Deciphera Pharmaceuticals announced positive, preliminary, top-line data from the ongoing dose escalation part of the Phase 1 clinical study with DCC-3014, the Company’s investigational small molecule switch control inhibitor of CSF1R, in patients with advanced malignancies. In addition, the Company announced a plan to expand the Phase 1 study to evaluate DCC-3014 in patients diagnosed with Tenosynovial Giant Cell Tumors. A review of further data from this Phase 1 study will be presented at a medical meeting in 2019. The Phase 1 study was designed to evaluate the safety, pharmacokinetics and pharmacodynamics of multiple doses of DCC-3014 in up to 55 patients. As of the data cut-off date of November 9, 2018, increasing doses of DCC-3014 were assessed in five dose cohorts across 24 patients with advanced solid tumor malignancies. This included one dose cohort that received 10 mg once daily and four dose cohorts that followed a schedule of once or twice-weekly maintenance dosing preceded by a five-day loading dose regimen at doses of up to 30 mg per dose. In addition, four patients are currently enrolled in a dose cohort that will receive 40 mg twice weekly preceded by a five-day loading regimen. Preliminary pharmacokinetic analysis showed dose-proportional exposure for DCC-3014 that we believe supports twice-weekly maintenance dosing preceded by a five-day loading dose regimen. Biomarker data demonstrated strong target engagement of CSF1R, including material reductions in CSF1R positive macrophages in the blood that we believe constitutes mechanistic proof-of-concept for DCC-3014. DCC-3014 was generally well tolerated in patients enrolled in the dose cohorts that received twice-weekly maintenance doses of DCC-3014 preceded by a five-day loading regimen at doses of up to 30 mg, which is summarized below: Treatment emergent adverse events were mostly Grade 1 or 2; No Grade 3 or 4 DCC-3014 related TEAEs in greater than or equal to10% of patients; No dose-limiting toxicities; and A maximum tolerated dose has yet to be established. In the dose-cohort that received 10 mg QD, clinically asymptomatic laboratory values were recorded as dose-limiting toxicities in two of seven patients.
Wednesday, January 2, 2019
Moderna initiated with an Overweight at Morgan Stanley
Morgan Stanley analyst Matthew Harrison started Moderna with an Overweight rating and $29 price target, stating that he currently models peak, risk-adjusted sales of $8B, with prophylactic vaccines, rare diseases and cancer vaccines driving the bulk of that. However, Harrison added that the company’s broad pipeline includes 21 programs and if the entire pipeline were to be successful he believes peak sales could be as high as about $35B.
Vertex downgraded to Market Perform on lack of catalysts at Raymond James
Raymond James downgraded Vertex to Market Perform from Outperform. Analyst Laura Chico said Vertex lacks near-term catalysts which makes it more vulnerable in a “risk off” environment and said success is largely priced in at this point.
https://thefly.com/landingPageNews.php?id=2842467
Ultragenyx cut to Market Perform on lack of catalysts at Raymond James
Raymond James downgraded Ultragenyx to Market Perform from Outperform. Analyst Laura Chico said Ultragenyx lacks near-term catalysts which makes it more vulnerable in a “risk off” environment. Chico remains encouraged by the Crysvita launch, but said the company is only proportion of the economics.
https://thefly.com/landingPageNews.php?id=2842469
Leerink says expectations high for Medidata, initiates with Market Perform
Leerink analyst David Larsen started Medidata with a Market Perform Rating and a $70 price target. The analyst likes how the company is considered to be the premium solution with respect to clinical trial electronic data capture. But while the fundamentals of the industry and company are favorable, Larsen thinks there is significant risk to the stock. The overall market has been volatile which will pressure higher multiple stocks, and investor expectations for revenue growth are very high and any disappointment could result in a significant sell-off, he contents. Further, the analyst believes 95%-plus of biopharma already have electronic clinical solutions in place, and the market is becoming more competitive.
https://thefly.com/landingPageNews.php?id=2842475
Allena Pharmaceuticals aligns with FDA on design of URIROX-2
Allena Pharmaceuticals announced that it has reached alignment with the FDA on both the design of URIROX-2, its second pivotal Phase 3 trial of reloxaliase in patients with enteric hyperoxaluria and its strategy to pursue a biologics license application, or BLA, submission for reloxaliase using the accelerated approval regulatory pathway. Allena’s URIROX program consists of two pivotal Phase 3 clinical trials, URIROX-1 and URIROX-2, which are designed to evaluate the safety and efficacy of reloxaliase in patients with enteric hyperoxaluria. URIROX-1 is currently enrolling patients, and Allena initiated URIROX-2 in Q4. The primary efficacy endpoint for URIROX-2 is the percent change from baseline in 24-hour urinary oxalate, or UOx, excretion measured during weeks 1-4, the same primary efficacy endpoint as URIROX-1. The FDA agreed that, if positive, biomarker data on 24-hour UOx excretion in URIROX-1 and URIROX-2 would be used for a BLA filing for accelerated approval of reloxaliase in enteric hyperoxaluria and that patients would continue in URIROX-2 to confirm clinical benefit during the long-term follow-up phase of the trial.
https://thefly.com/landingPageNews.php?id=2842489
Merus in strategic collaboration with Betta Pharmaceuticals for MCLA-129
Merus announced that it has agreed to grant Betta Pharmaceuticals an exclusive license to develop and commercialize Merus Biclonics MCLA-129 in China. Merus will retain all rights outside of China. Under the terms of the agreement, Betta Pharmaceuticals has agreed to be responsible for clinical development and commercialization of MCLA-129 in China. As a key strategic component of the collaboration, Betta will retain a contract manufacturing organization with experience in filing initial new drug, or IND, applications with U.S. and European regulatory authorities in order to produce clinical trial materials for the Chinese market and rest of world. Betta will facilitate regulatory filings and early stage clinical trial materials supply for potential use by Merus for development of MCLA-129 outside of China. In preclinical studies, MCLA-129 showed a significant reduction in tumor volume for EGFR inhibitor resistant lung cancer models lacking immune cells. Additionally, in cell lines that co-express both EGFR and c-MET, MCLA-129 effectively induced tumor cell lysis at low antibody concentrations. In addition to receiving an upfront payment, Merus will be eligible to receive payments contingent upon Betta Pharmaceuticals achieving certain specified development and commercial goals in China. Merus will also be eligible to receive tiered royalty payments on sales in China from Betta Pharmaceuticals. Betta Pharmaceuticals will be eligible to receive payments contingent upon Merus achieving certain specified development and commercial goals, and will be eligible to receive tiered royalty payments on sales outside of China from Merus.
https://thefly.com/landingPageNews.php?id=2842517
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