The Medicines CompanyNASDAQ
The FDA Should Approve The New Hemostatic Product From The Medicines Company
Clinically Sound Investor
Clinically Sound Investor
Tue, Nov. 15, 1:08 PM
- Alnylam (ALNY -3.9%) and collaboration partner The Medicines Company (MDCO -6.1%) hit the skids after the release of 180-day follow-up data from the Phase 2 ORION-1 study assessing inclisiran for the treatment of high cholesterol. The results were presented at the American Heart Association Annual Meeting in New Orleans, LA.
- Investors are heading for the exits in apparent response to the safety data. On slide #8, 54% (n=198/370) of patients treated with inclisiran experienced a treatment-emergent adverse event (TEAE), including 9% (n=34/370) considered serious or severe. For comparison purposes, the TEAE rate for placebo was also 54% (n=69/127), including 8% (n=10/127) considered serious or severe.
- There was one death (n=1/65) in the 500 mg cohort, however.
- The numbers for liver and muscle TEAEs (slide #9) do not appear to be unreasonable. Myalgia (muscle pain) occurred in 5.7% (n=21/370) of the pooled group while liver enzyme elevations were observed in only 1.4% (n=5/370). The rates for the 300 mg cohort were 6.6% (n=8/122) and 3.3% (n=4/122), respectively.
- The Medicines Company plans to advance inclisiran to Phase 3 development at the 300 mg dose.
- Inclisiran (PCSK9si) is an investigational GalNAc-conjugated RNAi therapeutic targeting PCSK9, a protein regulator of LDL receptor metabolism. In contrast to PCSK9 inhibitors, PCSK9si works by turning off PCSK9 synthesis in the liver.
Mon, Nov. 14, 8:47 AM
- Alnylam Pharmaceuticals (NASDAQ:ALNY) is up 2% premarket and collaboration partner The Medicines Company (NASDAQ:MDCO) is up 8%, both on light volume, in response to increasing optimism regarding cholesterol-lowering candidate inclisiran (PCSK9si), an investigational GalNAc-conjugated RNAi therapeutic targeting PCSK9, a protein regulator of LDL receptor metabolism. It lowers bad cholesterol (LDL-C) by turning off PCSK9 synthesis in the liver.
- Results from a Phase 1 study was just published in The New England Journal of Medicine. The data showed doses of at least 300 mg (single or multiple) significantly lowered PCSK9 and LDL cholesterol for at least six months and was generally well-tolerated.
- Specifically, single doses of inclisiran of at least 300 mg reduced PCSK9 74.5% at Day 84. Doses at least 100 mg reduced LDL-C 50.6% at Day 84. The reductions were maintained at Day 180 will minor variation at doses at least 300 mg. Multiple doses reduced PCSK9 and LDL-C 83.8% and 59.7%, respectively, at Day 84. Levels remained reduced in all cohorts at Day 196.
- No serious adverse events were observed following single and multiple subcutaneous injections. All observed adverse events were mild or moderate in severity.
- Inclisiran was initially developed by Alnylam. The Medicines Company assumed the role of lead developer in August 2015. 180-day follow-up data from the Phase 2 ORION-1 study will be presented tomorrow, November 15, at the American Heart Association Annual Meeting in New Orleans, LA. Management will host a conference call at 3:00 pm ET tomorrow to discuss the results.
Mon, Nov. 7, 6:22 PM
- The Medicines Company (NASDAQ:MDCO) slumps 6% after hours on average volume in response to its announcement that it has terminated development of MDCO-216, an investigational cholesterol efflux promoter that it once believed had therapeutic potential for reducing atherosclerotic plaque.
- The company's decision is not surprising. Results from the Phase 2 MILANO-PILOT study failed to demonstrate sufficient efficacy.
- The company says another factor influencing its decision is the emerging positive data from another mid-stage study, ORION-1, assessing its PCSK9si candidate for the treatment of high cholesterol. It intends to reallocate MDCO-216's budget to this more promising candidate.
- Previously: The Medicines Company's mid-stage study assessing plaque buster MDCO-216 falls short of criteria for early termination; shares off 8% after hours (Aug. 30)
Mon, Nov. 7, 5:36 PM
Fri, Nov. 4, 12:18 PM
- London-based Motif Bio (Pending:MTFB) is set for its global IPO of 42.5M ordinary shares. The U.S. offering will be American Depositary Shares (ADSs) at ~$12.40. Each ADS represents 20 ordinary shares. Buyers will receive one half of a warrant to purchase one ADS with each ADS purchased. Ordinary shares will be offered in Europe. Buyers will receive one half of a warrant to purchase one ordinary share with each share bought.
- The clinical stage biopharmaceutical firm develops novel antibiotics to treat serious life-threatening infections in hospitalized patients caused by multidrug-resistant bacteria.
- It lead product candidate is Phase 3-stage iclaprim, a dihydrofolate reductase (DHFR) inhibitor originally discovered by Roche who licensed it to Arpida AG. Development ceased after the FDA rejected its marketing application in 2008. The company believes that iclaprim is approvable in the U.S. due to a more favorable regulatory environment. It cites the subsequent approvals of two antibiotics that failed to win approval about the same time as iclaprim was rejected: dalbavancin (Allergan's (AGN +1.9%) DALVANCE) and oritavancin (The Medicines Company's (MDCO +3.8%) ORBACTIV). Two Phase 3 studies, REVIVE-1 and REVIVE-2, are recruiting patients. Each is comparing iclaprim to vancomycin in patients with acute bacterial skin and skin structure infections (ABSSSI). Both should be completed in August 2017.
- 2016 Financials (6 mo.)($M): Operating Expenses: 13.9 (+707.9%); Net Loss: (14.2) (-661.6%); Cash Burn: (8.9) (-671.6%).
Wed, Oct. 26, 8:26 AM
Wed, Oct. 26, 7:07 AM
Tue, Oct. 25, 5:30 PM
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Tue, Oct. 18, 10:29 AM
- Biotech industry offers "attractive buying opportunities" given pre-election weakness, Jefferies says.
- Valuation multiples are near-historic lows; policy concerns are likely overstated.
- Firm recommends using weak sentiment to buy attractive names. Likes: Gilead (NASDAQ:GILD) and Celgene (NASDAQ:CELG) in large-caps, The Medicines Co. (NASDAQ:MDCO), Alkermes (NASDAQ:ALKS) and Vertex (NASDAQ:VRTX) in mid-caps. Alder Biopharmaceuticals (NASDAQ:ALDR), Cempra (NASDAQ:CEMP), Ultragenyx (NASDAQ:RARE) and Immunomedics (NASDAQ:IMMU) in small-caps.
Tue, Oct. 18, 9:16 AM
Tue, Oct. 18, 7:57 AM
- The Medicines Company (NASDAQ:MDCO) is up 1% premarket, albeit on only 400 shares, in response to its announcement of positive 90-day interim results from its Phase 2 clinical trial, ORION-1, assessing PCSK9si for the treatment of high cholesterol.
- Top-line data from 501 patients at Day 90 show treatment with PCSK9si produced a durable and significant reduction in LDL-C (bad cholesterol). In addition, no material safety signals were observed.
- More complete results from ORION-1, including 180-day follow-up data on up to 200 subjects, will be presented on November 15 at the American Heart Association Annual Meeting in New Orleans, LA.
- PCSK9si is an investigational GalNAc-conjugated RNAi therapeutic targeting PCSK9, a protein regulator of LDL receptor metabolism. In contrast to PCSK9 inhibitors, PCSK9si works by turning off PCSK9 synthesis in the liver.
Thu, Oct. 6, 11:00 AM
Thu, Oct. 6, 9:14 AM
Wed, Oct. 5, 6:39 PM
- Alnylam (NASDAQ:ALNY) craters 43% after hours on robust volume in response to its announcement that it has decided to terminate development of revusiran, an RNAi therapeutic for the potential treatment of hereditary ATTR amyloidosis (hATTR) with cardiomyopathy, an Orphan Drug designation. The company made its decision yesterday and communicated it to investigators, study sites and regulatory authorities.
- The problem appeared to be an unacceptable safety profile in the Phase 3 ENDEAVOR study following reports of new onset or worsening peripheral neuropathy (nerve damage) in revusiran-treated patients in the Phase 2 OLE trial. At the company's request, the Data Monitoring Committee met to review the reports and unblinded data from ENDEAVOR. Although, it did not find conclusive evidence of revusiran-related neuropathy it concluded that continued dosing was inappropriate due to an unfavorable benefit-risk profile, specifically an "imbalance" in mortality in the revusiran arm compared to placebo.
- The company says its decision does not affect Phase 3-stage patisiran, a candidate for the treatment of hATTR with polyneuropathy, or any other RNAi therapeutic program. It adds that an assessment of safety data across its other programs, including the ALN-PCSsc program with The Medicines Company (NASDAQ:MDCO), revealed no evidence of a drug-related neuropathy signal in over 800 patients with exposures up to 34 months. Investors appear unconvinced. MDCO is down 12% after hours on robust volume.
Wed, Oct. 5, 5:35 PM
Wed, Sep. 21, 7:58 AM
- The Medicines Company (NASDAQ:MDCO) inks a deal with the U.S. Department of Health and Human Services' Biomedical Advanced Research and Development Authority (BARDA) to provide funding for the development of new antibiotics to fight drug-resistant Gram-negative infections.
- Under the terms of the agreement, BARDA will provide $32M in initial funding and up to an additional $100M over five years if all options to extend the partnership are exercised by BARDA.
- The $32M will support a Phase 3 study assessing MDCO's Carbavance (meropenem-vaborbactam) for the treatment of hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. In 2014, the company's Infectious Disease unit was awarded a BARDA-backed cost-sharing contract to support the development of Carbavance, under which $55.8M has been committed to date.
- Carbavance is been designated a Qualified Infectious Disease Product for Fast Track review by the FDA.