Date: 2017-07-17
Type of information: Milestone
Compound: antisense therapies for neurological diseases
Company: Biogen (USA - MA) Isis Pharmaceuticals , now Ionis Pharmaceuticals (USA - CA)
Therapeutic area: Neurological diseases - Neuromuscular diseases - Neurodegenerative diseases
Type agreement: development
Action mechanism:
- antisense drug. Antisense technology is designed to alter a gene's function, silencing a mutation or activating a gene to compensate for an underlying genetic defect. When the genetic sequence of a gene is known to cause a disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that binds to the messenger RNA (mRNA) produced by that gene and effectively turn that gene "on" or "off."
Disease: neurological diseases, neuromuscular diseases
Details:
- • On September 9, 2013, Biogen Idec and Isis Pharmaceuticals announced that the companies have entered into a broad, multi-year collaboration to leverage antisense technology to advance the treatment of neurological diseases. The agreement combines Biogen Idec's expertise in neurology with Isis' leadership in antisense technology to develop novel therapies to treat neurological disorders. This is the fourth collaboration between the two companies in the last two years. As part of the six-year research collaboration, Biogen Idec will gain exclusive rights to the use of Isis' antisense technology to develop therapies for neurological diseases.
- Biogen Idec and Isis will work closely together to select and validate neurological disease targets and conduct drug discovery activities with the goal of identifying clinical development candidates. Isis will be primarily responsible for drug discovery and early development of antisense therapies, while Biogen Idec will be responsible for the creation and development of small molecule treatments and biologics. In each case, Biogen Idec will determine the molecular modality best suited for advancement through clinical development. Biogen Idec will also be responsible for later stage development and commercialization of all drugs arising from the collaboration.
- This collaboration follows three previous collaborations between Biogen Idec and Isis. In the first half of 2012, Isis and Biogen Idec established collaborations to develop antisense drugs to treat spinal muscular atrophy and myotonic dystrophy type 1. Later that same year, Isis and Biogen Idec entered into a third collaboration to discover and develop antisense drugs against three novel targets for neurological disorders.
Financial terms:
- Under the terms of this agreement, Biogen Idec will make an upfront payment of $100 million. Isis is eligible to receive milestone payments, license fees and royalty payments for all treatments developed through this collaboration, with the specific amount dependent upon the modality of the molecule advanced by Biogen Idec. In the case of antisense molecules, the milestone payments could be as much as $220 million, plus additional amounts related to the cost of clinical trials conducted by Isis under the collaboration.
Latest news:
- • On July 17, 2017, Ionis Pharmaceuticals announced that it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. Ionis will continue to evaluate this newest target with the goal of advancing this program into development.
- Under this collaboration, Ionis developed and licensed Spinraza® to Biogen. This antisense drug is now approved in the U.S., EU, Japan and Canada for the treatment of patients with spinal muscular atrophy. Under this collaboration, Ionis and Biogen are also developing IONIS-SOD1Rx to treat amyotrophic lateral sclerosis, IONIS-MAPTRx to treat Alzheimer's Disease, and IONIS-BIIB5Rx, IONIS-BIIB6Rx and IONIS-BIIB7Rx to treat undisclosed neurodegenerative diseases.
- • On February 2, 2017, Ionis Pharmaceuticals announced it has earned a $5 million milestone payment from Biogen associated with the validation of an undisclosed neurological disease target. For this newest target, Ionis will continue to evaluate the target with the goal of advancing this program into development.
- • On March 31, 2016, Ionis Pharmaceuticals announced that it has earned a milestone payment of $7.5 million from Biogen for advancing nusinersen in the open-label extension study, SHINE. The SHINE study was initiated in October 2015 to provide nusinersen to infants and children with spinal muscular atrophy (SMA) who have completed their participation in the Phase 3 ENDEAR and CHERISH studies and are eligible to participate in SHINE.
- • On July 14, 2015, Isis Pharmaceuticals announced 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.
- • On April 30, 2015, Isis Pharmaceuticals announced it has earned a $10 million milestone payment from Biogen associated with the validation of an undisclosed target to treat a neurological disorder.
- • On June 17, 2014, Isis Pharmaceutical announced it has earned a $10 million milestone payment from Biogen Idec associated with the validation of an undisclosed target to treat a neurological disorder. This is the first milestone payment under Isis' and Biogen Idec's 2013 strategic collaboration to advance the treatment of neurological disorders.
Is general: Yes
