- FDA grants orphan desig­na­tion for PAT-SM6 in multiple myeloma
- Provides 7 years market exclusivity in the USA post approval

Melbourne, Australia (Press Release) - Patrys Limited (ASX: PAB), a clin­i­cal stage bio­technology com­pany, has received con­firmation of Orphan Medicinal Product Designation for its lead anti-cancer prod­uct PAT-SM6, from the USA Food and Drugs Admin­istra­tion (FDA).

PAT-SM6 has pre­vi­ously been granted orphan drug desig­na­tion for multiple myeloma in Europe.

Orphan prod­uct desig­na­tion is intended to provide incentives to encourage com­pa­nies to pursue cures and treat­ments for rare diseases with high unmet medical needs.

Under orphan drug status, PAT-SM6 qualifies for scientific and protocol assistance, poten­tial grant funding during devel­op­ment as well as a period of 7 years of market­ing exclusivity in the USA upon FDA approval.

In the USA, there are 78,000 people cur­rently living with multiple myeloma, with 22,000 new cases and around 11,000 deaths each year. Approval of the orphan desig­na­tion in the USA adds to the already existing orphan status granted in Europe and sig­nif­i­cantly expands the poten­tial market for PAT-SM6. It also presents a unique oppor­tu­ni­ty for Patrys to fast track the devel­op­ment of the PAT-SM6 prod­uct in both the USA and Europe.

No cure cur­rently exists for multiple myeloma patients and limitations of avail­able ther­a­pies are particularly evident in patients that have re­lapsed or become resistant to treat­ments. Even though the appearance of some new agents in recent years have shown promise in treat­ment of multiple myeloma, a clear need for the devel­op­ment of addi­tional novel thera­peutics still exists.

Patrys' lead anti­body drug PAT-SM6 is showing convincing evi­dence of poten­tial thera­peutic benefit in its ongoing Phase I/IIa clin­i­cal trial in patients with re­lapsed and refractory multiple myeloma and as such has a poten­tial to im­prove and add to current treat­ments for myeloma.

Dr. Marie Roskrow, Patrys’ CEO said: “The FDA's orphan drug desig­na­tion for PAT-SM6 rep­re­sents yet another con­firmation of the quality of our prod­uct and sup­ports and extends our efforts for a quicker develop­ment process in the USA as well as Europe."

About Patrys Limited

Based in Melbourne, Australia, Patrys (ASX: PAB) is focused on the devel­op­ment of natural human anti­bodies as ther­a­pies for cancer and other major diseases. Patrys has a deep pipe­line of anti-cancer natural human anti­bodies that enable both internal devel­op­ment and partnering oppor­tu­ni­ties. More in­­for­ma­tion can be found at www.patrys.com.

About PAT-SM6

The natural human anti­body PAT-SM6 has been shown to have potent anti-cancer properties in a large num­ber of laboratory and animal studies. More specifically, Patrys has now screened PAT-SM6 against more than 200 tumours from individual patients with various cancers, and the prod­uct binds to over 90% of the tumours screened re­gard­less of cancer type or patient age, gender or disease stage. With respect to mul­ti­ple myeloma PAT-SM6 has shown particularly strong promise. Patrys has filed patent appli­ca­tions to cover the PAT-SM6 anti­body molecule, disease target, and the mech­a­nism of action. Patrys has suc­cess­fully com­pleted a Phase I clin­i­cal trial to eval­u­ate PAT-SM6 as a ther­apy for mel­anoma.

About Multiple Myeloma

Multiple myeloma is a type of bone marrow cancer arising from plasma cells, and new ther­a­pies are des­per­ately needed to treat patients who become resistant to estab­lish­ed chemotherapeutics. There is an esti­mated 200,000 cases world­wide and the incidence is in­creas­ing. The five-year survival of patients is approx­i­mately 30% (at 10 years ~20%). Despite new marketed ther­a­pies, multiple myeloma remains largely incurable and fatal. The multiple myeloma market is dominated by three major prod­ucts: Revlimid, Velcade and Thalidomide with com­bined net sales greater than US$6 Billion in 2012.

Source: Patrys Limited.