Biotechnology Events


Ionis Pharmaceuticals, Inc.

Partner : Biogen Idec.




Stanley T Crooke, MD, PhD, CEO, gave guidance for the timing of data-reads for three important assets. He stated, "In 2015, we completed a target of three different phase-III studies for three different diseases. We are moving closer to completing two phase-III trials for Nusinersen, our drug to treat infants and children with spinal muscular atrophy. At the same time, we are completing a phase-III study of IONIS-TTRRx in patients with peripheral neuropathy, a form of TTR amyloidosis and we are completing a phase-III trial of Volanesorsen in patients with FCS. We plan to have data from all four of these phase-III studies in the first half of 2017 and so the regulatory preparations and launch planning are well underway for all three of these potentially transformational drugs. As 2015 was an important year for phase-III assets; 2016 and early 2017 will be a pivotal time for these drugs and for Ionis as we and our partners prepare to submit marketing applications and launch these important new medicines."
Source: Q4 2015 earnings conference call, 2/25/16.


Isis Pharmaceuticals Earns $2.15M For Advancing ISIS-SMN Rx in Children with Spinal Muscular Atrophy

CARLSBAD, Calif., July 14, 2015 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it has earned a $2.15 million milestone payment from Biogen related to advancing the ongoing pivotal Phase 3 study (CHERISH) evaluating ISIS-SMNRx in children with spinal muscular atrophy (SMA). To date, Isis has generated more than $120 million in payments from Biogen related to the development of ISIS-SMNRx.

Isis Pharmaceuticals, Inc.
CHERISH, a Phase 3 study of ISIS-SMNRx, is a randomized, double-blind, sham-procedure controlled fifteen month study in approximately 120 children who are non-ambulatory with SMA between the ages of 2 to 12. The study will evaluate the efficacy and safety of 12 mg doses of ISIS-SMNRx with a primary endpoint of a change in the Hammersmith Functional Motor Scale-Expanded (HFMSE), a validated method to measure changes in muscle function in patients with SMA. Additional efficacy endpoints are also included in the study. For further study information, please visit and search for ISIS-SMNRx or the identifier number NCT02193074 or visit the ISIS-SMNRx study site at
Source: press release, 7/14/15.


Stanley T Crooke, MD, PhD, CEO, gave guidance for data from the ISIS-SMNRx program. He stated, "ISIS-SMNRx remains one of the most important and exciting drugs with which I have had the privilege of being associated. All of us are deeply committed to bringing benefit to patients with SMA. Of course we have a lot more work to do. The phase-III program is progressing nicely toward a 2016 or 2017 completion and data. We also look forward to updating on the results of the phase-II program a little later this year. Certainly as time passes, we remain very encouraged by the performance of ISIS-SMNRx."
Source: Q4 2014 earnings conference call, 2/27/15.


Isis Pharmaceuticals Initiates Phase 3 Study of ISIS-SMN Rx in Infants with Spinal Muscular Atrophy

-- ENDEAR Trial To Enroll 110 SMA Infants at Sites Globally

-- Dosage of First Infant Will Trigger $18 Million Milestone Payment

CARLSBAD, Calif., Aug. 1, 2014 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today the initiation of a pivotal Phase 3 study evaluating ISIS-SMNRx in infants with spinal muscular atrophy (SMA), the most common genetic cause of infant mortality. Isis plans to dose the first infant in this study within the next few weeks, at which time Isis will earn an $18 million milestone payment from its development partner, Biogen Idec. The Phase 3 study, ENDEAR, is the first of several planned studies in a broad and comprehensive late-stage clinical development program for ISIS-SMNRx. Isis plans to initiate a second pivotal study in children with SMA later this year.

Isis Pharmaceuticals, Inc.
"The successful advancement of ISIS-SMNRx from a preclinical drug candidate to late-stage studies within just a few years reflects the effectiveness of our strategic alliance with Biogen Idec and the benefit of working closely together with combined expertise, and the support from the SMA community. As we continue development of ISIS-SMNRx and initiate the two pivotal studies in infants and children with SMA, we are in the planning stages for clinical studies in additional patient populations," said B. Lynne Parshall, chief operating officer at Isis. "The clinical and preclinical data we have generated to date, including data in multiple open-label clinical studies, across multiple measures with ISIS-SMNRx, support the initiation of these studies, which fully assess the safety and efficacy benefits of this experimental treatment."

"Families of SMA is pleased that Isis is advancing to the next phase of clinical trials for ISIS-SMNRx. Controlled trials are the gold standard in proving the safety and efficacy of any drug," said Kenneth Hobby, president of Families of SMA. "SMA is a devastating disease with no current therapeutic options. Families of SMA applauds Isis for progressing its development program in an expedient manner, and looks forward to additional trials in patients with SMA beginning later in 2014."

ENDEAR, a Phase 3 study of ISIS-SMNRx, is a randomized, double-blind, sham-procedure controlled thirteen month study in approximately 110 infants diagnosed with SMA. The study will evaluate the efficacy and safety of a 12 mg dose of ISIS-SMNRx with a primary endpoint of survival or permanent ventilation. Additional efficacy endpoints are also included in the study. For further study information, please visit and search for ISIS-SMNRx or the identifier number NCT02193074 or visit the ISIS-SMNRx study site at
Source: press release, 8/01/14.


A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy
Estimated Enrollment: 111
Study Start Date: July 2014
Estimated Study Completion Date: July 2017
Estimated Primary Completion Date: June 2017 (Final data collection date for primary outcome measure)
Source: clinical


Compound/DeviceSpecialtyIndicationCompound ClassTarget
Nusinersen (IONIS-SMNRx)NeurologySpinal Muscular Atrophyantisense drugSMN-2 gene

Mechanism of action: Nusinersen (IONIS-SMNRx) is an antisense drug designed to treat spinal muscular atrophy. SMA occurs from a genetic deletion of the survival motor neuron 1 (SMN-1) gene. SMN-1 produces most of the SMN protein necessary for normal motor function. The severity of SMA is determined by the amount of SMN protein in the cell. Humans also have one or more copies of a related gene, SMN-2, which produces a small fraction of SMN protein due to inefficient RNA splicing. ISIS-SMNRx increases the production of SMN protein by modulating the splicing of SMN-2, thereby compensating for the underlying genetic defect.

Phase of Development: III

Event Type: Data: Phase III trial results (interim)

Dates: 2016-07-01 - 2016-09-30



Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy
— Biogen Intends to File Marketing Applications for Nusinersen with Regulatory Authorities in the Coming Months —

— Biogen Exercises Option to Develop and Commercialize Nusinersen Globally —

— Companies to Host Webcast Today at 9:00 a.m. EDT to Provide Program Update —

CAMBRIDGE, Mass. & CARLSBAD, Calif.--(BUSINESS WIRE)--Aug. 1, 2016-- Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) today announced that nusinersen, their investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint pre-specified for the interim analysis of ENDEAR, the Phase 3 trial evaluating nusinersen in infantile-onset (consistent with Type 1) SMA. The analysis found that infants receiving nusinersen experienced a statistically significant improvement in the achievement of motor milestones compared to those who did not receive treatment. Nusinersen demonstrated an acceptable safety profile in the trial. As a result of these findings, Biogen has exercised its option to develop and commercialize nusinersen globally and paid Ionis a $75 million license fee. Biogen will initiate regulatory filings globally in the coming months.

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"We are grateful to the families participating in the clinical trials, who continue to inspire us. We want to thank them, along with the investigators who have worked tirelessly on this program and the broader SMA community, for their partnership. Without their contributions, we would not be here today," said Alfred Sandrock, M.D., Ph.D., executive vice president and chief medical officer at Biogen. "We share the community's sense of urgency as we strive to bring the first treatment for SMA, the leading genetic cause of infant mortality, to families facing this devastating disease. We remain committed to understanding the potential of nusinersen in the broader SMA population and will continue to focus on the rapid completion of our ongoing studies."

Based on the results of the pre-specified interim analysis, the ENDEAR study will be stopped and participants will be able to transition into the SHINE open-label study in which all patients receive nusinersen. Data from the other endpoints of ENDEAR will be analyzed when the full data set is available. Results will be presented at future medical congresses. Additionally, participants enrolled in the sham-controlled arm of EMBRACE, a Phase 2 study which also included infantile-onset patients, will have the opportunity to receive nusinersen.

The other studies in the nusinersen program, including CHERISH (later-onset consistent with Type 2) and NURTURE (pre-symptomatic infants), will continue as planned in order to collect the data to demonstrate the safety and efficacy of nusinersen in these populations.

"We are hopeful that nusinersen, if approved, will make a meaningful difference in the lives of patients and families affected by SMA. We look forward to working with Biogen on completing the clinical program and preparing for what we hope is a positive regulatory review," said B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals. "Nusinersen is the first antisense drug from our neurological disease franchise to advance to regulatory review, and it illustrates the potential of our antisense technology to address severe diseases that other therapeutic modalities are unable to address adequately."

Biogen is working to open a global expanded access program (EAP) for eligible patients with infantile-onset SMA (consistent with Type 1) in the coming months. The EAP can be initiated at existing nusinersen clinical trial sites in countries where EAPs are permitted according to local laws and regulations, can be operationalized, and where there is a path that can support long-term availability of nusinersen. Once the EAP is operational and required local approvals are in place, individual participating sites may start enrollment after they have transitioned ENDEAR study participants to the open-label extension study.

"Today is a hopeful day for the SMA community, which has worked tirelessly to support research and development for this terrible disease. Many of our families have participated in this and other clinical trials in order to advance our understanding of SMA. We are excited about reaching this important milestone, and the opportunity these results create to potentially bring the first treatment option for SMA to patients and families. We will continue to relentlessly support research into SMA until we have therapies for all and, ultimately, a cure," commented Kenneth Hobby, President, Cure SMA.

Biogen is now responsible for all nusinersen development, regulatory and commercialization activities and costs. Ionis will complete the Phase 3 studies and work with Biogen on regulatory filings. The two companies will also work together to transition the clinical programs that Ionis is conducting to Biogen. Ionis is eligible to receive tiered royalties on any potential sales of nusineren up to a percentage in the mid-teens, in addition to up to $150 million in milestone payments based on regulatory approvals.


The companies will host a live webcast to discuss the results of the Phase 3 ENDEAR interim results for nusinersen today, August 1, 2016, from 9:00 to 9:30 a.m. EDT. Participants may access the webcast through the Investors section of or Following the live webcast, an archived version of the call will be available at the same URLs for one month.
Source: press release, 8/01/16.