Amyotrophic lateral sclerosis — or ALS — is a rare, rapidly progressing and fatal neurodegenerative disorder that affects approximately 55,000 people globally, explains biotechnology sector expert John McCamant, editor of The Medical Technology Stock Letter.
Ionis Pharmaceuticals (IONS) recently announced the initiation of a Phase III trial of ION363 in patients with ALS with mutations in the fused in sarcoma gene (FUS). Patients with a mutation in the FUS gene develop a rare form of ALS, referred to as FUS-ALS, which is the most common cause of juvenile-onset ALS.
There is substantial evidence that mutations in the FUS gene are responsible for a toxic gain of function that can lead to rapid, progressive loss of motor neurons in patients with FUS-ALS. ION363 is an investigational antisense medicine targeting the FUS RNA to reduce the production of the FUS protein.
Antisense-mediated reduction of mutant FUS protein in a FUS-ALS mouse model prevents motor neuron loss. By targeting the root cause of FUS-ALS, ION363 has the potential to reduce or prevent disease progression in FUS-ALS patients.
FUS-ALS is the third most common genetic cause of ALS. The Phase III trial of ION363 is a global, multi-center study in up to 64 patients.
Part one of the trial will consist of patients randomized to receive a multi-dose regimen of ION363 or placebo for 29 weeks, followed by part two, which will be an open-label period in which all patients in the trial will receive ION363 for 73 weeks.
On a financial note, IONS it is raising $500 million of Convertible Senior Notes due 2026 in a private placement to qualified institutional buyers. It plans to use some of the proceeds to repurchase for cash some of its 1% Convertible Senior Notes due 2021 in private deals. It also plans to use some of the net proceeds to pay the cost of several convertible note hedge transactions.
IONS also announced an agreement with Progenity to evaluate the safety, tolerability and performance of their Oral Biotherapeutics Delivery System (OBDS) for oral delivery of antisense oligonucleotides.
The OBDS is an ingestible capsule based on a needle-free technology designed by Progenity to enable delivery of a drug formulated in a solution directly into the tissues of the small intestine, where it can be absorbed systemically. Oral delivery has the potential to open up huge additional markets for IONS platform.
Overall, the ALS trial has significant potential to address a large and sick population of patients and targeting FUS patients may be a smart way to finally create an effective ALS treatment. Cleaning up the balance sheet and retiring upcoming debt makes solid financial sense.
Lastly adding OBDS for oral delivery demonstrates management’s ability to create long-term value by addressing new and large drug marker opportunities.
