BeiGene, Ambrx announce collaboration to develop biologics

Ambrx and BeiGene announced a global research and development collaboration. Ambrx has developed proprietary expanded genetic code technology platforms designed to allow the efficient incorporation of non-natural amino acids into proteins in both E. Coli and CHO cells. This technology enables site-specific modification of proteins to create potentially first- and/or best-in-class innovative protein drugs. The collaboration leverages Ambrx’s clinically validated drug discovery technology platforms with BeiGene’s expertise and resources to pursue the development and commercialization of next-generation biologics drugs. Under the terms of the agreement, Ambrx will receive an upfront payment of $10M to fund the initial discovery and research activities and additional upfront payments of up to $19M if BeiGene elects to initiate additional programs. Ambrx is eligible to receive potential development, regulatory, and sales-based milestone payments up to an aggregate of $446M for all programs, in addition to tiered royalties on future global sales. BeiGene will have worldwide rights to develop and commercialize any drug products resulting from the collaboration.
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Moderna reports Q4 revenue $35.42M, consensus $19.57M

Reports Q4 net loss $141.43M vs. $37.94M last year. “Execution by our team has enabled us to make important pipeline progress so far this year. We now have two additional programs ready for Phase 2 clinical development, newly opened INDs for our first rare disease program and a fifth immuno-oncology program, dosed the first cohort in a study of our systemically delivered mRNA that encodes for a secreted monoclonal antibody, and recently reported positive interim Phase 1 data for a novel combination vaccine designed to protect against viruses that can cause severe respiratory diseases in children,” said Stephane Bancel, Moderna’s CEO. “We look forward to generating new clinical data for programs across our portfolio over the next 12-24 months. Our strong cash position enables us to focus on advancing investigational medicines in our pipeline, pursue new candidates within our existing modalities and continue to invest in our mRNA platform to discover new modalities and treatments for patients across a broader range of disease areas.”
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Teva downgraded to Equal Weight on lack of growth visibility at Morgan Stanley

As previously reported, Morgan Stanley analyst David Risinger downgraded Teva to Equal Weight from Overweight, citing his belief that the stock’s premium valuation largely prices in expectations for upside in the coming quarters due to cost cutting and conservative Copaxone guidance. However, given the aggressive cost cutting and a lack of pipeline visibility beyond 2019, Risinger thinks long-term revenue growth is unclear and he sees better relative value elsewhere in the space, he tells investors. Risinger made no changes to his estimates for Teva but lowered his multiple, driving a decrease in his price target to $17 from $22. 
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CVS Health price target lowered to $68 from $94 at Citi

Citi analyst Ralph Giacobbe lowered his price target for CVS Health to $68 from $94 but keeps a Buy rating on the shares. The analyst says that while headwinds within the company’s retail are significant, he’s most surprised with the sizable drop implied within the healthcare benefits segment. However, he sees “a bridge” that gives credence to mid-single digit underlying growth. While admitting it is difficult to gain visibility on 2020, Giacobbe sees a “reasonable path” to $7.20 in earnings per share for 2020.
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Marinus Pharmaceuticals initiates Phase 3 Violet Study

Marinus Pharmaceuticals announced that it is initiating a single global pivotal Phase 3 clinical study evaluating oral ganaxolone in children with PCDH19-related epilepsy, or PCDH19-RE, a rare genetic epilepsy. If successful, the Violet Study is intended to support the regulatory filings for approval of ganaxolone in this underserved and refractory patient population. The Violet Study is a global, double-blind, randomized, placebo-controlled pivotal Phase 3 clinical study evaluating ganaxolone in children with PCDH19-RE. The study will enroll up to 70 patients between the age of one and 17 with a confirmed PCDH19 mutation. All patients that meet eligibility will be stratified into one of two biomarker groups and randomized within each stratum. The trial will consist of an 8-week prospective baseline period to collect seizure data, followed by a 17-week double-blind treatment phase. Patients randomized to ganaxolone will titrate over four weeks to a dose of up to 600 mg of ganaxolone oral liquid suspension three times a day and maintain that dose for the following 13-weeks. After the double-blind period, all patients who meet certain eligibility requirements will have the opportunity to receive ganaxolone in an open label phase of the study. The company expects to begin screening patients for enrollment into the study in Q2 and data from the study are estimated to be available in 2021.
https://thefly.com/landingPageNews.php?id=2875031

Baxter’s cardiovascular medicine eptifibatide approved by FDA

Baxter announced the FDA approval and launch of ready-to-use eptifibatide. Eptifibatide is a platelet aggregation inhibitor that prevents platelets-specialized blood cells-from sticking together and clotting. Eptifibatide is indicated for medical treatment of acute coronary syndrome, a broad term that includes heart attack and other emergency conditions in which the blood supply to the heart is suddenly stopped. Eptifibatide is also indicated for treatment of patients undergoing percutaneous coronary intervention, in which physicians insert a catheter to visualize and open blocked coronary arteries and may, if needed, implant a mesh tube, called a stent, to keep the artery open. Eptifibatide uses Baxter’s proprietary Galaxy container technology. Eptifibatide is currently available from Baxter in the U.S.
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Vertex says 2 Phase 3 studies of VX-445 combo studies met primary endpoint

Vertex Pharmaceuticals announced that treatment with the triple combination of the next-generation corrector VX-445, tezacaftor and ivacaftor resulted in statistically significant improvements in lung function in two Phase 3 studies in people with cystic fibrosis, or CF. Data from a pre-specified interim analysis of the Phase 3 study in people with one F508del mutation and one minimal function mutation showed a mean absolute improvement in ppFEV1 of 13.8 percentage points from baseline at week 4 of treatment compared to placebo. In the Phase 3 study in people with two F508del mutations, the addition of VX-445 in patients already receiving tezacaftor and ivacaftor resulted in a mean absolute improvement in ppFEV1 of 10.0 percentage points from baseline at week 4 of treatment compared to the control group in whom placebo was added to tezacaftor and ivacaftor. The VX-445 triple combination regimen was generally well tolerated, and the safety and efficacy profile from the results released today supports the potential submission of a New Drug Application for the VX-445 triple combination regimen. The Phase 3 data announced today for the VX-445 triple combination regimen follow Phase 3 data announced in late 2018 for the triple combination of VX-659, tezacaftor and ivacaftor that also showed a safety and efficacy profile supportive of a potential NDA submission. Given the similarity of the data for the 4-week primary efficacy endpoint for the VX-659 and VX-445 regimens and the near-term availability of the final 24-week data for both regimens in the second quarter of 2019, Vertex plans to utilize these final 24-week data to choose the best regimen to submit for regulatory approvals globally. Because these submissions will include the final 24-week data, Vertex will seek approval for patients ages 12 and older with one F508del mutation and one minimal function mutation and for patients with two F508del mutations concurrently. Vertex plans to submit an NDA to the U.S. FDA in the third quarter of 2019 and a Marketing Authorization Application in Europe in the fourth quarter of 2019 for either the VX-659 or VX-445 triple combination regimen. The company also plans to disclose more detailed data from the Phase 3 studies of the selected triple combination regimen, including 24-week data from the study in patients with one F508del mutation and one minimal function mutation, in the second quarter of 2019. “Both the VX-659 and VX-445 triple combination regimens showed highly consistent and significant improvements in lung function across our Phase 3 programs, underscoring the important clinical benefit that a triple combination regimen may provide to patients with two F508del mutations and to those with one F508del and one minimal function mutation,” said Reshma Kewalramani, M.D., Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “We look forward to submitting global regulatory applications for one of these triple combination regimens for both patient populations later this year.”

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