Biogen Acquires Remedy Pharmaceuticals’ Late-Stage Drug CIRARA
May 18, 2017: Biogen acquires Remedy Pharmaceuticals’ late-stage drug CIRARA for $120 million upfront payment. Remedy Pharmaceuticals, a privately-held pharmaceutical company focused on bringing life-saving treatments to people affected by central nervous system diseases and injuries, today announced that Biogen completed an asset purchase of its Phase 3 candidate, CIRARA. Biogen made an upfront payment of $120 million to Remedy and may also pay additional milestone payments and royalties.
The target indication for CIRARA is large hemispheric infarction, a severe form of ischemic stroke where brain swelling often leads to disproportionately large share of stroke-related morbidity and mortality. The U.S. Food and Drug Administration (FDA) recently granted CIRARA Orphan drug designation for severe cerebral edema in patients with acute ischemic stroke. The FDA has also granted CIRARA Fast Track designation.
April 25, 2017: Biogen reports Q1 EPS of $5.20 versus the $4.97 estimate. Revenue also beat coming in at $2.81 billion versus the $2.73 billion estimate.
Since the end of the quarter, the Company has repurchased an additional approximately 2 million shares for a total value of $543 million.
The CEO Michel Vounatsos said, “I am very pleased with the results of the first quarter. We saw continued stability in our MS business, executed a strong launch of SPINRAZA, grew market share for our biosimilars business across Europe, and reinforced the intellectual property for TECFIDERA. Furthermore, we continued to build our neurology pipeline with the anticipated addition of our new Phase 2-ready anti-tau antibody… We are encouraged by the progress we made launching SPINRAZA in the U.S., and, following the positive CHMP opinion, we are ramping up pre-launch activities in Europe. The value this therapy provides to patients is compelling, and we are working to accelerate patient access globally. Overall, I believe we’re building positive momentum at the company, and I look forward to leading Biogen into a new and exciting era.”
Business Development Highlights
In April 2017, Biogen announced an agreement with Bristol-Myers Squibb to exclusively license BMS-986168, an experimental medicine with potential in Alzheimer’s disease and progressive supranuclear palsy (PSP), a rare condition that affects movement, speech, vision, and cognitive function. Biogen plans to initiate Phase 2 studies for BMS-986168 in both of these indications. Biogen anticipates making an upfront payment of $300 million to Bristol-Myers Squibb in the second quarter of 2017 as well as a near-term $60 million milestone payment to the former stockholders of iPierian, Inc. upon initiation of a Phase 2 trial for BMS-986168. These amounts exceed the estimated $100 million in business development expense assumed in Biogen’s previously announced 2017 full year financial guidance. This agreement is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 in the United States, and is expected to close in the second quarter of 2017.
April 24, 2017: Biogen will present Phase 3 end of study SPINRAZA (nusinersen) data from CHERISH, which demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset (most likely to develop Type 2 or Type 3) spinal muscular atrophy (SMA) compared to untreated children. The overall findings continue to support the robust efficacy and favorable safety profile of SPINRAZA across a broad range of individuals with SMA. The SPINRAZA development program represents the largest body of clinical data of its kind in SMA. SPINRAZA data will be presented at the American Academy of Neurology (AAN) annual meeting in Boston, Mass., April 22-28, 2017.
CHERISH: Later-onset SMA (Most Likely to Develop Type 2 or Type 3)
CHERISH is a Phase 3, multicenter, randomized, double-blind, sham-procedure controlled study to assess the efficacy and safety of SPINRAZA in children with later-onset SMA. The 15-month study investigated SPINRAZA in 126 non-ambulatory children 2 to 12 years old who experienced symptom onset at greater than 6 months of age.
In the CHERISH end of study analysis, children on SPINRAZA demonstrated a highly statistically significant and clinically meaningful improvement in motor function, as observed by the treatment difference of 4.9 points in the mean change from baseline to Month 15 in the Hammersmith Functional Motor Scale Expanded (HFMSE) score (p=0.0000001). The HFMSE is a validated tool specifically designed to assess motor function in children with SMA. When measuring changes from baseline, children who received SPINRAZA (n=84) achieved a 3.9 point mean improvement at Month 15, while children who were not on treatment (n=42) experienced a mean decline of 1.0 point. Primary endpoint results of the end of study analysis were consistent with results observed at the interim analysis.
Data from the other endpoints analyzed, including attainment of new motor milestones and upper limb motor function, were consistently in favor of children who received treatment.
SPINRAZA demonstrated a favorable safety profile. Treatment-emergent adverse events (AEs), severe AEs and serious AEs (SAEs) were reported less frequently in children treated with SPINRAZA than those not on treatment. The majority of the AEs were considered to be either related to SMA disease, common events in the general population, or events related to the lumbar puncture procedure. No children discontinued the study due to AEs.
NURTURE: Presymptomatic Infants with SMA
Biogen will also present new interim data from the Phase 2, multicenter, open-label, single-arm NURTURE study evaluating SPINRAZA for the treatment of infants under six weeks old with genetically diagnosed SMA who were presymptomatic at treatment initiation. At the time of the interim analysis, infants (n=20) were enrolled for a median of 317.5 days, and all infants were alive and none required respiratory intervention (chronic non-invasive ventilation, invasive ventilation or tracheostomy). Further, most infants achieved motor milestone and growth parameter gains generally consistent with normal development, such as head control, independent sitting, standing and walking independently, as measured by validated scales.
Three infants experienced AEs considered possibly related to SPINRAZA by the investigator, all of which were resolved. No infants have discontinued or withdrawn from the study due to AEs, and no new safety concerns have been identified.
January 23, 2017: TheStreet.com’s Feuerstein says Celgene should considering buying Biogen. Source: http://realmoney.thestreet.com/articles/01/23/2017/5-reasons-celgene-should-buy-biogen
January 13, 2017: Biogen presented new data from the Phase 3 ENDEAR study of SPINRAZA (nusinersen), which demonstrated a statistically significant reduction in the risk of death or permanent ventilation in SPINRAZA-treated infants with spinal muscular atrophy (SMA) compared to untreated infants. The data were presented at the British Paediatric Neurology Association (BPNA) annual conference in Cambridge, UK, 11-13 January 2017.
In August 2016, Biogen reported that ENDEAR met its pre-specified primary endpoint at the interim analysis, the proportion of motor milestone responders as measured by the Hammersmith Infant Neurological Examination (HINE). Following the positive interim analysis, Biogen ended the study early so that all participants could have the option to receive SPINRAZA in an open-label extension study. Today, Biogen provided the first presentation of the pre-specified primary endpoint, time to death or permanent ventilation, from the end of study (EOS) analysis. The EOS results presented at BPNA include data from patients final study visit, which occurred after the announcement that the study was being stopped and was not part of the interim analysis.
SPINRAZA met the pre-specified primary endpoint at the ENDEAR EOS, demonstrating a statistically significant 47% reduction in the risk of death or permanent ventilation (p<0.01). In the EOS analysis, a greater percentage of untreated infants (68%) died or required permanent ventilation compared to infants treated with SPINRAZA (39%).
SPINRAZA demonstrated a favorable safety profile, with commonly reported adverse events including respiratory events and constipation, consistent with those expected in the general population of infants with SMA. Further EOS efficacy and safety results from ENDEAR will be presented at a future medical congress.
ENDEAR was a randomized, double-blind, sham-controlled study in patients with infantile-onset (most likely to develop Type 1) SMA. The EOS efficacy analysis included all patients (n=121) who had their final study visit after the interim analysis (n=78) and had the opportunity to attend the six-month study visit assessment.
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals (NASDAQ: IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval in 2016.
December 28, 2016: Biogen, Inc. spokesperson says to set Spinraza wholesale acquisition cost (WAC) annual price at $375K after loading doses. Sees wholesale acquisition cost at $750K first 12 months, with price at $125K per vial. On 12/23/16, the FDA approved the first drug for spinal muscular atrophy; Spinraza is marketed by Biogen.
December 20, 2016: Biogen announced that its board of directors has approved the planned spin-off of its hemophilia business, which will be known as Bioverativ Inc., and declared a special dividend distribution of all of the outstanding shares of Bioverativ common stock.
For every two shares of Biogen common stock held of record as of the close of business on January 17, 2017, Biogen shareholders will receive one share of Bioverativ common stock. Registered shareholders will receive cash in lieu of fractional shares. The special dividend distribution is expected to be paid on February 1, 2017.
The distribution of Bioverativ common stock will complete the proposed separation of the hemophilia business from Biogen. After the separation, Bioverativ will become an independent, publicly-traded company focused on hemophilia and other rare blood disorders, and Biogen will retain no ownership interest. Bioverativ has applied for listing of its common stock on the NASDAQ Global Select Market under the ticker symbol BIVV.
The spin-off has been structured to qualify as a tax-free distribution to U.S. holders of Biogen’s common stock for U.S. federal income tax purposes. However, cash received in lieu of fractional shares may be taxable to such shareholders. Shareholders should consult their tax advisors with respect to U.S. federal, state, local and non-U.S. tax consequences of the separation and distribution of Bioverativ. No action is required by Biogen shareholders to receive shares of Bioverativ common stock as part of this special dividend distribution. Any holder of Biogen common stock who sells shares of Biogen common stock on or before the distribution date may be selling the entitlement to receive shares of Bioverativ common stock.
Following the spin-off, Bioverativ will be a global biotechnology company dedicated to advancing the research, development and commercialization of innovative therapies to address areas of serious unmet need for people with hemophilia and other rare blood disorders.
December 20, 2016: Bernstein reiterates an Outperform rating on Biogen and sets a price target of $345. Biogen has appointed Chief Commercial Officer Michel Vounatsos as CEO. Mr. Vounatsos joined Biogen in March after 20 years at Merck, most recently as the president of the Primary Care Business Line and Merck Customer Centricity. Mr. Vounatsos has a C.S.C.T. certificate in Medicine from the Universite Victor Segalen in France and received his M.B.A. from the HEC School of Management.
December 19, 2016: Biogen reportedly will promote Chief Commercial Officer Michel Vounatsos to CEO post; succession announcement may come this week. Mr. Vounatsos will replace Scangos, who has been CEO since July 2010.
December 9, 2016: Health Canada has approved ZINBRYTA (daclizumab beta), a new once-monthly, self-administered, subcutaneous treatment for adult patients with active relapsing-remitting multiple sclerosis (RRMS), who have had an inadequate response to, or are unable to tolerate, one or more therapies indicated for the treatment of multiple sclerosis, Biogen and AbbVie announced today. The Health Canada approval of ZINBRYTA is based on results from two clinical trials, including DECIDE, the largest and longest head-to-head global Phase 3 clinical trial conducted in MS. The Phase 2b SELECT and Phase 3 DECIDE studies were global, randomized, double-blind, controlled studies that involved approximately 2,400 people living with relapsing MS. Some patients in DECIDE were treated for up to three years.
December 9, 2016: Cowen reiterates an Outperform rating on Biogen, and sets a price target of $368. Cowen says that the BIIB and Lilly release of Ab antibody data sets in association with the CTAD meeting increases their optimism in the Ab hypothesis, the potential for aducanumab to be safe and efficacious, and the possibility that aducanumab could become one of the largest drugs the industry has ever seen.
December 8, 2016: TheStreet.com’s Feuerstein tweets: “I believe updated and embargoed aducanumab titration data have leaked from CTAD conference. Stock ticking higher.”
— Adam Feuerstein (@adamfeuerstein) December 8, 2016
@adamfeuerstein: “Seeing others tweet $BIIB aducanumab data so here they are: titration dose and long-term extension from PRIME study. CTAD screwed up.”
Abstract being circulated suggests: “after 12 months, significant decreases in brain amyloid plaque burden (as assessed by standard uptake value ratio) were observed with titrated aducanumab compared with placebo.”
– “The clinical efforts in the titration cohort were consistent with those observed in the fixed dosing cohorts and the slowing of decline on the CDR-SB was statistically significant with titrated aducanumab versus placebo.”
– “Conclusions: At 24 months, patients who were treated with aducanumab from the beginning of the double-blind phase through the LTE period continued to show a decrease in brain amyloid plaque burden in a dose and time-dependent manner as measured by amyloid PET. CDR-SB and MMSE data over 24 months also suggest a sustained benefit from aducanumab for those patients with continued treatment. The incidence of ARIA in patients switching to aducanumab treatment was consistent with that observed during the double-blind phase of the study, and no new cases of ARIA-E were observed in patients in the aducanumab continued groups.”
Biogen Stock Chart
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Through cutting-edge science and medicine, Biogen Idec discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hematologic conditions, and autoimmune disorders. Biogen has the leading portfolio of medicines to treat multiple sclerosis (MS) and is at the forefront of research into new drugs for neurological conditions and rare genetic disorders. Led by world-class research and development, Biogen uses new science and leading-edge technologies to create, commercialize, and manufacture transformative therapies for patients with few or no treatment options. Our global organization of more than 7,000 employees has a clear focus: to have the greatest impact on patients of any biotechnology company in the history of our industry. For more than two decades, Biogen has transformed the understanding and treatment of MS. Our research is focused on developing new MS treatments with the goal of repairing damage caused by the disease – and ultimately working to find a cure.