortune 500 pharmaceutical companies (Novartis, Johnson & Johnson, Merck, Eli Lilly and AbbVie) and China’s leading pharmaceutical companies (Hengrui and Hansoh). They have extensive project leadership and team management experience in novel drug development and clinical trials. Abbisko will focus on unmet needs of patients especially those of Chinese patients, follow international standards for new drug development, and dedicate itself to the research and development of innovative therapeutics with global intellectual property rights.

Dr. Yao-chang Xu, Abbisko’s founder, board chairman and CEO, commented on this round of financing: "We are gratified that several of the world’s leading investment institutions participated in this very important round of financing. We are a new but highly experienced drug development team. The investors agree with our strategy and philosophy. As a result, our team can set sail quickly and seize the opportunities brought by the rapid development of biotech sector in China. We’ll work hard to bring more revolutionary treatment solutions to suffering patients as soon as possible."

Mr. Aimin Wu, Sinopharm Capital’s president and partner, said: "At present, China’s innovative drug R&D are in a golden age of development. Abbisko has established a top-notch R&D team with a fast growing cutting-edge product pipelines. Sinopharm fully recognize and appreciates Abbisko’s R&D strength and supports its direction. Led by Dr. Yao-chang Xu, Abbisko is capable of becoming a super star company in the biomedical sector. Sinopharm is honored to participate in the establishment and development of Abbisko, helping domestic biomedical companies to compete with international innovative enterprises."

With Abbisko’s successful establishment and completion of this round of investment, an important new force has risen for China’s innovative drug R&D.

On February 6, 2017 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH) reported top-line results from the randomized, double-blind, placebo-controlled Phase II trial testing the efficacy of lirilumab as a single agent maintenance treatment in elderly patients with acute myeloid leukemia (AML) in first complete remission ("EffiKIR" trial) (Press release, Innate Pharma, FEB 4, 2017, View Source [SID1234517640]). The study did not meet its primary efficacy endpoint of leukemia-free survival (LFS).

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Two arms of the trial tested single agent lirilumab at different doses and treatment intervals (0.1 mg/kg q3months or 1 mg/kg q1month) whereas in the third arm, patients received placebo. There was no statistically significant difference between either lirilumab arms and the placebo arm on the LFS nor on other efficacy endpoints. As announced in March 2015, one arm of the trial was discontinued upon DSMB recommendation. This arm has now been identified as the 1 mg/kg q1month arm of the trial. At the time of its decision, the DSMB assessed that the objective of achieving a superior LFS in this arm compared to placebo could not be reached. There was no concern with tolerance.

The adverse events encountered with lirilumab were consistent with the previously reported safety profile of lirilumab.

Data analyses are ongoing and the full trial data will be submitted to a future medical conference and for publication.

Lirilumab is being investigated in six trials sponsored by Bristol-Myers Squibb, across a range of solid and hematological cancer indications in combination with other agents, including nivolumab (see on clinicaltrials.gov).

Pierre Dodion, Chief Medical Officer of Innate Pharma, said: "Although we knew that this setting was challenging, we are disappointed by the results of the EffiKIR study and will investigate further to better understand the data in its entirety. However, Effikir is only one of seven studies currently investigating lirilumab. Lirilumab is tested in a broad and comprehensive combination program in multiple indications and we saw encouraging early efficacy signals of lirilumab in combination with nivolumab at the 2016 SITC (Free SITC Whitepaper) meeting. We are looking forward to the next data sets as well as the next steps for the program in 2017."

On February 2, 2017 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel oncology therapeutics, reported an update on its ongoing pivotal Phase 2 trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN), using Stemline’s experimental compound, SL-401. BPDCN at present has no approved treatment (Press release, Stemline Therapeutics, FEB 2, 2017, View Source [SID1234517627]).

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On January 18, the Company received a report that a patient death had occurred. The patient had developed capillary leak syndrome (CLS), a known, sometimes fatal, and well-documented side effect of SL-401. The cause of the patient’s death has not yet been determined. The safety profile for SL-401 includes CLS, and there have been previous deaths reported in patients with CLS in this trial, which have been disclosed in public presentations. That CLS is an expected complication of the administration of SL-401 has also been identified in filings with the Securities and Exchange Commission (SEC) and U.S. Food and Drug Administration (FDA), as well as in the study’s informed consent forms and other information provided to investigators.

As with all study events, the Company has and will continue to report the data to the FDA in accordance with the study protocol and applicable regulations. Stemline plans to provide a clinical and safety update on this cohort when the cohort and data are complete. The pivotal Phase 2 trial with SL-401 in BPDCN is currently ongoing, patient enrollment is ahead of schedule, and patients continue to receive SL-401 in the trial. Our timelines for study completion and BLA submission remain on trac