The Swiss biotech, which is still at the seed stage of funding, could receive more than US $1 billion in combined payments from the US big pharma if co-developed products reach the clinic.
Haya’s approach focuses on the so-called ‘dark’ or non-protein coding genome. The company, which was founded in 2019, is looking for long non-coding RNAs (lncRNAs) that could be driving fibrotic disease and that could be targeted to create new treatments.
One way of targeting such RNA’s is through an allele-specific oligonucleotide (ASO) treatment. Haya’s lead candidate therapy (HTX-001) is an ASO targeting lncRNA Wisper, a driver of fibrosis in the heart, to treat nonobstructive hypertrophic cardiomyopathy. It is also working on a number of other undisclosed, early-stage treatments for lung, liver, pancreas, and kidney diseases, as well as cancer indications linked to the tumor microenvironment.
The new partnership adds to Lilly’s strong focus on developing drugs for metabolic conditions such as diabetes and obesity. Lilly has developed drugs for diabetes for some time and has enjoyed recent success with its blockbuster GLP-1 receptor agonist Mounjaro (tirzepatide) for treatment of obesity.
The collaboration between the two companies will make use of Haya’s platform technology to identify and target lncRNAs linked to obesity and other metabolic diseases. The company is able to focus on specific tissues and cells that could be linked to disease in its search for targets.
Haya is receiving an undisclosed upfront payment and equity investment from Lilly. If all possible pre-clinical, clinical and commercial milestones are met then it could be eligible for payments amounting to a total of US $1 billion.
Shining light on the dark genome
For a long time, most if not all non-coding DNA that did not lead to protein production in the body was considered to be ‘junk DNA’. However, in recent years it has increasingly become clear that not all non-coding DNA is junk and that much of it plays an important role in regulation of gene expression and cellular processes that can be important for disease initiation and prevention.
Haya is not the only company exploring this space. A number of biotech companies have recently been founded to try and investigate and exploit this relatively unknown area of the genome such as Enara Bio, Rome Therapeutics and Nucleome Therapeutics, among others.
Of course, the dark genome is implicated in a wide range of diseases and last week NextRNA and German big pharma Bayer signed a similar deal to the one signed by Lilly and Haya to develop small molecule drugs to target lncRNA’s linked to cancer.
"This partnership with Lilly demonstrates the significant advances we have made with our revolutionary regulatory genome RNA-guided platform and validates the potential of targeting lncRNA for chronic conditions," said Samir Ounzain, CEO, Haya Therapeutics, in a press statement.
"We look forward to working closely with Lilly, an established leader in developing treatments for metabolic diseases, to help bring patients novel disease-modifying therapeutics that could offer greater efficacy, safety and accessibility than currently available treatments."
