ISA Pharmaceuticals Receives US Orphan-Drug Designation for ISA101b in HPV16-positive Cervical Cancer

Primes the immune system to attack cancer cells in HPV16-positive cancers

LEIDEN, Netherlands, July 1, 2020 /PRNewswire/ -- ISA Pharmaceuticals B.V., a clinical-stage company dedicated to developing rationally designed immunotherapeutics for oncology and infectious disease, today announced that it received Orphan Drug Designation from the Food and Drug Administration (FDA) in the USA for its lead product ISA101b for treatment of Human Papilloma Virus type 16 (HPV16)-positive cervical cancer.

According to the latest statistics of the Centers for Disease Control and Prevention, HPV causes more than 34,000 cases of cancer in the United States each year. Cervical cancer is the most common HPV-associated cancer in women, with HPV16 being responsible for approximately 50% of cases. HPV16-positive cervical cancer is the number 2 cause of cancer for women in the age 15-44 in the US. At 1% annually, rates of cervical cancer have been dropping only marginally due to prevention through screening and prophylactic vaccination. There is an important medical need for disease modifying therapies to treat women with this terrible disease. For 2020, SEER databases project 13,800 new cases and 4,290 women dying from cervical cancer in the US.

ISA101b is a clinical-stage immunotherapy targeting HPV16-induced diseases such as cervical and oropharyngeal cancer. It induces specific immune responses to the oncogenic E6 and E7 proteins of HPV16 and is based on ISA's proprietary Synthetic Long Peptide (SLP®) technology.

"We are pleased to have Orphan Drug Designation for our ISA101b program targeting HPV16-positive cervical cancers," said Kees Melief, Chief Scientific Officer of ISA Pharmaceuticals. "Our mission is to unleash the power of the patient's own immune system to eradicate their cancer while maintaining optimal quality of life. We believe the best and most logical way to do this is through the use of SLPs. The metastatic form of cervical cancer has a particularly high unmet medical need with 5 year survival rates not going beyond 17%. The Orphan Drug Designation for ISA101b recognizes the urgent need for more efficacious treatment options for patients suffering from metastatic HPV16-positive cervical cancer."

About Orphan Drug Designation (ODD)

Orphan drugs are intended for the treatment, diagnosis or prevention of serious diseases that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. FDA evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. FDA provides incentives for sponsors to develop products for rare diseases, including development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the US once the product has been approved, provided that the product is first to market.

About ISA 101b

ISA101b is a Synthetic Long Peptide (SLP^®) therapeutic designed to mount a highly specific, broad and durable, T-cell mediated attack by the immune system on tumors positive for human papilloma virus type 16 (HPV16). The product has successfully and safely completed multiple human clinical studies in over 200 patients

• ISA101 has been shown to be effective as monotherapy in patients with early stage, HPV16 positive, premalignant neoplasias¹.
• In late stage HPV16 positive cervical cancer, ISA101b acts favorably in synergy with chemotherapy as assessed by both size of HPV-specific immune response and clinical benefit^2,3. Late stage cancer patients suffer from local and systemic immunosuppression by immune suppressive myeloid cells, that are depleted by standard of care chemotherapy such as carboplatin and paclitaxel. This enables ISA101b to more effectively attack the cancer via a T-cell mediated immune response.
• Importantly, in a Phase 2, open label, Proof of Concept study, ISA101b in combination with nivolumab (anti-PD1 antibody) has shown tumour response rates and overall survival approximately double those seen with anti-PD1 monotherapy in 2^nd line HPV16-positive head and neck cancer⁴.
• ISA101b is protected by several patent families covering different aspects of the vaccine product. Patent protection of the vaccine product is expected until at least 2037.

ISA is currently carrying out a randomized, controlled phase 2 trial of ISA101b in combination with Libtayo^® (cemiplimab) in oropharyngeal cancer, under a strategic immuno-oncology collaboration with Regeneron, which was strengthened in June 2020. Further clinical trials are in preparation, including a potentially pivotal clinical trial for the combination of ISA101b and Libtayo in oropharyngeal cancer as well as a phase 2 study investigating the same combination in cervical cancer. Libtayo is being jointly developed by Regeneron and Sanofi.

About ISA Pharmaceuticals

ISA Pharmaceuticals is an immunotherapy company developing treatments for various cancers and infectious diseases. ISA Pharmaceuticals has best-in-class technology to stimulate and activate the human immune system, specifically T cells, to fight diseased or infected cells. ISA Pharmaceuticals has a collaboration with Regeneron for its lead asset, ISA101b, an immunotherapy targeting human papillomavirus type 16 (HPV16)-induced cancer. ISA101b is currently in a Phase 2 clinical trial for first- and second-line HPV16-induced head-and-neck cancer, in combination with Libtayo, a PD-1 antibody. For more information, please visit www.isa-pharma.com.

References

1. Kenter G, et al. New England J Med. 2009
2. Welters MJ, et al. Sci Transl Med. 2016
3. Melief C, et al. Sci Transl Med. 2020
4. Massarelli E, et al. JAMA Oncology. 2018