DISEASE TARGETS

PharmaIN is developing peptide-based therapeutics to address critical unmet medical needs in conditions that affect relatively small patient populations. Longer-term, our proprietary capabilities to modify peptides have potential applications in a wide range of therapeutic areas.

Our most advanced development programs are focused on liver cirrhosis and oncology. In liver cirrhosis, which is typically caused by alcoholism, hepatitis B and C, and increasingly non-alocholic steatohepatitis (NASH) due to fatty lever disease linked to obesity and diabetes, the initial disease target is refractory ascites.

In oncology, our initial focus is on developing immunological drugs to treat solid tumor cancers. The development status of our lead new drug candidates can be seen in our pipeline, for more information about the conditions we’re targeting, click on the buttons below:

 



Our Drug Development Pipeline

* Other indication includes portal hypertension related disease such as Esophageal Variceal Bleeding (EVB).

PHIN-214 is an investigational drug therapy that has not been approved by the FDA or any other regulatory agency around the world.

PHIN-214
FOR REFRACTORY ASCITES

PharmaIN has advanced PHIN-214, our novel compound being developed for treating refractory ascites due to advanced liver cirrhosis, into a US Phase 1b clinical trial in cirrhotic patients.

This is a single dose escalation (dose-ranging) study to evaluate its safety in Child-Pugh A and B grade liver cirrhosis. There are also exploratory efficacy measures in this pioneering study with drug administration by subcutaneous injection.

Our goal is to advance PHIN-214 into a phase 2a clinical trial in 2023.

More details may be found at www.clinicaltrials.gov, trial identifier NCTO5490888

PHIN-214 is an investigational drug therapy that has not been approved by the FDA or any other regulatory agency around the world.

 

PHIN-1314
FOR ONCOLOGY

Our early-stage new drug candidate PHIN-1314 is in development as a first-in-class tumor microenvironment modifier targeting a wide range of solid tumor cancers, either as a single agent or in combination with approved immuno-oncology drugs.

With a unique mechanism of action (MOA), PHIN-1314 is being developed for administration by subcutaneous injection.

Efficacy has been demonstrated in multiple animal models.

INTELLECTUAL
PROPERTY ESTATE

The scientific breakthroughs achieved by our team are protected by a robust and growing intellectual property estate.

This includes more than 20 families of composition of matter and utility patents and patent applications, both US and global.

We will continue to pursue compound-specific IP over time.



Liver Cirrhosis

Our Initial Focus: New Therapies for Advanced Liver Cirrhosis

Approximately 1 million Americans suffer from liver cirrhosis, primarily as a result of viral hepatitis, alcoholism, or increasingly, non-alcoholic steatophepatitis (“NASH”). Cirrhosis due to NASH, which arises from fatty liver disease, is increasing rapidly due to the epidemic of obesity that is frequently accompanied by diabetes. The first sign that liver cirrhosis has progressed into a medically serious condition is often the appearance of ascites fluid in the abdomen, detected by ultrasound. This signals that liver status has changed from “compensated” to “decompensated” and the patient is now at increased risk of serious medical complications.

In most patients, early-stage ascites can be controlled with a salt-restricted diet and pharmacologic therapy using diuretics (“water pills”). However, in about 10% of patients with ascites, the ascites gets much worse and eventually does not respond to diuretic therapy, or the patients can no longer tolerate this therapy. This stage is called “refractory ascites” and these patients are frequently hospitalized with life-threatening infections, kidney failure, and other complications such as hepatorenal syndrome (“HRS”) which has a life expectancy of only a few weeks unless it can be reversed. If they are not lucky enough to receive a liver transplant, which is the only definitive cure, they face a mortality rate of about 50% with 12-24 months.

HOW DOES ASCITES DEVELOP?
PHIN-214:

A Potential New Therapy for Refractory Ascites

PharmaIN is currently conducting a Ph1b safety trial of PHIN-214, our investigational new drug candidate, in the first step towards creating what could become the first pharmacological therapy for refractory ascites. The FDA has never approved a drug specifically to treat ascites.

Diuretic drugs are used off-label by physicians in the early stages to help expel the ascites fluid, but they often become ineffective as the ascites worsens.

The therapeutic goal of PHIN-214 therapy will be to shutdown the RAAS signaling pathway, thereby halting the retention of excess salt and water by the kidneys and ending ascites fluid production.

PHIN-214 is an investigational drug therapy that has not been approved by the FDA or any other regulatory agency around the world.

TARGETING SOLID TUMOR CANCERS

 

PharmaIN is focused on the natriuretic peptide receptors in solid tumor cancers. The natriuretic peptide receptor A (NPRA), B (NPRB), and/or C (NPRC) are expressed in certain types of cancer cells, and natriuretic peptides have been implicated in several different types of cancer, including lung, prostate and ovarian.

Studies in multiple animal models have demonstrated the potential for our lead drug candidate, PHIN-1314, as a first-in-class tumor microenvironment modifier. The results indicate potential as a single agent or in combination with approved immunotherapy drugs, which may yield a synergistic effect. Further studies are planned to elucidate this compound’s unique mechanism of action (MOA). Our goal is to commence a GLP toxicity study next year.