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BOSTON and LONDON, May 03, 2018 (GLOBE NEWSWIRE) — Orchard Therapeutics, a leading commercial stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation to the company’s gene therapy candidate OTL-200, for the treatment of patients with metachromatic leukodystrophy (MLD).

MLD is a rare, fatal, neurodegenerative, inherited metabolic disease caused by mutations in the ARSA gene. In its late infantile and juvenile forms, which represents the majority of MLD patients, mortality at 5 years is estimated at 75% and 30%, respectively¹.

The FDA grants Rare Pediatric Disease Designations for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The Rare Pediatric Disease Designation makes the program eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of OTL-200 by the FDA. OTL-200 was acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and the Telethon Foundation, acting through their joint Telethon Institute for Gene Therapy, in Milan, initiated in 2010. This collaboration led to the development of Strimvelis, the world’s first approved autologous ex vivo gene therapy product. Orchard anticipates filing OTL-200 for market authorization with regulatory authorities from 2019.

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BOSTON and LONDON, April 24, 2018 (GLOBE NEWSWIRE) — Orchard Therapeutics, a leading commercial and clinical-stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies, today announced the opening of a new U.S. office in Boston’s Seaport District.

The new Boston office builds upon the company’s footprint in London as well as Foster City and Menlo Park, California. Orchard’s Boston office will become the company’s hub for its commercial operations and several key financial and administrative functions to support the rapidly expanding global organization.

“The opening of our Boston office represents a significant milestone in the growth and global expansion of Orchard Therapeutics” commented Mark Rothera, president and CEO of Orchard. “We look forward to joining the thriving biotechnology and academic communities in Boston, Cambridge and the surrounding area. The area has a deep pool of talent with complementary capabilities to help us realize our vision of becoming a global, fully integrated company transforming the lives of patients with rare diseases.”

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CAMBRIDGE, Mass.–Checkmate Pharmaceuticals (Checkmate) announced that it had initiated treatment with CMP-001 combined with atezolizumab (TECENTRIQ®) in a Phase 1b clinical trial of patients with advanced non-small cell lung cancer (NSCLC) and disease progression on prior anti-PD-1/PD-L1 therapy.

CMP-001 is designed to activate innate immunity to convert “uninflamed” tumors, which generally do not respond to anti-PD-1/L1 therapy, into “inflamed” tumors, which are responsive to PD-1 inhibition. When used in combination with PD-1/PD-L1 inhibitors, CMP-001 has the potential to increase the number of cancer patients who respond to checkpoint inhibitor therapies and to increase the magnitude and duration of the antitumor responses, possibly providing added clinical benefit.

“We are pleased to be advancing the clinical development of CMP-001 into a second tumor type,” stated Art Krieg, founder and CEO of Checkmate Pharmaceuticals. “The mechanism of action of CMP-001 should apply across most or all tumor types, and so we expect CMP-001 to reverse PD-1 resistance in NSCLC, just as we reported for PD-1 resistant advanced melanoma last week at the 2018 American Association of Cancer Research Annual Meeting in Chicago,” noted Dr. Krieg.

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CAMBRIDGE, Mass. — Surface Oncology, Inc., a clinical-stage immuno-oncology company developing next-generation antibody therapies that target the tumor microenvironment, today announced the pricing of its initial public offering of 7,200,000 shares of common stock at a public offering price of $15.00 per share. In addition, Surface has granted the underwriters a 30-day option to purchase up to 1,080,000 additional shares of common stock at the initial public offering price, less the underwriting discount. The gross proceeds from the offering, before deducting underwriting discounts and estimated offering expenses, are expected to be $108.0 million, excluding any proceeds from the exercise of the underwriters’ option to purchase additional shares. Surface’s common stock is expected to begin trading on the Nasdaq Global Market under the ticker symbol “SURF” on April 19, 2018. All of the shares are being offered by Surface.

Goldman Sachs & Co. LLC, Cowen and Company, LLC and Evercore Group L.L.C. are acting as joint book-running managers for the offering.

In addition to the shares sold in the public offering, Surface announced the concurrent sale of an additional 766,666 shares at the initial offering price of $15.00 per share, for gross proceeds of $11.5 million, in a private placement to Novartis Institutes for Biomedical Research, Inc. The sale of these shares will not be registered under the Securities Act of 1933, as amended, and will be subject to a 180-day lock-up agreement.

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CAMBRIDGE, Mass. — Checkmate Pharmaceuticals (Checkmate) announced the first presentation of data from an ongoing Phase 1b study evaluating CMP-001, Checkmate’s Toll like receptor 9 (TLR9) agonist, in combination with pembrolizumab (KEYTRUDA®), an anti-PD-1 therapy, in patients with advanced melanoma resistant to prior anti-PD-1 checkpoint inhibition. CMP-001 is designed to activate innate immunity to convert “uninflamed” tumors, which generally do not respond to anti-PD-1 therapy, into “inflamed” tumors, which are PD-1 responsive. Data were presented in the clinical trials plenary session on Tuesday, April 17 at the American Association for Cancer Research (AACR) annual meeting in Chicago by principal investigator Mohammed Milhem, MBBS, clinical professor of internal medicine, University of Iowa, Iowa City.

The combination of CMP-001 and pembrolizumab was generally well tolerated and induced deep and durable clinical responses with systemic regression of noninjected cutaneous, nodal, hepatic, and splenic metastases in patients who had progressed on a median of 2 prior therapies. “The abscopal effect observed in these patients is a hallmark of successful intratumoral immunotherapy treatment,” said Dr. Milhem. “In this patient population, pembrolizumab alone would be unlikely to provide more than a 7% response rate. If the current results are confirmed, it appears that this combination could offer an effective treatment option for patients with advanced melanoma who are not responsive to pembrolizumab,” he noted.

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