Emulate, AstraZeneca Collaborate on Organs-on-Chips Technology

Privately-held Emulate, Inc. struck a deal with pharma giant AstraZeneca’s Innovative Medicines and Early Development (IMED) Biotech Unit to embed its Organs-on-Chips technology within the laboratories of the IMED Drug Safety organization.

In an announcement this morning, Boston-based Emulate said the agreement with AstraZeneca will accelerate the development of Organs-on-Chips technology and testing within the context of a pharmaceutical organization. AstraZeneca and Emulate have been collaborating on the Organs-on-Chips technology since 2013. The two companies published some of their recent work during the Society of Toxicology meeting in March 2018. AstraZeneca is the first pharmaceutical company to work with Emulate to develop and embed its Organ-on-Chips technology in its laboratories.

Emulate’s organ chips are about the size of a AA battery and contain hollow channels “lined with tens of thousands of living human cells and tissues.” An organ-chip is a living, micro-engineered environment that recreates the natural physiology and mechanical forces that cells experience within the human body, according to data from Emulate. The Organs-on-Chips technology comprises of the Organ-Chips, instrumentation, and software apps. In the future, the system is also expected to reduce the use of animals in research as well as the cost and time of discovery and development.

Mene Pangalos, head of AstraZeneca’s IMED Biotech Unit, said the Organs-on-Chips technology has the “potential to enhance and accelerate our ability to translate science into innovative medicines for patients.”

“Working side by side with Emulate scientists will enable us to better develop the platform and may improve our ability to predict adverse and non-adverse effects in humans. The partnership exemplifies how we are creating permeable research environments where our scientists work together to foster collaborative scientific advancement,” Pangalos said in a statement.

Geraldine Hamilton, president and chief scientific officer of Emulate, said the partnership with the AstraZeneca business unit is an example of how the company can progress toward its goal of “increasing the success of drug discovery and development by providing a platform that recreates human-relevant biology.” Hamilton added that the company is establishing a model of his its Organ-on-Chips technology can be integrated into the pharma labs and existing workflows of the industry.

“The research conducted with AstraZeneca will allow us to further develop and add greater functionality to our technology platform,” Hamilton said in a statement.

The initial focus of the collaboration between the two companies will be to use Emulate’s Liver-Chip for safety testing of drug candidates across the AstraZeneca pipeline. The goal will be to submit Organ-Chip data within the regulatory framework for new drugs. Emulate said the terms of the agreement with AstraZeneca will allow for the technology to be adopted across AstraZeneca’s therapeutic areas and will enable the two companies to develop functionality of three other Emulate Organ-Chips — the Lung Tumor-Chip, Lung-Chip, and Glomerulus Kidney-Chip.

Last year Emulate and the U.S. Food and Drug Administration entered into a Cooperative Research and Development Agreement to “evaluate and qualify” Emulate’s organ-on-a-chip technology. The company and the regulatory agency will determine if the technology is a strong enough platform for toxicology testing to meet regulatory evaluation criteria for new products. The products first on the block will include foods, dietary supplements and cosmetics, the company announced this morning. The studies will use Emulate’s Human Emulation System, which includes Organ-Chips, instrumentation and software apps.

https://bit.ly/2KrL09N

Alkermes upped to buy by Citi

Alkermes upgraded to Buy from Neutral at Citi. Citi analyst Liav Abraham upgraded Alkermes to Buy with an unchanged price target of $62. The stock closed yesterday down 80c to $46.24. Following a “deep dive” and physician survey, the analyst has increased conviction around the ALKS 3831 value proposition. ALKS 3831 is a once-daily, oral atypical antipsychotic drug candidate for the treatment of schizophrenia. Abraham views Alkermes’ valuation as compelling and is positive on the company’s “pipeline optionality and near-term catalyst flow.”

https://bit.ly/2k0VuSd

Zoetis to acquire Abaxis for $2B

Zoetis Inc. (ZTS) and Abaxis, Inc. (ABAX) announced a definitive merger agreement pursuant to which Zoetis will acquire Abaxis, a leader in the development, manufacture and marketing of diagnostic instruments for veterinary point-of-care services for $83 per share in cash, or approximately $2B in aggregate. The acquisition is expected to enhance Zoetis’ presence in veterinary diagnostics, a category of the animal health industry with approximately 10% compound annual growth over the last three years. “This acquisition brings Zoetis a company that has a proven, competitive diagnostic platform for growth that we can help to accelerate in the U.S. and worldwide with our global scale and direct customer relationships in approximately 45 countries,” said Juan Ramon Alaix, CEO of Zoetis. “Together we can bring more veterinarian customers a broader range of products that fit into our comprehensive solutions and innovations, from prediction and early detection of disease in animals to prevention and treatment. We are very excited by the passion for customers that Abaxis and Zoetis colleagues share.” The transaction is subject to customary closing conditions, including regulatory approvals and the approval of Abaxis shareholders. Zoetis expects to complete the acquisition before the end of 2018, and it intends to fund the purchase through a combination of existing cash and new debt. Zoetis anticipates the transaction will have an impact on 2018 reported earnings related to customary closing activities; however, on an adjusted basis, the company does not expect a material impact. Zoetis will provide additional updates as the transaction progresses towards closing. Zoetis expects the transaction to be accretive to the company’s earnings in 2019.

https://bit.ly/2jZ85We

Fate, Sloan Kettering expand license pact

Fate Therapeutics announced the company has gained access to additional intellectual property from Memorial Sloan Kettering Cancer Center that enables the development of gene-edited T-cell immunotherapies. The newly-licensed portfolio of intellectual property covers new chimeric antigen receptor constructs as well as off-the-shelf CAR T cells, including the use of CRISPR and other innovative technologies for their production. Fate Therapeutics is utilizing gene editing under its ongoing collaboration for the research and development of off-the-shelf CAR T-cell immunotherapies with Michel Sadelain, M.D., Ph.D., Director of the Center for Cell Engineering and the Stephen and Barbara Friedman Chair at MSK. At the American Society of Gene and Cell Therapy Annual Meeting, Dr. Sadelain will present preclinical data on FT819, an off-the-shelf, TCR-less, CD19 CAR T-cell product manufactured from a clonal master induced pluripotent stem cell line. The use of clonal master iPSC lines can overcome the complexity, heterogeneity and substantial costs associated with using cells from a patient or an allogeneic donor. Instead, iPSC-derived T-cell immunotherapies can be consistently and repeatedly mass produced and delivered in an off-the-shelf manner, significantly reducing the cost of, and time to, patient treatment. Fate Therapeutics has exclusively licensed from MSK intellectual property covering the production and composition of iPSC-derived T cells for human therapeutic use. In addition, Fate Therapeutics owns an extensive intellectual property portfolio that broadly covers compositions and methods for the genome editing of iPSCs using CRISPR and other nucleases, including the use of CRISPR to insert a CAR in the TRAC locus for endogenous transcriptional control

https://bit.ly/2L4dxn2

Albireo enrolls 1st patient in Phase 3 pediatric liver disease med trial

Albireo Pharma announced the first patient has been enrolled in PEDFIC-1, a Phase 3 clinical trial of lead product candidate A4250, an ileal bile acid transporter inhibitor being studied for the treatment of patients with progressive familial intrahepatic cholestasis. PFIC is estimated to affect between one in every 50,000 to 100,000 children born worldwide and causes progressive, life-threatening liver disease. Moderate to severe pruritus is a common and problematic clinical presentation of PFIC that can severely diminish quality of life. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life, and nearly all patients with PFIC require treatment before age 30. There are currently no approved pharmacological treatment options for PFIC. The Phase 3 program includes a single randomized, double-blind, placebo-controlled clinical trial designed to evaluate A4250 in 60 patients, ages 6 months to 18 years, with PFIC, elevated serum bile acid levels and pruritus, and an open-label extension study to assess long-term safety and durability of response. Patients in the double-blind trial will receive a 40 or 120 mug/kg oral dose of A4250 or placebo once daily for 24 weeks. The primary endpoint for the US Food and Drug Administration evaluation will be an assessment of change in pruritus, and the primary endpoint for the European Medicines Agency evaluation will be sBA responder rate.

https://bit.ly/2IpB9EP

J&J to relaunch its baby-care line: CNBC

After redesigning its baby-care unit for millennial moms, Johnson & Johnson will relaunch it in August, according to CNBC. The new-look line will have half the ingredients and will be easier to use while holding a baby. Since 2011 sales of the brand have declined 20%

https://bit.ly/2k2gLLz

FDA stiffs-arms Evolus rival to Botox, but execs promise a snap response

Evolus’ $EOLS attempt to field a rival to Allergan’s Botox has run into problems at the FDA.

The biotech announced Wednesday morning that the FDA has handed the company a complete response letter for DWP-450 (prabotulinumtoxinA). But the executive team quickly pinned the blame on manufacturing issues, saying they can bat their revised application back to regulators in 90 days.

The biotech’s statement starts with an announcement that the FDA has confirmed “the favorable completion of its pre-approval inspection of Daewoong’s manufacturing facility in South Korea which was purpose built for production of DWP-450.” They go on to say that despite the rejection, they’re staying upbeat — regardless of what investors might think.

And they weren’t nearly as upbeat as management. The biotech’s stock plunged 30% on the news.

CEO David Moatazedi — an Allergen vet who took the job just a little more than a week ago — says the rejection has nothing to do with data, statistics, safety or pharmacology of their product. “The remaining questions are manageable,” he told investors on a call this morning.

The biotech signaled at the beginning of the year that it had some serious manufacturing issues to deal with after the FDA issued a heavily redacted report on their inspection of the facility.

But the company declined to get specific, even after being pressed by Wells Fargo analyst David Maris.

“Obviously it’s a confidential document so we’re not going to give granularity,” noted CMO Rui Avelar.

The company says that it will get a Type 2 review for the revised pitch, setting up timeline for a planned launch next spring.

“We’ve largely de-risked the clinical development program,” says the CEO. “The pathway to commercialization is now clear” after the feedback. And Moatazedi adds he’ll start scaling for a commercial launching 2019.

He declined, though, to get into the specifics of the FDA’s questions.

“We are pleased with the progress we continue to make with the FDA, and this CRL confirms our confidence in our clinical submission,” said Moatazedi in a statement. “Deficiencies cited within the CRL are isolated to CMC matters and we expect to respond comprehensively within 90 days. Overall, we view these updates as positive, which together give us the line of sight necessary to build our commercial infrastructure. We look forward to working closely with the FDA and remain committed to bringing DWP-450 to market by spring 2019.”

https://bit.ly/2KpSnyv