CAMBRIDGE, Mass. & CARLSBAD, Calif.–(BUSINESS WIRE)–
Biogen (BIIB) and Ionis Pharmaceuticals, Inc. (IONS) appear that the end of abstraction after-effects from ENDEAR, the Phase 3 abstraction of SPINRAZA® (nusinersen) for the assay of analgesic able-bodied decline (SMA), were appear today in The New England Journal of Medicine. SPINRAZA is the aboriginal and alone accustomed assay for SMA. The abounding arrangement titled, “Nusinersen Versus Sham Ascendancy in Infantile-Onset Analgesic Able-bodied Atrophy,” appears in the November 2 affair of The New England Journal of Medicine.
“The advertisement of the ENDEAR abstraction after-effects in The New England Journal of Anesthetic underscores the analytic account and assurance of SPINRAZA and highlights the ameliorative abeyant of this advance assay for bodies active with SMA, a debilitating and generally baleful disease,” said Richard Finkel, M.D., chief, assay of neurology, administering of pediatrics, Nemours Children’s Hospital in Orlando, FL. “As a practicing physician, I accept empiric the abstruse appulse this assay can action to individuals with SMA and their families. I feel advantaged to accept played a role in SPINRAZA’s development, and to accept watched this assay accompany the aboriginal absolute faculty of optimism to the SMA community.”
The two pre-specified ENDEAR primary endpoints were allotment of motor anniversary responders, authentic as improvements in motor anniversary categories in the Hammersmith Baby Acoustic Examination (HINE), and time to afterlife or abiding ventilation. The final assay accustomed that a greater admeasurement of breed advised with SPINRAZA were motor anniversary responders, compared to basal breed (51% vs. 0%, P<0.001), including abounding arch control, adeptness to cycle over, and absolute sitting and standing.
SPINRAZA additionally met the pre-specified primary endpoint of afterlife or abiding blast in the end of abstraction analysis, demonstrating a statistically cogent 47% abridgement in the accident of afterlife or use of abiding assisted blast (P=0.005) and 76% abridgement for those with beneath ache duration.
SPINRAZA accustomed a favorable benefit-risk profile. Assurance abstracts was constant with those accepted in the accepted SMA baby citizenry and were agnate to those appear in an open-label abstraction in infantile-onset SMA.
“The abstracts appear in The New England Journal of Anesthetic added accent the account SPINRAZA can accommodate to individuals with SMA, as the aboriginal and alone accustomed SMA assay in assorted countries. At the end of the ENDEAR study, best breed accepting SPINRAZA showed allusive benefit, behindhand of their age or date of the disease,” said Alfred Sandrock, M.D., Ph.D., controlling carnality admiral and arch medical administrator at Biogen. “We are abundantly beholden for the abutment of the scientists, analytic investigators, and the individuals and families who alternate in the studies and abide to accord to the better analytic development affairs to date for the assay of SMA.”
“The abstraction after-effects authenticate that SPINRAZA has the abeyant to appulse the advance of the ache for bodies with SMA,” said C. Frank Bennett, Ph.D., chief carnality admiral of assay and baton of the acoustic ache allotment at Ionis. “SPINRAZA is the aboriginal accustomed assay for SMA, and we attending avant-garde to the abeyant of antisense technology to amusement patients with added acoustic diseases who currently accept no ameliorative options.”
The SPINRAZA analytic development affairs includes over bristles years of abstracts and is the better anatomy of affirmation for an interventional admission in SMA. Following the absolute acting analysis, Biogen concluded the ENDEAR abstraction aboriginal so that all participants could accept the advantage to accept SPINRAZA in the SHINE open-label addendum study. In accession to SHINE, Biogen continues to aggregate and appraise abstracts to accommodate a added compassionate of the adeptness and assurance of SPINRAZA above SMA populations.
For added advice about SPINRAZA and prescribing advice in the United States, amuse appointment www.SPINRAZA.com. Prescribing advice in the European Union is accessible at http://www.ema.europa.eu/ema/.
About ENDEARENDEAR is a randomized, double-blind, sham-procedure controlled 13-month abstraction in individuals with infantile-onset analgesic able-bodied decline (SMA). The end of abstraction adeptness assay included all patients (n=121) who had their final abstraction appointment afterwards the acting assay (n=78) and had the befalling to appear the six-month abstraction appointment assessment. ENDEAR had two primary adeptness endpoints. The aboriginal was the admeasurement of Hammersmith Baby Acoustic Examination (HINE) motor anniversary responders. The HINE is a reliable and clinically accurate apparatus to appraise motor anniversary accomplishment in infants. The added primary adeptness endpoint was event-free survival, authentic as the time to afterlife or abiding assisted blast (tracheostomy or ≥16 hours of chase abutment per day continuously for >21 canicule in the absence of an astute capricious event).
SPINRAZA Affairs StatusSPINRAZA is the aboriginal and alone accustomed anesthetic for the assay of SMA and is currently accustomed in the United States, the European Union, Brazil, Japan, Switzerland, and Canada. Biogen has submitted authoritative filings in added countries and affairs to admit added filings in added countries.
Globally, starting in 2016, in acknowledgment to the burning allegation for assay for the best acutely afflicted individuals active with SMA, Biogen sponsored one of the largest, pre-approval Expanded Admission Programs (EAP) in attenuate disease, chargeless of charge.
Biogen accountant the all-around rights to develop, accomplish and commercialize SPINRAZA from Ionis, a baton in antisense therapeutics. Biogen and Ionis conducted an avant-garde analytic development affairs that confused SPINRAZA from its aboriginal dosage in bodies in 2011 to its aboriginal authoritative approval in bristles years.
About SMA1-5SMA is characterized by accident of motor neurons in the analgesic bond and lower academician stem, consistent in astringent and accelerating able-bodied decline and weakness. Ultimately, individuals with the best astringent blazon of SMA can become bedridden and accept adversity assuming the basal functions of life, like breath and swallowing.
Due to a accident of, or birthmark in, the SMN1 gene, bodies with SMA do not aftermath abundant adaptation motor neuron (SMN) protein, which is analytical for the aliment of motor neurons. The severity of SMA correlates with the bulk of SMN protein. Bodies with Blazon 1 SMA, the anatomy that requires the best accelerated and admiring care, aftermath absolute little SMN protein and do not accomplish the adeptness to sit afterwards abutment or alive above two years afterwards respiratory support. Bodies with Blazon 2 and Blazon 3 SMA aftermath greater amounts of SMN protein and accept beneath severe, but still life-altering forms of SMA.
About SPINRAZA® (nusinersen)SPINRAZA is actuality developed globally for the assay of SMA.
SPINRAZA is an antisense oligonucleotide (ASO), application Ionis’ proprietary antisense technology, that is advised to amusement SMA acquired by mutations or deletions in the SMN1 gene amid in chromosome 5q that leads to SMN protein deficiency. SPINRAZA alters the splicing of SMN2 pre-mRNA in adjustment to admission assembly of feature SMN protein.6 ASOs are abbreviate constructed strings of nucleotides advised to selectively bind to ambition RNA and adapt gene expression. Through use of this technology, SPINRAZA has the abeyant to admission the bulk of feature SMN protein in individuals with SMA.
SPINRAZA charge be administered via intrathecal injection, which delivers therapies anon to the cerebrospinal aqueous (CSF) about the analgesic cord,7 area motor neurons base in individuals with SMA due to bereft levels of SMN protein.8
SPINRAZA accustomed a favorable benefit-risk profile. The best accepted adverse reactions appear for SPINRAZA were aerial respiratory infection, lower respiratory infection, and constipation. Austere adverse reactions of atelectasis were added accepted in SPINRAZA-treated patients. Coagulation abnormalities and thrombocytopenia, including astute astringent thrombocytopenia, accept been empiric afterwards administering of some ASOs. Individuals may be at added accident of bleeding complications. Renal toxicity has been empiric afterwards administering of some ASOs. SPINRAZA is present in and excreted by the kidney.
About BiogenAt Biogen, our mission is clear: we are antecedents in neuroscience. Biogen discovers, develops, and delivers common avant-garde therapies for bodies active with austere acoustic and neurodegenerative diseases. Founded in 1978 as one of the world’s aboriginal all-around biotechnology companies by Charles Weissman and Nobel Prize winners Walter Gilbert and Phillip Sharp, today Biogen has the arch portfolio of medicines to amusement assorted sclerosis; has alien the aboriginal and alone accustomed assay for analgesic able-bodied atrophy; and is focused on advancing neuroscience assay programs in Alzheimer’s ache and dementia, neuroimmunology, movement disorders, neuromuscular disorders, pain, ophthalmology, neuropsychiatry, and astute neurology. Biogen additionally articles and commercializes biosimilars of avant-garde biologics. We commonly column advice that may be important to investors on our website at www.biogen.com. To apprentice more, amuse appointment www.biogen.com and chase us on amusing media – Twitter, LinkedIn, Facebook, YouTube.
About Ionis Pharmaceuticals, Inc.Ionis is the arch aggregation in RNA-targeted biologic assay and development focused on developing drugs for patients who accept the accomplished unmet medical needs, such as those patients with astringent and attenuate diseases. Application its proprietary antisense technology, Ionis has created a ample action of first-in-class or best-in-class drugs, with over three dozen drugs in development.
SPINRAZA® (nusinersen) has been accustomed in all-around markets for the assay of analgesic able-bodied decline (SMA). Biogen is amenable for commercializing SPINRAZA. Drugs that accept auspiciously completed Phase 3 studies accommodate inotersen, an antisense biologic Ionis is developing to amusement patients with ancestral TTR amyloidosis (hATTR), and volanesorsen, an antisense biologic apparent by Ionis and co-developed by Ionis and Akcea Assay to amusement patients with either familial chylomicronemia affection or familial fractional lipodystrophy. Akcea, an associate of Ionis, is a biopharmaceutical aggregation focused on developing and commercializing drugs to amusement patients with austere cardiometaic diseases acquired by lipid disorders. If approved, volanesorsen will be commercialized through Ionis’ affiliate, Akcea. Volanesorsen filings for business approval accept been submitted in the U.S., EU and Canada. Inotersen is advanced against authoritative filings for business authorization. Ionis’ patents accommodate able and all-encompassing aegis for its drugs and technology. Added advice about Ionis is accessible at www.ionispharma.com.
Biogen Safe HarborThis columnist absolution contains advanced statements, including statements fabricated pursuant to the safe anchorage accoutrement of the Private Securities Litigation Reform Act of 1995 apropos to the abeyant benefits, safety, and adeptness of SPINRAZA, the after-effects of assertive real-world data, the cachet of Biogen’s accepted authoritative filings, Biogen’s affairs for added authoritative filings in added jurisdictions, and availability of accommodating admission and agreement pathways, which may alter on a country-by-country basis. These advanced statements may be accompanied by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “except,” “forecast,” “intend,” “may,” “plan,” “potential,” “possible,” “will,” and added words and agreement of agnate meaning. Biologic development and commercialization absorb a aerial amount of risk. You should not abode disproportionate assurance on these statements or the accurate abstracts presented.
These statements absorb risks and uncertainties that could account absolute after-effects to alter materially from those reflected in such statements, including afterwards limitation ambiguity of success in commercialization of SPINRAZA, which may be impacted by, amid added things, the akin of accommodation of healthcare providers to amusement patients, difficulties in accepting or changes in the availability of agreement for SPINRAZA, the capability of sales and business efforts, problems with the accomplishment action for SPINRAZA, the accident of adverse assurance contest and/or abrupt apropos that may appear from added abstracts or analysis; authoritative authorities may crave added advice or added studies, or may abort to accept or may adjournment approval of Biogen’s biologic candidates or amplification of artefact labeling; Biogen may appointment added abrupt hurdles which may be impacted by, amid added things, the accident of adverse assurance events, abortion to access authoritative approvals in assertive jurisdictions, or abortion to assure bookish acreage and added proprietary rights; artefact accountability claims; or third affair accord risks. The above sets alternating many, but not all, of the factors that could account absolute after-effects to alter from Biogen’s expectations in any advanced statement. Investors should accede this cautionary statement, as able-bodied as the accident factors articular in Biogen’s best contempo anniversary or anniversary address and in added letters Biogen has filed with the U.S. Securities and Exchange Commission. These statements are based on Biogen’s accepted behavior and expectations and allege alone as of the date of this columnist release. Biogen does not undertake any obligation to about amend any advanced statements, whether as a aftereffect of new information, approaching developments, or otherwise.
Ionis Pharmaceuticals’ Forward-Looking StatementThis columnist absolution includes advanced statements apropos Ionis’ cardinal accord with Biogen and the development, activity, ameliorative potential, assurance and commercialization of SPINRAZA. Any account anecdotic Ionis’ goals, expectations, banking or added projections, intentions or behavior is a advanced account and should be advised an at-risk statement. Such statements are accountable to assertive risks and uncertainties, decidedly those inherent in the action of discovering, developing and commercializing drugs that are safe and able for use as animal therapeutics, and in the endeavor of architecture a business about such drugs. Ionis’ advanced statements additionally absorb assumptions that, if they never actualize or prove correct, could account its after-effects to alter materially from those bidding or adumbrated by such advanced statements. Although Ionis’ advanced statements reflect the acceptable acceptance acumen of its management, these statements are based alone on facts and factors currently accepted by Ionis. As a result, you are cautioned not to await on these advanced statements. These and added risks apropos Ionis’ programs are declared in added detail in Ionis’ anniversary address on Anatomy 10-K for the year concluded December 31, 2016, and its best contempo anniversary address on Anatomy 10-Q, which are on book with the SEC. Copies of these and added abstracts are accessible from Ionis.
Ionis Pharmaceuticals™ is a brand of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™ is a brand of Ionis Pharmaceuticals, Inc. SPINRAZA® is a registered brand of Biogen.
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