Agreements

Date: 2017-01-05

Type of information: Licensing agreement

Compound: Spinal Muscular Atrophy (SMA) programme

Company: PTC Therapeutics (USA - NJ) Roche (Switzerland) SMA Foundation

Therapeutic area: Neuromuscular diseases - Rare diseases

Type agreement: licensing

Action mechanism:

Disease: spinal muscular atrophy

Details:

• On November 29, 2011, Roche, PTC Therapeutics and the SMA Foundation, have announced a licensing agreement for PTC's Spinal Muscular Atrophy (SMA) programme. PTC Therapeutics' programme has been developed in partnership with the SMA Foundation, which will remain active in the collaboration. SMA is caused by a missing or defective SMN1 gene, which results in reduced levels of the survival motor neuron (SMN) protein. The compounds in PTC's research treat the underlying cause of the disorder and demonstrate increases in SMN levels in nervous system, muscles and other tissues in SMA models. SMA is a rare disorder and could be eligible for orphan status by regulatory authorities, thereby potentially reducing the time needed for a drug to reach patients. Roche and PTC announced their first collaboration in September of 2009 for the development of orally bioavailable small molecules utilizing PTC's technology called Gene Expression Modulation by Small-molecules (GEMS™). The SMA programme has been developed by PTC utilizing a different scientific approach than GEMS, called alternative splicing.

Financial terms:

Under the terms of the agreement, Roche gains an exclusive worldwide license to PTC's SMA programme, which includes three compounds currently in preclinical development, as well as potential back-up compounds. PTC receives $30 million as an upfront payment, up to $460 million upon successful completion of certain development and commercialization milestones, and up to double-digit royalties on commercial sales. Development will be overseen by a joint steering committee comprised of members from Roche, PTC and the SMA Foundation.

Latest news:

• On January 5, 2017, PTC Therapeutics announced that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) initiated a clinical study in infants with Type I SMA. The study, named FIREFISH, will investigate the safety, tolerability and efficacy of RG7916 in babies aged 1 to 7 months.
• On October 20, 2016, PTC Therapeutics announced that its joint development program in Spinal Muscular Atrophy (SMA) with Roche and the SMA Foundation (SMAF) initiated a Phase 2 SUNFISH study in pediatric and adult Type 2/3 SMA patients. A trial in Type I SMA patients, FIREFISH, is planned to initiate in the coming months. FIREFISH is also planned to be a two-part study.
• On January 22, 2014, PTC Therapeutics, the SMA Foundation, and Roche have announced that their joint research program in Spinal Muscular Atrophy (SMA) has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers.
• On August 8, 2013, PTC Therapeutics has announced the selection of a development candidate in its spinal muscular atrophy (SMA) collaboration with Roche and the SMA Foundation. The achievement of the milestone triggers a $10 million payment to PTC from Roche. PTC may receive up to an additional $450 million upon successful completion of other development and commercialization milestones, plus tiered royalties on worldwide net product sales.