Ventus Therapeutics has dosed the primary participant in its Phase 1 trial testing VENT-02, a novel oral remedy that options an inhibitor that reaches the mind and targets NLRP3 — a protein that’s linked to mind irritation in Parkinson’s illness.
The trial goals to evaluate the security and tolerability of VENT-02, in addition to its results on the physique, via each single and escalating a number of doses. Initial outcomes are anticipated within the first half of 2024, the corporate stated in a press launch.
“This trial marks a new chapter for Ventus as we become a clinical-stage organization,” stated Marcelo Bigal, MD, PhD, the president and CEO of Ventus.
Phase 1 trial to check security, tolerability of novel Parkinson’s remedy VENT-02
The NLRP3 protein is discovered inside cells, the place it really works as sensor for hazard indicators, corresponding to pathogens, or disease-causing brokers, and environmental triggers. Once a risk is detected, it triggers the formation of an inflammasome, a molecular advanced concerned in inflammatory responses.
While these processes assist the immune system reply when there’s a drawback, in sure conditions the NLRP3 protein can change into too lively and trigger extra irritation than wanted. That, in flip, can result in well being points like mind irritation in circumstances like Parkinson’s, Alzheimer’s illness, and amyotrophic lateral sclerosis, extra generally often known as ALS.
“NLRP3 is a key driver of many neuroinflammatory diseases,” stated Mike Crackower, PhD, Ventus’ chief scientific officer.
The firm is creating quite a few therapeutic candidates that block the exercise of NLRP3. According to Ventus, VENT-02 has proven a robust capability to inhibit NLRP3 in cells and blood from completely different animals.
[VENT-02] has best-in-class potential primarily based on its distinctive construction, distinctive mind penetration, and beautiful efficiency.
VENT-02 additionally confirmed a superb capability in a number of preclinical fashions to penetrate the mind — key in treating irritation — the corporate famous.
That’s an enormous hurdle and a shortcoming for a lot of therapies, that are discovered to be unable to cross the blood mind barrier, often known as the BBB. That barrier is a semi-permeable membrane that shields the central nervous system, comprised of the mind and spinal wire, from basic blood circulation and potential threats.
“Our extensive characterization of VENT-02 in multiple disease-relevant preclinical models has de-risked our disease selection process, and we believe VENT-02 has the potential to treat patients across a wide range of neuroinflammatory diseases, including Parkinson’s, Alzheimer’s, and refractory epilepsy, as well as systemic diseases,” Crackower stated.
Bigal stated VENT-02 “has best-in-class potential based on its unique structure, exceptional brain penetration, and exquisite potency.”
“We are bringing a highly differentiated NLRP3 inhibitor into the clinic with VENT-02,” Bigal stated.
“VENT-02 has demonstrated extraordinary safety margins — among the highest I have seen in 20-plus years of developing drugs. These benefits allow us to design an incredibly innovative clinical development plan for VENT-02 and to fully explore the potential impact of NLRP3 inhibition across multiple patient populations suffering from neuroinflammatory diseases,” Bigal added.
The Michael J. Fox Foundation in April awarded Ventus a$150,000 grant to develop a positron emission tomography (PET) tracer for NLRP3. The tracer is anticipated to assist reveal NLRP3’s position in nerve-related irritation, in line with the corporate.
