- Orphan desig­na­tion con­firmed for PAT-SM6 clin­i­cal prod­uct
- Provides 10 years market exclusivity in Europe post approval 
- PAT-SM6 is active in multiple myeloma patients with end stage disease who are resistant to other marketed ther­a­pies

Melbourne, Australia (Press Release) - Patrys Limited (ASX: PAB), a clin­i­cal stage bio­technology com­pany is pleased to announce that lead anti-cancer prod­uct PAT-SM6 has been granted orphan drug desig­na­tion by the European Medicines Agency (EMA) for multiple myeloma.

Orphan drug status is awarded to drugs that offer poten­tial thera­peutic value in the treat­ment of rare dis­eases and con­di­tions. Orphan drug desig­na­tion will entitle PAT-SM6 to:

• Ten years of market exclusivity for multiple myeloma;
• Scientific advice and protocol assistance by the EMA to optimise drug development;
• Regulatory assistance and facilitated access to the Centralised Procedure for marketing approval;
• Numerous financial incentives and fee reductions for regulatory activities; and
• Access to grant funding schemes.

In Europe, there are 60,000 people cur­rently living with multiple myeloma, with over 20,000 new cases and around 15,000 deaths each year. Importantly, the global market is much larger with multiple myeloma the second most common haematological malig­nan­cy affecting approx­i­mately 200,000 people world­wide with approx­i­mately 100,000 new cases annually. The disease is deadly, as the lives of approx­i­mately 70,000 multiple myeloma patients world­wide are lost every year.

No single standard ther­apy cur­rently exists for multiple myeloma patients that have re­lapsed or become resistant to treat­ment and these patients have an ex­pec­ted survival of just 6-9 months. Despite the ap­pear­ance of some new agents with sig­nif­i­cant activity in re­lapsed disease, multiple myeloma remains an incur­able disease, with a clear need for the devel­op­ment of addi­tional novel thera­peutics.

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 the poten­tial to im­prove and add to current treat­ments for multiple myeloma. Orphan drug status presents a unique oppor­tu­ni­ty for Patrys to fast track PAT-SM6 devel­op­ment in Europe, which can sub­se­quently be leveraged in larger global markets.

“Patrys is committed to the devel­op­ment of new ther­a­pies to help address the unmet medical needs of multiple myeloma patients world­wide” said Dr. Marie Roskrow, Patrys’ CEO. “The EMA's orphan drug desig­na­tion for PAT-SM6 rep­re­sents an important mile­stone in PAT-SM6 devel­op­ment and will sup­port our efforts to move PAT-SM6 as quickly as possible through the clin­i­cal and regu­la­tory devel­op­ment process."

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 qualify for 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’ PAT-SM6 is cur­rently showing convincing evi­dence of poten­tial thera­peutic benefit in its ongoing Phase I/IIa clin­i­cal trial in patients with re­lapsed and refractory multiple myeloma. Patrys has also 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.