US FDA Grants Orphan Drug Status to SRI’s TALL® for Pancreatic Cancer

SRI's Targeted Antigen Loaded Liposomes (TALL) has received Orphan Drug Designation (ODD) from the U.S. Federal Drug Administration (FDA) for the treatment of pancreatic ductal adenocarcinoma (PDAC). This innovative therapy enhances the advantages of immunotherapy, including check-point inhibitors, and offers new hope for patients with PDAC (1^✔ ✔Trusted Source
SRI earns FDA Orphan Drug Designation for pancreatic cancer

Go to source). SRI is an independent nonprofit research institute. It is a renowned global leader in R&D with a long-standing history of providing support to both government and industry.

This approval has led to SRI's prospective strategic collaborators the opportunity to obtain tax credits for eligible clinical trials and possibly secure market exclusivity for a duration of seven years following the drug's approval, in addition to other advantages.

Kathlynn Brown, president of SRI’s biosciences division said, “FDA's orphan drug designation brings worthy attention to the demonstrated impact of SRI's TALL biotherapeutic for pancreatic cancer,”.

“This milestone fuels SRI’s deep commitment to pioneering health and precision medicine solutions that bring hope to patients and their families."

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Novel Immunotherapy for Pancreatic Tumors

Pancreatic cancer is challenging to identify in its early stages and has the highest mortality rate among major cancers, with only a 3% relative survival rate at 5 years for the advanced form. Pancreatic tumors exhibit strong resistance to chemotherapy and radiation, leading to limited treatment options. SRI's TALL represents an innovative immunotherapy that has the potential to overcome these obstacles and significantly expand the benefits to a large patient population.

Dr. Timothy Cote, former head of the FDA ODD Program and current CEO of Only Orphans Cote said, “SRI is to be congratulated for advancing to this pivotal step,”. He further added, “Their science in this field is strong and the FDA has affirmed that they are providing a genuine orphan drug. SRI’s work in the precision medicine domain sets a standard for the industry and offers life-changing solutions for those in need.”

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Orphan Drug Status

As per U.S. FDA, an orphan drug is a drug intended for use in rare diseases, affecting in lesser than 200,000 individuals in the United States (2^✔ ✔Trusted Source
FDA/CDER Small Business Chronicles

Go to source). There are more than 7,000 identified rare diseases. It is estimated that one in 10 Americans has a rare disease, with half of them being children. Despite this, approximately 95% of these diseases do not have FDA-approved treatments. In 1983, the federal government enacted the Orphan Drug Act to encourage organizations to develop treatments for patients with unmet needs.

A company granted orphan drug status for a designated drug gets a seven-year marketing exclusivity and a tax credit for expenses on clinical investigations.

“This greatly streamlines the go-to-market process toward final FDA clinical drug approval,” said Brown. “We are looking forward to working with our strategic partners to offer the ability to take advantage of the tremendous cost savings and other benefits that come with this designation, including tax credits for clinical trials and a faster go-to-market timeline.”

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Recall Immunity Process Used to Stimulate Immune System

TALL has been developed to deceive the immune system by making tumor cells appear as if they were infected with pathogens. Essentially, TALL changes the appearance of cancer cells to resemble those with measles, exploiting the biological process of recall immunity to stimulate the human immune system to identify and eliminate an intruder it has encountered before.

SRI developed TALL utilizing the company's groundbreaking FOX Three platform, specifically created to address a crucial issue in drug development: the difficulty of delivering biotherapeutic compounds into the interior of cells, thus rendering the targets within those cells unresponsive to drugs. FOX Three utilizes unique processes to identify specific peptide delivery agents (known as Molecular Guidance Systems, or MGS) that target particular cell types and deliver a therapeutic moeity in a cell-specific manner.

For enabling its use as immunotherapy, a synthetic peptide originating from the measles virus is enclosed within liposomes, which are neutral and stealthy particles. Subsequently, a FOX Three MGS that targets tumors is affixed onto the surface of liposomes. These MGSs specifically target tumor cells, allowing the liposomes to be delivered into the tumor cells upon arrival.

The peptide derived from measles is subsequently discharged within the affected cells and ultimately exhibited on their exteriors, marking them for the immune system. The tumor cells 'infected' by measles are subsequently targeted and eliminated by memory T cells, and the patient's immune system eradicates the tumor. Evidence indicating TALL administered as a co-therapy with check-point inhibitors has exhibited enhanced tumor shrinkage for tumors that have conventionally been unresponsive to check-point inhibitor treatments. TALL essentially transforms a cold tumor into a hot tumor and allows medications to function more efficiently.

Brown concluded, “The FDA ODD program and its potential brings hope to many cancer patients fighting this horrible disease and we look forward to reaching more patients through the accelerated process.”

This achievement highlights SRI's dedication to leading the way in developing innovative healthcare and precision medicine solutions that brings hope to patients and their families.

References:

1. SRI earns FDA Orphan Drug Designation for pancreatic cancer - (https://www.sri.com/press/story/sri-earns-fda-orphan-drug-designation-for-pancreatic-cancer/)
2. FDA/CDER Small Business Chronicles - (https://www.fda.gov/media/83372/download)

Source-Medindia