Atalanta aims for clinical trial of RNAi therapy against toxic protein
Raises $97M in financing to advance its RNA interference candidate, ATL-101
Atalanta Therapeutics has raised $97 million to support planned Phase 1 clinical trials of its experimental RNA interference (RNAi)-based therapies for Huntington’s disease and a genetic form of epilepsy.
Each treatment aim to silence the activity of genes that underlie these diseases. Atalanta plans to submit investigational new drug (IND) applications to the U.S. Food and Drug Administration this year seeking approval to launch the trials in the country.
The company’s Series B financing round was reported to raise more than the targeted financial goal.
“This Series B will support a path to the clinic for two programs for serious neurological diseases that today lack disease-modifying therapies … and will anchor our growing franchise of investigational medicines for Huntington’s disease,” Alicia Secor, president and CEO of Atalanta, said in a company press release. “We are diligently progressing these programs toward IND submissions this year so that we can start our Phase 1 trials and reach patients who are waiting.”
RNAi therapies aim to hamper protein production by regulating gene activity
Atalanta is developing a class of medications that work to silence disease-related gene activity through RNAi, a process the body naturally uses to regulate gene activity.
Under this process, information in DNA used to make a working protein is first converted into a template molecule called messenger RNA. In RNAi, short fragments of RNA — a type of genetic material — bind to messenger RNA and cause it to be broken down, preventing protein production.
Atalanta’s therapeutic candidates are lab-made molecules called divalent small interfering RNAs (di-siRNA) that are designed to mimic this process as a way of turning off genes associated with neurological diseases.
“RNA interference” the company states on its website, “is a Nobel prize-winning discovery that allows for targeted silencing of disease-causing genes.” But as a form of therapy, its use has been limited by “the difficulty of achieving distribution throughout the central nervous system,” the brain and spinal cord.
In preclinical work, Atalanta’ reports that its candidates “show unparalleled distribution throughout the brain and spinal cord as well as substantial persistence in the brain.”
Huntington’s is caused by mutations in the HTT gene that give rise to an abnormal version of the huntingtin protein, which toxically accumulates in nerve cells to damage them and cause them to die off.
Drop in huntingtin protein levels seen in brains of primates given ATL-101
Atalanta’s RNAi-based therapeutic candidate, called ATL-101, is designed to silence HTT gene activity, thereby lowering production of the huntingtin protein.
Preclinical studies involving nonhuman primates showed that a single dose of ATL-101, delivered directly into the spinal canal, led to a strong reduction in huntingtin levels — including in deep brain regions implicated in Huntington’s — with effects that lasted for at least six months, the company reported.
ATL-101 also was well tolerated, with no significant safety signals observed.
Atalanta’s other potential Phase 1 trial candidate is ATL-201, which aims to silence the activity of the KCNT1 gene that’s associated with a severe form of childhood epilepsy.
While ATL-101 and ATL-201 move toward clinical studies in people, the company reports that two other RNAi-based therapies for Huntington’s disease are in discovery stages.
The recent funding round was co-led by EQT Life Sciences and Sanofi Ventures, with participation from investors that include RiverVest Venture Partners and Novartis Venture Fund.
“Atalanta’s di-siRNA technology has shown promising ability to durably silence disease-promoting genes throughout previously inaccessible regions of the brain and spinal cord — opening a wide range of treatment possibilities for devastating neurological diseases,” said Arno de Wilde, MD, PhD, managing director of EQT Life Sciences.
de Wilde, along with Jason Hafler, PhD, managing director of Sanofi Ventures, and Niall O’Donnell, PhD, managing director of RiverVest Venture Partners, were appointed to Atalanta’s board of directors.
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