AveXis builds out SMA program with new studies

Dive Brief:

AveXis Inc. will add three more clinical trials to the slate of research planned for its lead candidate, AVXS-101, a potential treatment for spinal muscular atrophy (SMA).
The trials include STR1VE EU, a pivotal study of AVXS-101 in patients with Type 1 SMA patients that will take place in Europe; SPRINT, a multi-national study of AVXS-101 in pre-symptomatic patients with Type 1, 2 or 3 SMA; and REACH, another multi-national study of AVXS-101 that will be open to pediatric patients with Type 1, 2 or 3 SMA who do not qualify for other investigations of the drug.
In addition to new R&D efforts, AveXis disclosed on Monday that researchers have dosed the first patient in the ongoing Phase 1 STRONG trial, which is testing AVXS-101 in Type 2 SMA patients. The Chicago-based biotech has also been working on the STR1VE pivotal trial of AVXS-101 in Type 1 SMA patients, a study expected to read out in 2020.

Dive Insight:

Biogen Inc. currently controls the SMA market with Spinraza (nusinersen), the first Food and Drug Administration-approved treatment for the rare neuromuscular disease. From July through September, the drug brought in $271 million — putting it on a good path to reach blockbuster status in the coming years.

Despite its impressive trajectory, not everyone has been so keen on Spinraza. Some major insurers have limited coverage of the drug due to its high price tag. Biogen investors, though initially pleased with the Spinraza's performance, also became more apprehensive when the company reported essentially flat U.S. sales of the drug during the third quarter.

Competition could pose another threat to Spinraza's present standing.

PTC Therapeutics Inc. and Roche AG are developing a small molecule modifier of Survival Motor Neuron 2 splicing that recently notched positive preliminary results in a Phase 2 trial evaluating it in patients with Type 2/3 SMA. The oral administration of PTC and Roche's drug is particularly advantageous, given Spinraza is given via injection.

From its end, AveXis sees great promise in AVXS-101 across a variety of SMA types, which refer to when the disease symptoms arise. "[O]ur expanded clinical development program is designed to evaluate the impact of AVXS-101 in a broader set of SMA patients," Sukumar Nagendran, chief medical officer at AveXis, said in a Jan. 16 statement.

Jacob Bell / BioPharma Dive, market data

Over the first nine months of 2017, AveXis shelled out just shy of $100 million on research and development — more than double what it spent during the same period in 2016.

AveXis intends to spend between $225 million and $270 million to fund research, manufacturing and clinical activities on its SMA program, as well as preclinical programs for Rett syndrome and ALS, according to a company spokesperson. Another roughly $140 million to $175 million will go toward pre-commercial activities.

AveXis anticipates the STR1VE EU and SPRINT trials to initiate in the first half of this year, while the REACH study should kick off in either the fourth quarter or early 2019.