New chapter for neurodegenerative diseases unfolds

Roche and reMYND, developer of treatments against protein misfolding disorders, jointly pursue disease-modifying programs for Parkinson’s and Alzheimer’s diseases

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LEUVEN, Belgium—As companies engaged in neurodegenerativedisease research pack their bags and head to San Diego for this year's Societyfor Neuroscience annual meeting, one biotech company will be celebrating animportant show-related milestone. Two years ago, Belgium-based biotech reMYNDmet with researchers at Swiss pharma Roche at the show to discuss its platformof compounds that inhibit disease progression in Alzheimer's and Parkinson'sdiseases, diabetes and other protein misfolding disorders.
Now, as the companies prepare to head back to the annualconference, they are partners, having just announced that they arecollaborating on the development of two of reMYND's preclinical, small-moleculeprograms:
One that inhibits α-synuclein neurotoxicity in Parkinson's disease,and a second program that inhibits tau neurotoxicity in Alzheimer's disease.
According to the companies, what makes these programs uniquein the field of Alzheimer's and Parkinson's disease research—and potentiallyvery lucrative for both partners—is that they are considered disease-modifyingand could slow down neurodegeneration in patients with these devastatingprogressive conditions, while most currently available treatments for thesediseases treat only their symptoms.
"Being disease-modifying, these programs have the potentialto delay symptoms or aggravation of symptoms—that gives them blockbusterpotential because in the research field, there is nothing disease-modifyingright now," says Koen De Witte, managing director of reMYND.
In fact, reMYND was one of the first companies to embraceand pursue this approach, which is increasingly becoming en vogue in the field of central nervous system diseaseresearch, De Witte notes. The company was founded in 2001 as a spinoff of theUniversity of Leuvin and immediately focused on developing treatments forprotein misfolding disorders through two independently managed business units,one an in vivo contract researchorganization (CRO) and the other a drug discovery and development unit.
According to De Witte, this strategy put reMYND several years ahead of itscompetition, as pharmas pursuing Parkinson's disease treatments have focusedmostly on symptomatic treatments, while in Alzheimer's disease, tau has onlyrecently garnered researcher interest.
"We think this was a pivotal consideration by Roche, as theywere looking for neurodegenerative disease modifiers, and not for symptomatictreatments," he says. "Our programs really played into their sweet spot, particularlywith regard to Alzheimer's. They are also very complimentary, as Rochecurrently has no top program or disease-modifying program in Parkinson's."
Under the agreement, Roche and reMYND will work together toprogress the programs toward clinical studies. Roche will provide input intochemistry, lead optimization and preclinical development, while reMYND willcontinue to conduct non-clinical pharmacology studies and further elucidate theunderlying molecular mechanisms. Roche will be responsible for all clinicaldevelopment and worldwide commercialization.
While the companies did not disclose the specific financialterms of their agreement, they say that reMYND could receive "over half abillion Euros in milestone payments," as well as "additional FTE payments androyalties on resulting net sales potentially reaching a double-digit level."
According to the latest statistics from the World HealthOrganization, there are currently around 35 million people diagnosed withAlzheimer's disease. Parkinson's disease is the most common motor disorder,typically affecting patients over the age of 65 and approximately 0.1 percentto 0.2 percent of the Western population.
Commenting on the agreement, Dr. Luca Santarelli, head ofRoche CNS, said in a statement, "The addition of these programs strengthens andcomplements our existing research. We are excited to have licensed these novelcompounds for our CNS pipeline because we believe that they offer a uniqueapproach to combat Parkinson's and Alzheimer's disease. We are committed tobringing new drugs to the patients that suffer from these devastatingneurodegenerative diseases."
Roche and Aileron in drug development pact
BASEL, Switzerland—In September, Roche announced a secondpartnership deal with U.S.-based biopharmaceutical firm Aileron Therapeutics toproduce a novel class of drugs to treat diseases such as inflammation andmetabolic ailments.
According to Roche, the deal speeds up recent efforts tobroaden its R&D pipeline by entering into licensing agreements andacquisitions. The deal with Aileron should help Roche develop a new class ofmedicines that are based on the U.S. firm's stapled peptides technology, whichallows for a different treatment of diseases and could speed up the developmentof drugs for illnesses for which there are yet no or ineffective medicines.
As part of the transaction, closely held Aileron may receiveup to $1.1 billion in milestone payments and fees, if Roche and the Cambridge,Mass.-based firm succeed in developing drug candidates against five targetareas, including oncology, virology, inflammation, metabolism and the centralnervous system.
Aileron's stapled peptides target the intracellularinteractions between proteins.
According to the company, unlike regularpeptides that dissolve when they enter into a cell, Aileron's stapled peptidespromise to remain intact and could thus be used to treat diseases moreeffectively as the peptides can become active within an affected cell,increasing treatment chances.

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