Israeli startup ProteKt Therapeutics is developing a drug to treat early stages of Alzheimer’s by targeting an enzyme that is believed to play a part in the development of the devastating neurodegenerative disease that causes memory loss and the inability to function.
The startup hopes that by inhibiting the enzyme, it will be able to bring about cognitive improvements in patients and slow the progress of the disease. It also hopes that the same treatment, when developed, could help with other neurodegenerative diseases such as ALS, Huntington and Parkinson’s.
The company was founded by the Carmel-Haifa University Economic Corporation and the Carmel Innovations Fund, based on the research of Prof. Kobi Rosenblum of the University of Haifa and was part of the FutuRx biotech incubator.
“It has been shown by research papers done by the inventor, Prof. Kobi Rosenblum, and other scientists that inhibiting this enzyme, called PKR, can lead to cognitive improvement,” said Yotam Nisemblat, the CEO of the Ness Ziona-based firm, in a phone interview.
Everyone has the enzyme, he explained, but people with Alzheimer’s were found to have elevated levels, or “over-activation,” of PKR. “Inhibiting” the enzyme, he said, can bring it back to its normal level of functioning.
Using a robotic and computerized process to sift through millions of molecules, ProteKt has identified a series of 150 small synthetic compounds that could help inhibit the enzyme.
After testing, “we identified several molecules that could be inhibitors of the enzyme,” Nisemblat said. “We showed that the compounds we identified are beneficial on Alzheimer’s models in the lab.”
The compounds were found to be able to reduce the neuro-inflammatory markers that are typical of Alzheimer’s and protect neurons in the brain from the toxicity of amyloid betas, peptides made up of 36-43 amino acids that are the main components of amyloid plaques found in the brains of people with Alzheimer’s.
The compounds, Nisemblat added, were also found to have the ability, in the lab, to improve critical elements of the neuronal functionality of the cells, indicating their potential to improve cognition.
In November the firm announced it had raised $3.6 million from investors including Quebec’s Fonds de solidarité FTQ fund and Korean pharma firm Bukwang Pharmaceutical, which joined existing investors FutuRx, OrbiMed, Johnson & Johnson Innovation – JJDC, Inc., Takeda Ventures, Inc. and RM Global Partners (RMGP) BioPharma Investment Fund for a stake in the firm.
Proceeds from the financing will be used primarily to help the firm whittle down the group it had selected and identify the most effective molecule, further improve it and make sure it is “selective, stable and soluble” — all factors needed when developing a drug.
“We will focus on developing one molecule” out of the many identified, Nisemblat said.
The firm also hopes to prove that the compound can be effective in animals and not just in the lab, and that it has the ability to enter the brain via the bloodstream by crossing the blood-brain barrier, a semipermeable border that separates the circulating blood from the brain and other fluids in the central nervous system.
Nisemblat is aware that the way ahead is long and precarious and because there is no clear, single cause of Alzheimer’s disease there is also “no single target” to aim at in trying to beat the illness.
“The traditional treatments are not beneficial,” he said. The industry has been seeking new ways to slow down the illness’s progression and halt patents’ deterioration.
“We believe our new mechanism could do that,” he said. “Development will take some time — we are only at the beginning of our development road — but we believe that targeting the PKR mechanism will have a significant effect on disease progression and magnitude.”
The financing will also enable the company reach a development stage, after which it will be able to raise additional funds to undertake clinical trials with patients, he said.
The FutuRx biotech incubator was set up through a partnership between Johnson & Johnson Innovation, Takeda Pharmaceutical Company, OrbiMed Partners Israel, and the Israel Innovation Authority, with the aim of fostering early stage innovation and creating breakthrough medications that can transition from the lab to the market.
The Fonds de solidarité FTQ’s investment in ProteKt is the fund’s first in the field of neurodegenerative diseases such as Alzheimer’s, said Didier Leconte, vice president for Investments, Life Sciences and Funds-of-Funds at the Fonds, in a statement announcing the funding in November.