Singh Biotechnology Receives Second FDA Orphan Drug Designation for its Novel Single Domain Antibody for the Treatment of Osteosarcoma by Targeting Intracellular STAT3

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Singh Biotechnology today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SBT-100, its novel anti-STAT3 (Signal Transducer and Activator of Transcription 3) B VHH13 single domain antibody (sdAb) for the treatment of osteosarcoma.

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“SBT-100 represents a promising new approach for a targeted immuno-therapy in the treatment of osteosarcomas since it represents a first-in-class therapeutic which specifically targets intracellular proteins", said Sunanda Singh, M.D., Ph.D., Founder & President/CEO of Singh Biotechnology.

Singh Biotechnology today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SBT-100, its novel anti-STAT3 (Signal Transducer and Activator of Transcription 3) B VHH13 single domain antibody (sdAb) for the treatment of osteosarcoma. SBT-100 is able to cross the cell membrane and bind intracellular STAT3 and inhibit its function. Dysregulation of STAT3 activity has been implicated in the development of many malignancies including osteosarcoma. Many studies have shown that STAT3 expression and survival of human tumors is coupled with elevated STAT3 expression and associated with a poor prognosis of many solid tumors, including osteosarcoma. Due to stagnation in survival rate under current standard of care, innovative drugs and approaches to treating osteosarcoma are needed.

“Since the STAT3 pathway plays an important role in development of osteosarcoma and represents a new potential therapeutic target, developing anti-STAT3 therapeutics for osteosarcoma is essential to treating this disease,” said Sunanda Singh, M.D., Ph.D., Founder & President/Chief Executive Officer of Singh Biotechnology. "We believe, SBT-100, our single domain antibody represents a promising new approach for a targeted immuno-therapy in the treatment of osteosarcomas since it represents a first-in-class therapeutic which specifically targets intracellular proteins." STAT3 can be inhibited either by directly targeting the protein or indirectly, by targeting the upstream regulators of the STAT3 pathway. In vitro and in vivo models of tumor growth and metastasis have shown that targeting STAT3 activation via inhibitors is a promising treatment for cancer. Singh Biotechnology is developing SBT-100, a sdAb to STAT3 to provide a novel treatment option for patients with osteosarcoma as an effective and alternative treatment over the currently available options of surgery, radiation therapy, and chemotherapy.

Orphan designation is granted by the FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. For a drug to qualify for orphan drug designation both the drug and the disease must meet certain criteria specified in the Orphan Drug Act (ODA) and FDA's implementing regulations at 21 CFR Part 316. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

About STAT3
Impairment in the regulation of STAT3 activity has been implicated in the development of many malignancies, including osteosarcoma. To this end Singh Biotechnology has developed an anti-STAT3 single domain antibody that binds specifically to the intracellular STAT3 protein for the treatment of osteosarcoma. Currently there are no STAT3 inhibitors available clinically to help treat patients.

About Osteosarcoma
Osteosarcoma is an aggressive rare disease defined by the presence of malignant mesenchymal bone forming cells depositing immature bone matrix. With an annual incidence rate of only 3.1 cases per million in the US, it is considered a rare disease. Although the survival of patients with osteosarcoma has improved with the introduction of chemotherapy, the rates have since plateaued. The 5-year survival rate of patients with high grade osteosarcoma is 60-66% but is dependent on stage at diagnosis. Patients with localized disease have a 5-year survival rate as high as 60-78%, however the rate is only 20-30% for those with metastatic disease. In addition, older adults diagnosed with osteosarcoma have shown a decreased response and toleration to chemotherapy leading to a poor prognosis. Recurrent disease level reduces the 5-year survival rate to 20%. Therefore, there is an unmet clinical need and incentive for developing more suitable and specific anti-STAT3 inhibitors that can provide an effective and alternative treatment of osteosarcoma in patients over the currently available options of surgery, radiation therapy, and chemotherapy.

About Singh Biotechnology
Singh Biotechnology (SBT) is a start-up biotech organization established in 2014 which discovers and develops unique and proprietary therapeutic single domain antibodies (sdAbs) for the treatment of a variety of cancers, autoimmune and ophthalmic diseases by leveraging its novel technology platform. Using this technology platform, SBT has been able to generate therapeutic sdAbs that specifically target intracellular molecules of interest, which play an important role in the pathogenesis of these diseases. Currently SBT is at the pre-IND stage for triple negative breast cancer (TNBC) and is targeting to file an IND in early 2018. In addition, SBT has several concurrent scientific programs in various stages of pre-clinical development for oncology, autoimmune, and ophthalmic diseases. On August 29th, 2016 Singh Biotechnology’s SBT-100 was granted orphan drug designation for the treatment of pancreatic cancer.

Singh Biotechnology is a privately held company headquartered in Tampa Bay, Florida. For additional information visit: http://www.singhbiotechnology.com

Contacts:
Sunanda Singh, M.D., Ph.D.
Founder & President/CEO
Singh Biotechnology
ssingh@singhbiotechnology.com

Ashutosh S. Parihar
Vice President Research & Development
Singh Biotechnology
aparihar@singhbiotechnology.com

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