Octant, Inc. has received Orphan Drug Designation from the FDA for OCT-980, an investigational candidate intended for the treatment of retinitis pigmentosa (RP).
Let’s start with this company.
Founded in 2017 and headquartered in Emeryville, California, Octant is a clinical-stage biotechnology company “pioneering a new type of small molecule drug discovery” that modulates cellular mechanisms.
More specifically: The company is developing “correctors of protein molding diseases”—specialized small-molecule drugs that bind to malformed proteins to essentially restore their proper cellular function.
- Its target therapeutic areas: oncology, metabolism, immunology, and genetic diseases (such as RP).
And what technology is it utilizing to do this?
That would be the Octant Navigator, a proprietary platform built with three components designed to discover and develop therapeutics against complex cellular mechanisms in human cells:
- Generative biology: uses mammalian synthetic biology toolkits to rapidly design, build, and test cellular sensors that report on activities like signaling, expression, trafficking, stability, and interactions of disease target pathways
- Generative chemistry: a nano-scale automated synthesis platform that makes large libraries of related analogs rather than screening unrelated compounds
- These are tested direct-to-biology in iterative learning loops.
- Generative artificial intelligence (AI): machine learning and automation connect the two in weekly cycles, screening “tens of thousands” of new chemicals and converting millions of readouts into design decisions
Anything else?
Also worth mentioning: deep mutational scanning (DMS).
Referred to as “a new frontier for understanding protein function,” DMS uses that aforementioned generative biology platform to design, build, and screen “thousands to millions” of cell lines.
The intended result: to generate insights into a cell’s function.
- See here for more on how this works.
So how is Octant’s proprietary tech targeting RP?
To note: It’s specifically targeting rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP).
The company is using the Navigator to design small molecule correctors that block toxic accumulation of misfolded RHO in the retina.
- As Octant stated: “We’ve engineered human cells to read out on trafficking and biophysical properties of RHO mutations, generating millions of data points from each screen that guide drug design.”
As with the other rare disease programs in the company’s pipeline, the RP program applies multi-mutant approaches designed to “maximize the addressable patient population from the start” and target multiple mutations—with RHO-adRP its initial focus.
Is this where OCT-980 comes in?
Indeed. The “first-in-disease” oral small molecule is designed to:
- Correct protein misfolding for RHO gene mutations—the root case of RHO-adRP
- Restore proper cellular trafficking of RHO
Click here for how DMS is utilized to measure its OCT-980’s efficacy and potentiality.
Any clinical data on it yet?
Yes! Preclinical data, that is. Though we don’t have the details on those evaluations, Octant shared that OCT-980 demonstrated “strong efficacy and a favorable safety profile.”
Nice! So what’s the significance of this ODD?
Quick refresh: ODD is a special regulatory status granted by the FDA for certain investigational drug candidates with the potential for diagnosing, treating, or preventing so-called “orphan” (rare, life-threatening, or serious) diseases.
And what advantages come with this designation?
Candidates considered to be “orphan drugs” undergo the same scientific review process as any other drug seeking regulatory approval for commercial licensing in the United States.
However, they’re also qualified to receive:
- Tax credits for qualified clinical trials
- User fees exemption
- (Potentially) 7 years of market exclusivity following approval
Noted. And circling back to OCT-980—what’s next?
The oral therapy is currently undergoing a phase 1/2 clinical study (NCT07408232), in which investigators are evaluating a daily oral dosing among two groups: healthy volunteers and RHO-adrp patients.
The study is comprised of two parts:
- Phase 1a among healthy patients
- Phase 1b/2 designed as an open-label, multi-center multiple ascending dose escalation (MAD)
- Five cohorts (with five corresponding doses of OCT-980) are included in this portion
- Dosing began in September
See here for more on participant criteria and study design.
Do we know what outcome measures are?
The study has been described as designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of OCT-980 in patients, including measures of retinal structure and visual function.
However the lone primary outcome is the safety and tolerability of multiple doses of OCT-980 among patients with a genetic diagnosis of RHO-adRP.
- The timeframe: baseline to Week 60.
And any idea on when we may see results?
As we mentioned, the study kicked off this past March (with those healthy patient dosings) and dosed the first RHO-adRP patient in late September.
Per ClinicalTrials.gov, it’s expected to run until November 2028.