On January 24, 2019 Inovio Pharmaceuticals, Inc. (NASDAQ: INO) reported that a second patient with HPV-related head and neck cancer treated with INO-3112 (now called MEDI0457) in a Phase 1 trial achieved a sustained complete response (full remission) after subsequent treatment with a PD-1 checkpoint inhibitor (Press release, Inovio, JAN 24, 2019, View Source;Neck-Cancer-after-Treatment-with-Synthetic-DNA-Vaccine-and-a-PD-1-Checkpoint-Inhibitor/default.aspx [SID1234532884]). This marks the second patient with metastatic cancer observed in full remission (complete response) after treatment with synthetic DNA vaccine followed by a PD-1 checkpoint inhibitor.

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Dr. J. Joseph Kim, Inovio’s President and CEO, said, "Achieving sustained complete responses with immunotherapy in metastatic cancer patients is what you hope for with novel cancer treatments. The fact that the treatment with our synthetic DNA vaccine followed with two different PD-1 inhibitors in this HPV-related cancer patient population showed a complete response in 2 out of 4 progressors is very encouraging as the best complete response rate by PD-1 inhibitors as a monotherapy in metastatic head and neck cancer is approximately 4%. While additional data from Phase 2 clinical studies will provide more insights to the power of synthetic DNA vaccine, this newly reported data provides additional validation for Inovio’s overall cancer combination strategy using a T cell activator combined with a checkpoint inhibitor against an array of cancers with big pharma partners providing various checkpoint inhibitors. In addition to our partnership around HPV-related cancers, Inovio is also collaborating with F. Hoffman-La Roche Ltd./Genentech and Regeneron in efficacy trials coupling Inovio’s INO-5401 with their checkpoint inhibitors designed to increase response rates in metastatic bladder and GBM, respectively, with interim efficacy data expected later this year."

Both patients who achieved full cancer remission were treated with four doses of synthetic DNA vaccine as part of a Phase 1 monotherapy trial of 22 patients with HPV-related head and neck squamous cell carcinoma in which 91% of patients (20/22) showed T cell activity in the blood or tissue. This demonstrates that synthetic DNA vaccine generated robust HPV16/18 specific CD8+ T cell responses in peripheral blood and increased CD8+ T cell infiltration in resected tumor tissue samples.

Of the four patients who developed progressive disease and were subsequently administered a PD-1 checkpoint inhibitor, two patients rapidly exhibited a complete response. The most recent patient for which data was presented yesterday received pembrolizumab (KEYTRUDA); while the previously reported complete responder was treated with nivolumab (OPDIVO). The patients moved from metastatic head and neck cancer to no evidence of disease and they remain alive two years after treatment. Detailed results of the first patient with head and neck cancer who received nivolumab were published in the October issue of Clinical Cancer Research.

These results were presented on January 23 at the Keystone Symposia Conference/Cancer Vaccines being held in Vancouver, Canada, by David B. Weiner, Ph.D., executive vice president of The Wistar Institute, director of its Vaccine & Immunotherapy Center, and the W.W. Smith Charitable Trust Professor in Cancer Research.

KEYTRUDA is a registered trademark of Merck & Co. (MRK); OPDIVO is a registered trademark of Bristol-Myers Squibb Company (BMY).

About HPV-Related Head & Neck Cancer

Human papillomavirus (HPV) is the most common sexually transmitted disease in the United States, currently infecting about 79 million Americans. HPV is known to play a major role in the development of head and neck cancers, which include cancers of the oral cavity, oropharynx, nose/nasal passages and larynx. In 2019 an estimated 53,000 persons will get oral cavity or oropharyngeal cancer in the U.S. New cases of head and neck cancer occur nearly three times more often in men as in women. Incidence rates of head and neck cancers have been on the rise, especially HPV-related oropharyngeal cancer in men, and are expected to continue growing.

On January 24, 2019 Immunomic Therapeutics, Inc. reported that its’ founder and CEO, William Hearl, Ph.D., will present an overview of the company and its UNITE technology platform at the 2019 BIO CEO & Investor Conference on February 12, 2019 at 2:00 pm EST in the Hudson/Empire Room at the Marriott Marquis in New York City (Press release, Immunomic Therapeutics, JAN 24, 2019, View Source [SID1234532885]).

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On January 24, 2019 ArQule, Inc.’s (Nasdaq: ARQL) partner, Basilea Pharmaceutica Ltd. (SIX: BSLN), reported that it entered into a collaboration with Roche (SIX: RO, ROG) to explore a combination of derazantinib (BAL087) and Roche’s PD-L1-blocking immune-checkpoint inhibitor, atezolizumab (Tecentriq), in patients with urothelial cancer. Basilea expects to start a biomarker-driven multi-cohort phase 1/2 study in mid-2019 (Press release, ArQule, JAN 24, 2019, View Source [SID1234532887]).

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The planned study will assess the safety, tolerability and efficacy of the derazantinib-atezolizumab combination in patients with advanced urothelial cancer and confirmed FGFR genomic aberrations. Basilea will be the sponsor of the study, and Roche will provide clinical supply of atezolizumab.

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Peter Lawrence, President and Chief Operating Officer of ArQule, said, "This new combination trial with Tecentriq represents an important step in the development of derazantinib (BAL087, formerly ARQ087) and has the potential to expand meaningfully its therapeutic utility. We look forward to further progress and updates from Basilea."

Derazantinib was licensed to Basilea Pharmaceutica in April 2018 in the US, EU, Japan and the rest of world excluding Greater China. Under the terms of the license agreement, ArQule is eligible to receive up to $326 million in regulatory and commercial milestone payments.

On January 23, 2019 BridgeBio Pharma, a clinical-stage biopharmaceutical company focused on genetic diseases, reported a new financing round of $299.2 million (Press release, BridgeBio, JAN 23, 2019, View Source [SID1234576267]). The round was co-led by existing investors KKR and Viking Global Investors. Other existing investors participating included Perceptive Advisors, AIG, Aisling Capital, Cormorant Capital, and Hercules Capital; and they were joined by new investors Sequoia Capital, and a blue-chip long-term investor. The financing will be used to support BridgeBio Pharma’s existing drug research and development programs and expand its efforts to rapidly develop medicines for patients with unmet needs.

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"We are privileged to be working with investors who believe in our goal of creating medicines for patients with genetic disease. We are aware that many of these patients lack effective treatment options, and we take our mission to help them seriously," said Neil Kumar, Ph.D, co-founder and chief executive officer of BridgeBio Pharma. "The path from promising early-stage science to a drug that makes a difference for patients requires a long-term vision and steady commitment. We are fortunate to have our investors’ support as we develop these treatments."

Genetic diseases are conditions that derive directly from mutations in their patients’ DNA. These mutations can be either inherited or spontaneous, and the diseases stemming from them include both Mendelian diseases, which tend to affect pediatric patients, and cancers. While there are more than 7,000 genetic diseases affecting 25 to 30 million Americans in aggregate, fewer than 500 drugs are approved for these conditions, leaving a significant number of patients without any therapeutic options.

BridgeBio Pharma was formed in 2015 by a team of drug research and development veterans from both the biotech industry and academia. The company seeks to translate novel scientific discoveries from universities, academic medical centers, and pharmaceutical research groups into genetically-targeted therapeutics that address the fundamental causes of disease. Its portfolio of more than 15 assets includes several in the pre-clinical stages of development, as well as four programs in or approaching pivotal trials.

Each of these drug assets is housed in its own subsidiary company, with access to centralized resources and capabilities courtesy of a novel corporate structure developed in conjunction with Dr. Andrew Lo of MIT’s Sloan School of Management. BridgeBio employs a lean, capital-efficient model that harnesses a central research and development platform to simultaneously operate multiple programs that fit the company’s stringent science criteria. Using this model, personnel and funding can be efficiently redistributed amongst the assets on an as-needed basis. The portfolio assets span therapeutic areas including genetic dermatology, oncology, cardiology, neurology, endocrinology, renal disease, and ophthalmology. Some of the specific indications targeted include transthyretin amyloidosis (ATTR-CM and ATTR-PN), pantothenate kinase-associated neurodegeneration (PKAN), Gorlin syndrome and frequent basal cell carcinomas, dystrophic epidermolysis bullosa (DEB), Darier and Hailey-Hailey diseases, Netherton syndrome, venous malformations, Canavan disease, Leber’s hereditary optic neuropathy, molybdenum cofactor deficiency Type A, achondroplasia, and FGFR, SHP-2, and K-RAS-driven cancers.

On January 23, 2019 Gossamer Bio, Inc., a clinical-stage biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutics in the disease areas of immunology, inflammation and oncology, reported that it has filed a registration statement on Form S-1 with the U.S. Securities and Exchange Commission (the "SEC") in connection with the commencement of a proposed initial public offering of its common stock. Gossamer Bio is offering 14,375,000 shares of its common stock at an initial public offering price of $16.00 per share. Gossamer Bio has applied to list its stock for trading on the Nasdaq Global Select Market under the symbol "GOSS (Press release, Gossamer Bio, JAN 23, 2019, View Source [SID1234532832])." Gossamer Bio expects to grant the underwriters a 30-day option to purchase up to an additional 2,156,250 shares of common stock in connection with the offering. All of the shares are being sold by Gossamer Bio.

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Gossamer Bio has included in the registration statement the proposed public offering price and the number of shares offered and specific language under Rule 473(b) promulgated under the Securities Act of 1933, as amended (the "Securities Act"), such that the registration statement is expected to become automatically effective 20 calendar days after today’s filing, or February 12, 2019, pursuant to Section 8(a) of the Securities Act. Gossamer Bio expects to complete the pricing of the offering on such date. In the event that the federal government and the SEC resume normal operations prior to February 12, 2019, Gossamer Bio will re-evaluate the use of Section 8(a) in connection with the offering.

BofA Merrill Lynch, SVB Leerink, Barclays and Evercore ISI are acting as joint book-running managers for the proposed offering.

A registration statement relating to these securities has been filed with the SEC, but has not yet become effective. These securities may not be sold, nor may offers to buy be accepted, prior to the time the registration statement becomes effective. This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any offer or sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such state or jurisdiction.

The proposed offering will be made only by means of a prospectus. Copies of the preliminary prospectus relating to the proposed offering may be obtained, when available, from: BofA Merrill Lynch, NC1-004-03-43, 200 North College Street, 3rd Floor, Charlotte, NC 28255-0001, Attention: Prospectus Department, or by email at [email protected]; or from SVB Leerink, Attention: Syndicate Department, One Federal Street, 37th Floor, Boston, MA 02110, or by email at [email protected], or by telephone at (800) 808-7525, ext. 6132; or from Barclays, c/o Broadridge Financial Solutions, Attn: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, or by telephone at (888) 603-5847, or by email at [email protected]; or from Evercore ISI, Attention: Equity Capital Markets, 55 East 52nd Street, 36th Floor, New York, NY 10055, or by telephone at (888) 474-0200, or by email at [email protected].