Mon, Mar. 23, 4:26 PM
- According to Thomson Reuters' 2015 Drugs to Watch report, as many as 11 blockbuster drugs (peak sales of at least $1B within five years) are expected to clear the regulatory process this year. This is a significant jump from 2014's tally of three (Gilead's (NASDAQ:GILD) Sovaldi and Zydelig; Glaxo (NYSE:GSK) and Theravance's (NASDAQ:THRX) Anoro Ellipta). In reverse order (forecasted 2019 consensus sales in parentheses):
- 11. Novartis' (NYSE:NVS) Cosentyx (secukinumab) for psoriasis and psoriatic arthritis ($1.08B); 10. Sanofi's (NYSE:SNY) Toujeo (insulin glargine) for diabetes ($1.26B); 9. Otsuka Pharmaceutical (OTCPK:OTSKY) and Lundbeck's (OTCPK:HLUYY) Brexpiprazole for schizophrenia and depression ($1.35B); 8. Merck's (NYSE:MRK) Gardisil 9 HPV vaccine ($1.64B); 7. Amgen's (NASDAQ:AMGN) evolocumab for hypercholesterolemia/hyperlipidemia ($1.86B); 6. AbbVie's (NYSE:ABBV) Veikira Pak for hepatitis C (cleared 12/19/14) ($2.5B); 5. Vertex Pharmaceuticals' (NASDAQ:VRTX) lumacaftor plus ivacaftor for cystic fibrosis ($2.74B); 4. Pfizer's (NYSE:PFE) Ibrance (palbociclib) for breast cancer ($2.76B); 3. Novartis' LCZ696 for chronic heart failure ($3.73B); 2. Regeneron Pharmaceuticals' (NASDAQ:REGN) Praluent (alirocumab) for hypercholesterolemia ($4.41B) and 1. Bristol-Myers Squibb's (NYSE:BMY) Opdivo (nivolumab) for melanoma ($5.68B).
Mon, Mar. 23, 10:33 AM
- Vertex Pharmaceuticals (VRTX -4.9%) eases on average volume in response to its announcement of Phase 2 data for two doses of Orphan Drug-designated VX-661, in combination with ivacaftor (Kalydeco) in cystic fibrosis (CF) patients at least 18 years old with the two copies of the F508del mutation.
- The primary endpoint was safety, while the secondary endpoints assessed the effect of the drug combination on lung function as measured by ppFEV1 (percent predicted forced expiratory volume in one second). The mean improvement from baseline in ppFEV1 for the 100 mg dose of VX-661 was 4.4% (n=15; p=0.009) and 3.0% (p=0.026) at Week 4 and through 12 weeks of treatment, respectively. After patients completed treatment, lung function returned to baseline. The mean absolute change in ppFEV1, though, was only 1.0% compared to placebo's -0.4%. Investors were apparently expecting a bit more.
- VX-661 is CF transmembrane conductance regulator ((CFTR)) corrector. CF is caused by loss-of-function mutations in the CFTR protein. A CFTR corrector addresses the defects in the cellular processing and chloride channel function of CF-causing mutant CFTR alleles, thereby producing a clinical benefit.
Wed, Mar. 18, 9:05 AM
- The FDA approves an expanded label for Vertex Pharmaceuticals' (NASDAQ:VRTX) cystic fibrosis drug Kalydeco (ivacaftor) to now include G551D mutation-positive children ages 2 - 5. It was previously cleared for use in children at least six years old.
- Previously: PDUFA date approaches for Kalydeco label expansion (March 16)
Mon, Mar. 16, 12:53 PM
- Tomorrow is the PDUFA date for the FDA's decision an expanded indication for Vertex Pharmaceuticals' (VRTX +3.1%) Kalydeco (ivacaftor) to include children ages 2 to 5 with cystic fibrosis (CF) who have the G551D mutation. Kalydeco is currently cleared for use in G551D-positive children with CF at least 6 years old.
- Previously: FDA clears expanded label for Kalydeco (Dec. 29, 2014)
Thu, Mar. 12, 8:09 AM
- Concert Pharmaceuticals (NASDAQ:CNCE) starts a Phase 1 clinical program for deuterium-modified ivacaftor, a treatment for cystic fibrosis sold under the brand name Kalydeco by Vertex Pharmaceuticals (NASDAQ:VRTX). The trial will enroll ~45 healthy volunteers to assess single doses of two deuterium-modified compounds, each of which has demonstrated greater metabolic stability compared to Kalydeco in preclinical testing. Based on the results, Concert will select one compound for advancement into the single ascending dose portion of the trial. The final phase, expected to start in H2, will assess multiple ascending doses compared to placebo.
- Concert modifies currently-approved products based on deuterium (heavy hydrogen) chemistry. Deuterated compounds can have an increased half-life in the body which increases systemic exposure compared to the non-deuterated versions which can improve their safety and efficacy.
Thu, Mar. 5, 8:52 AMAbbVie's high-priced takeout of Pharmacyclics bodes well for other biotechs with high potential drugs
- AbbVie's (NYSE:ABBV) extraordinarily generous buyout of Pharmacyclics (NASDAQ:PCYC) is a crystal clear example of how keen big pharma is to boost its prospects with biotech drugs with blockbuster potential. The transaction's $20B tab, backing out PCYC's $1B cash balance, values Imbruvica (ibrutinib) at $40B since Pharmacyclics' commercial partner Johnson & Johnson (NYSE:JNJ) gets 50% of Imbruvica's revenue. This implies a multiple of almost 7x Imbruvica's projected peak sales of $6B. By comparison, Amgen's (NASDAQ:AMGN) takeout of Onyx Pharma and Sanofi's (NYSE:SNY) takeout of Genzyme were both at 5x premiums.
- The looming patent expiration for Humira ($12.5B in sales the past four quarters) undoubtedly provided AbbVie extra incentive to get the deal done.
- Adding 50% of Imbruvica's sales to AbbVie's top line should increase its revenue growth 3 - 7% and EPS growth 8 - 11% through 2020.
- According to RBC analyst Michael Yee, other biotechs on the big ticket acquisition radar are: BioMarin Pharmaceuticals (NASDAQ:BMRN), Dyax (NASDAQ:DYAX), Esperion Therapeutics (NASDAQ:ESPR), United Therapeutics (NASDAQ:UTHR), Vertex Pharmaceuticals (NASDAQ:VRTX), Intercept Pharmaceuticals (NASDAQ:ICPT), Juno Therapeutics (NASDAQ:JUNO), Kite Pharma (NASDAQ:KITE), PTC Therapeutics (NASDAQ:PTCT) and Receptos (NASDAQ:RCPT).
Wed, Jan. 28, 4:20 PM
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Wed, Jan. 21, 11:41 AM
- Merck (MRK) plans to withdraw its hepatitis C medicine, Victrelis (boceprevir), from the U.S. market by the end of this year due to declining demand in the wake of the Gilead Sciences (GILD +1.1%) juggernaut. Victrelis will remain available internationally.
- Vertex Pharmaceuticals (VRTX +0.4%) pulled its offering, Incivek (telaprevir) in August for the same reason and exited the hepatitis arena.
- Merck is not giving up yet. It plans to file an NDA by mid-year for an all-oral ribavirin-free hep C regimen, grazoprevir/elbasvir.
Dec. 29, 2014, 3:35 PM
- The FDA approves Vertex Pharmaceuticals' (VRTX +1%) supplemental new drug application (sNDA) expanding the label of Kalydeco (ivacaftor) to include patients aged 6 and older with cystic fibrosis (CF) who have the R117H mutation in the CFTR gene. Kalydeco is now approved for the treatment of CF in patients with one of 10 mutations: R117H, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D.
- Approximately 500 people in the U.S. at least six years old with CF have the R117H mutation. Kalydeco is now approved to treat ~3,100 CF patients in the U.S. with one of the specific mutations.
- Previously: Ad Comm vote positive for Kalydeco label expansion (Oct. 21)
- Previously: Vertex applies for a label expansion for Kalydeco (June 30)
Dec. 19, 2014, 5:36 PM
Nov. 20, 2014, 8:03 AM
- The Cystic Fibrosis Foundation sells the royalty stream it receives from Vertex Pharmaceutics (NASDAQ:VRTX) to Royalty Pharma for $3.3B. It plans to reinvest the money into new research efforts.
- The transaction is an example of successful mission investing, a growing trend in philanthropy. Instead of giving charitable grants, the foundation acts as a business partner by making an investment and expecting a share of the profits.
- Vertex's Kalydeco (ivacaftor), which helps CF patients with a rare genetic mutation, costs more than $300K per year. CF Foundation Chief Robert J. Beall says, "While the drug's high price may have boosted the royalties the foundation gets, we would give up our royalties in a second to drive down the prices. We were not at the pricing table." He adds that the foundation has no plans to subsidize the cost of drugs to patients. "What we're really excited about is the move to a one-time cure." At present, the meds are daily therapies.
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Nov. 14, 2014, 12:00 PM| 41 Comments
Nov. 5, 2014, 7:55 AM
- Vertex Pharmaceuticals (NASDAQ:VRTX) files an NDA with the FDA and a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for a fully co-formulated combination of lumacaftor and ivacaftor for patients at least 12 years old with cystic fibrosis (CF) who have two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene.
- There are ~22,000 people who fit this profile in North America, Europe and Australia.
- The FDA tagged it a Breakthrough Therapy in late 2012. The NDA includes a request for Priority Review which, if approved, will shorten the regulatory review time to ~eight months. Europe's Committee for Medicinal Products for Human Use (CHMP) granted the company's request for Accelerated Assessment of its MAA which shortens the review time to ~150 days.
Oct. 28, 2014, 6:57 PM
- Vertex Pharmaceuticals (VRTX +2.2%) Q3 results: Revenues: $179M (-19.3%); Product Sales: $137.1M (-26.6%); Kalydeco Sales: $126.8M (+25.4%); Operating Expenses: $266.2M (-13.0%); Operating Loss: ($142.2M) (-15.0%); Net Loss: ($170.1M) (-37.1%); Loss Per Share: ($0.72) (-33.3%); Quick Assets: $1.48M (+0.7%).
- 2014 Guidance: Revenues: $520M - 550M (unch); Kalydeco sales: $470M - 500M (unch); Operating Expenses: $910M - 920M from $890M - 930M.
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