The Medicines Company (MDCO) – Inclisiran Indeed! Following up on the positive top-line results of the ORION 11 trial (released 8/26), the actual data presented at the ESC Congress 2019 were uniformly favorable for both efficacy and safety. In addition, despite the relatively smaller study and not powered for outcomes (n=1607), there was a strong reduction in Cardio Vascular Outcomes (CVO) in the Inclisiran treated arm versus placebo. (… more)
The Medicines Company (MDCO) – MDCO presented interim results from Group 1 of the ORION-3 Phase II study (n=290) at the NLA (Miami) on Saturday. The long-term 3-year data showed that patients given additional inclisiran doses had LDL cholesterol reduced by >50% over time, consistent with the shorter-term ORION-1 results. (…more)
This morning ALKS released their Q1:19 financial results that showed a big miss for Aristada coming in at $30 million after guiding for $40 million. While a disappointment, the company did not change overall FY guidance and stated that an inventory work down was the primary cause.
BioInvest News Novavax (NVAX) – This morning, NVAX reported disappointing top-line data from the ResVax Phase III PREPARE study for infant RSV prevention. Unfortunately, the trial failed on the primary endpoint of reducing medically significant RSV lower respiratory tract infection (LRTI).
The Medicines Company (MDCO) – With efficacy consistent with the Phase I and II studies and a safety profile at least as favorable as in previous trials (e.g., ORION-1 Phase 2 and ORION-3 open-label extension studies), Inclisiran is well on its way to FDA approval and, in our view, blockbuster status… more
(ESPR) — FDA Accepts Both BA NDAs with No Need for Advisory Panel – The FDA has accepted both New Drug Applications for bempedoic acid and the bempedoic acid/ezetimibe combination tablet for filing and regulatory review.
Ionis (IONS) — An update from Spinraza competitor Zolgensma (NVS) and the shift out of health care stocks are driving today’s decline. Shares are down 14% after Novartis presented a positive update to its STRIVE study for Spinal Muscular Atrophy (SMA) yesterday at the MDA 2019 meeting.
BioInvest News – NKTR’s stock is under pressure after the early update to NKTR’s Phase 2 PIVOT-02 trial of NKTR-214 in combination with Opdivo which includes 13 additional urothelial cancer (UC) patients vs. the 10 reported at ASCO 2018. The data remains solid despite the small decrease in ORR which is now 48% vs. 60% prior.
BioMarin (BMRN) – The widely anticipated BioMarin hemophilia A gene therapy delivered good news this morning, albeit with some caveats. BMRN announced both positive Phase I/II and Phase III data for ValRox (valoctocogene roxaparvovec) for adults with severe hemophilia A.
Pacira (PCRX) – On Tuesday (4/30), Heron (HRTX) received a surprise Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for HTX-011 for the management of postoperative pain. The CRL stated that the FDA is unable to approve the NDA in its present form based on the need for additional CMC and non-clinical information.
BioInvest News – Sangamo (SGMO) has delivered excellent news across their proprietary ZFN drug development platform with promising human data from both the Hemophilia A program with Pfizer and Beta Thal with Sanofi.
BioInvest News – Sangamo (SGMO) Updated MPS I & II Trials Disappoint But ERT Withdrawals Continue & That’s Key, More Powerful 2nd Generation ZFNs in Clinic by Year-End, Stock Overreaction
