Ionis Pharmaceuticals is in focus

Biogen, Ionis Pharmaceuticals expand drug development collaboration

Biogen will pay Ionis $1B in cash

Biogen (BIIB) and Ionis Pharmaceuticals (IONS) announced they have expanded their strategic collaboration through a new ten-year collaboration agreement to develop novel antisense drug candidates for a broad range of neurological diseases.

This collaboration capitalizes on Biogen’s expertise in neuroscience research and drug development and Ionis’ leadership in RNA targeted therapies with the goal of developing a broad pipeline of investigational therapies.

Biogen says BAN2401 did not meet primary endpoint. Stockwinners.com
Biogen will pay Ionis $1B in cash

It builds upon a productive collaboration that produced SPINRAZA, the first and only approved treatment for patients with spinal muscular atrophy.

Under the terms of the collaboration, Biogen will pay Ionis $1B in cash, which will include $625M to purchase 11,501,153 shares of Ionis common stock at a price of $54.34 per share, at an approximately 25% cash premium, and a $375M upfront payment.

Biogen will have the option to license therapies arising out of this collaboration and will be responsible for their development and commercialization.

In addition, Biogen may pay milestone payments, license fees and royalties on net sales.

The companies plan to advance programs for a broad range of neurological diseases for which few treatment options exist today.

Disease areas include dementia, neuromuscular diseases, movement disorders, ophthalmology, diseases of the inner ear, and neuropsychiatry.

Biogen will have the first choice of neurology targets on which to exclusively collaborate with Ionis.

In this collaboration, Ionis will be responsible for the identification of antisense drug candidates based on selected targets, while Biogen will be responsible for and pay for non-clinical studies, clinical development, manufacturing, and commercialization. Biogen and Ionis expect the deal to close in the second quarter of 2018.

IONS closed at $45.85. BIIB closed at $266.02.


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Abeona Therapeutics granted Orphan Drug Designation

Abeona Therapeutics granted Orphan Drug Designation for ABO-202 in Europe

Abeona Therapeutics granted Orphan Drug Designation, Stockwinners
Abeona Therapeutics granted Orphan Drug Designation, 

Abeona Therapeutics (ABEO) announced that the European Medicines Agency Committee for Orphan Medicinal Products has granted Orphan Drug Designation for Abeona’s gene therapy program ABO-202 for the treatment of subjects with neuronal ceroid lipofuscinosis, also known as Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children.

ABO-202 is an adeno-associated virus developed to introduce a functional copy of the CLN1 gene into cells in order to restore the enzyme activity that is needed to break down certain lipopigment proteins that are deficient in patients with CLN1 disease.

Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children, Stockwinners
Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children

ABO-202 is anticipated to enter clinical trials in 2018. ABO-202 has been granted Orphan Drug and Rare Pediatric Disease Designations from the US Food and Drug Administration.

Preclinical data from the program were recently presented at the WORLDSymposium held in San Diego and an update will be presented at the American Society for Gene and Cell Therapy later this year.

Key findings reported included: The data demonstrate that a single intravenous, single intrathecal or combination therapy utilizing both administration routes of a self-complementary adeno-associated virus encoding the human CLN1 gene to CLN1 mice significantly increased their survival, improved behavior and reduced motor deficits.

Higher doses further improved these observations, suggesting that methods increasing total CNS exposure may be beneficial and provided some survival and behavioral benefit to symptomatic mice.

A combination approach delivering ABO-202 by both intravenous and intrathecal routes of administration further increased survival efficacy and improved potential treatment options for older animals with advanced disease manifestations.

ABEO closed at $20.05.


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