Reuters reports that the pace of FDA approvals of new medicines is trending over double the rate of last year, with 21 getting the thumbs up so far compared to nine at the same point last year. A total of 22 new drugs were approved in all of 2016.
A key driver of the trend is drug makers' move into specialized therapies that commonly qualify for accelerated review.
In terms of sales potential, the top three recently approved products are Sanofi and Regeneron's eczema med Dupixent ($5B projected peak sales), Roche's MS drug Ocrevus ($4B) and AstraZeneca's durvalumab ($3B). Sanofi and Regeneron's rheumatoid arthritis med Kevzara (sarilumab), approved a few days ago, should be in the mix as well.
According to QuintilesIMS, the pharmaceutical sector has a robust enough pipeline to yield 40 - 45 approvals each year through 2021.
Roche (OTCQX:RHHBY) is looking into a case of a potential deadly brain infection called progressive multifocal leukoencephalopathy (PML) that occurred in a German patient after taking one dose of MS drug Ocrevus (ocrelizumab). The person had been treated with Biogen's (BIIB +2.2%) Tysabri (natalizumab) for three years before switching.
Roche says the rare condition was reported as a carryover from Tysabri by the doctor treating the patient. Biogen declined comment on the matter.
There were no reported cases of PML in Ocrevus clinical trials, but the company included warnings about the risk from taking the drug.
The European Commission grants standard marketing authorization for Biogen's (NASDAQ:BIIB) multiple sclerosis (MS) drug FAMPYRA (prolonged-release fampridine tablets) based on the results from the Phase 3 ENHANCE study which confirmed the med's efficacy and safety.
FAMPYRA was conditionally approved in the EU in 2011.
Celgene (NASDAQ:CELG) is up a fraction premarket on the heels of its announcement of positive results from a second Phase 3 clinical trial, RADIANCE, assessing ozanimod (formerly RPC1063) for the treatment of relapsing multiple sclerosis (RMS).
The study met the primary endpoint of reducing annualized relapse rate compared to Biogen's (NASDAQ:BIIB) Avonex (interferon beta-1a).
The company reported successful results from its first Phase 3, SUNBEAM, in February.
Detailed results will be submitted for presentation at an upcoming medical conference. Global regulatory filings will commence by year-end.
Johnson & Johnson (JNJ -0.3%) Janssen unit has filed a lawsuit in a New Jersey court attempting to block the entry of a biosimilar to top seller Remicade (infliximab) made by Samsung Bioepis, the joint venture between Samsung BioLogics and Biogen (BIIB +0.2%).
Janssen accuses Bioepis of violating Remicade patents and refusing to participate in a patent resolution process for biosimilars. It seeks to block the U.S. market launch of the product, to be marketed by Merck (MRK -0.1%) after last month's FDA approval, in addition to damages.
Noting the read-through for aducanumab from Ionis Pharma's TTR Neuropathy data this morning, Piper is out with a whopping $110 price target hike for Biogen (BIIB +1.2%) to $442.
Though the aducanumab data readout isn't coming until 2020, Piper thinks there's a 50/50 chance of success, and that - combined with 50% ownership of the asset - is incredibly valuable no matter what happens to the MS franchise.
Biogen (NASDAQ:BIIB) acquires CIRARA (intravenous glyburide) from Remedy Pharmaceuticals. The target indication is the treatment of a severe form of ischemic stroke with brain swelling called large hemispheric infarction (LHI). In the U.S. it has Orphan Drug and Fast Track status.
Under the terms of the transaction, Remedy will receive an upfront payment of $120M, undisclosed milestones and sales-based royalties.
Remedy says CIRARA inhibits a specific channel called Sur1-Trpm4 that is upregulated following ischemic and traumatic injury. The channel allows excess sodium to move into cells followed by in influx of water which leads to swelling, damage and death.
Biotechs are among the leaders in a broad market rally today. Novartis (NYSE:NVS) (+2.4%) added punch to the party with its earnings call comments about seeking new early-stage acquisitions and in-licensing deals to beef up its pipeline.
Pfizer's (NYSE:PFE) Besponsa (inotuzumab ozogamicin) receives a positive recommendation from the European Medicines Agency's CHMP for the treatment of acute lymphoblastic leukaemia. The drug has orphan designation.
Biogen's (NASDAQ:BIIB) Spinraza (nusinersen) receives postive recommendation for treatment of spinal muscular atrophy.
BioMarin's (NASDAQ:BMRN) Brineura (cerliponase alfa) receives positive recommendation for treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
Glaxo's (NYSE:GSK) Celsentri (maraviroc) receives use extension recommendation.
Bristol-Myers' (NYSE:BMY) Opdivo (nivolumab) receives use extension recommendation.
Roche's (OTCQX:RHHBY) Avastin (bevacizumab) receives use extension recommendation.
Expect drug-price growth to slow, Barron's cover story says, noting that investors should be wary of “serial price-hikers” who could find it tough to keep up with Wall Street forecasts as a result.
Since 2008, prices for the most commonly used branded prescription drugs have soared 208%, compared with a 14% rise in consumer prices.
Until recently, investors have fared well with shares of AbbVie (NYSE:ABBV), Amgen (NASDAQ:AMGN) and Biogen (NASDAQ:BIIB). Now, they should consider companies like Regeneron Pharmaceuticals (NASDAQ:REGN) and Vertex Pharmaceuticals (NASDAQ:VRTX) that are increasing revenue by launching new drugs and growing patient populations.
Vertex is an example of a biotech “poised to grow quickly” by winning patients, not just charging more. Shares are up more than 50% YTD on positive trial results.
Biogen has licensed BMS-986168, an anti-eTau compound, for the potential treatment of a rare brain disorder called progressive supranuclear palsy (PSP), also known as Steele-Richardson-Olszewski syndrome. The most frequent first symptom is the loss of balance while walking. Under the terms of the agreement, Biogen will pay $300M upfront and milestones valued up to $410M.
Roche has licensed BMS-986089, a novel fusion protein designed to suppress a protein called myostatin, a negative regulator of muscle growth. It will be developed for the potential treatment of Duchenne muscular dystrophy (DMD). Under the terms of the agreement, Roche will pay $170M upfront and milestones up to $205M.
BMY will earn tiered double-digit royalties on either product candidate, if approved.
Preliminary data from an ongoing Phase 1/2 clinical trial evaluating Spark Therapeutics' (ONCE +1.4%) factor IX product SPK-9001 show a significant treatment effect. The results will be presented tomorrow at the Hemostasis and Thrombosis Research Society 2017 Scientific Symposium in Scottsdale, AZ.
As of March 24, 10 patients who received a single administration SPK-9001 have discontinued routine factor IX infusions. Nine of 10 have not taken factor IX concentrates to prevent or control bleeding events. Two of 10 experienced a temporary elevation in liver enzymes or decline in factor IX activity, potentially indicating an immune response to the Spark100 vector capsid. Treatment with corticosteroids resolved both.
Based on patient histories prior to the study, annualized bleed rates (ABRs) were reduced 96% to a mean of 0.39 annual bleeds. Annual infusion rates (AIRs) were reduced 99% to an average of 0.98 annual infusions.
SPK-9001 is a bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimized high-activity human factor IX variant designed to deliver and express a therapeutic gene in the liver. It was developed using Spark's proprietary technology platform called SPK-FIX which is being advanced under a 2014 collaboration with Pfizer.