On October 1, 2018 Corcept Therapeutics Incorporated (NASDAQ: CORT), a company engaged in the discovery, development and commercialization of drugs to treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of the stress hormone cortisol, reported that the Food and Drug Administration (FDA) has granted orphan drug status to Corcept’s selective cortisol modulator relacorilant to treat patients with pancreatic cancer (Press release, Corcept Therapeutics, OCT 1, 2018, https://ir.corcept.com/news-releases/news-release-details/corcept-therapeutics-receives-orphan-designation-relacorilant [SID1234530684]). Corcept is conducting clinical trials of relacorilant in combination with nab-paclitaxel (Celgene Corporation’s drug, Abraxane) as a treatment for pancreatic cancer and other solid tumors.

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Orphan drug designation is granted by the FDA to encourage the development of treatments for diseases that affect fewer than 200,000 patients in the United States. Drugs that receive orphan status obtain seven years of marketing exclusivity from the date of drug approval, tax credits for clinical trial costs, marketing application filing fee waivers and assistance from the FDA in the drug development process. Receiving orphan drug designation does not alter the standard regulatory requirements and process for obtaining marketing approval.

"We are pleased the FDA has granted relacorilant orphan drug status for the treatment of pancreatic cancer. Pancreatic cancer has a poor prognosis and patients have few treatment options," said Joseph K. Belanoff, M.D., Corcept’s Chief Executive Officer. "The data we have generated so far in this indication have been very encouraging. Five of nine patients who received the minimum therapeutic dose in our Phase 1/2 trial of relacorilant plus nab-paclitaxel demonstrated durable disease control. By year-end we expect to have learned enough to determine a potential path toward a pivotal study in this indication."

About Pancreatic Cancer

Pancreatic cancer is the fourth leading cause of cancer-associated mortality, with a five-year survival rate of five percent. In the United States, an estimated 33,000 patients are diagnosed with the disease each year.

On October 1, 2018 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported that it earned a $22 million payment from Verastem Oncology under the license agreement between the Company and Verastem for COPIKTRA (duvelisib) (Press release, Infinity Pharmaceuticals, OCT 1, 2018, View Source [SID1234530645]). The payment was earned upon the approval by the U.S. Food and Drug Administration (FDA) on September 24, 2018 of duvelisib for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two prior therapies, as well as accelerated approval for the treatment of adult patients with relapsed or refractory follicular lymphoma after at least two prior systemic therapies.

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"We are really pleased that duvelisib is now available for patients with CLL/SLL and follicular lymphoma and are proud of the role Infinity played in its development," said Adelene Perkins, Chief Executive Officer and Chair of Infinity. "This $22 million FDA approval payment from Verastem supports Infinity’s continued expansion of the development of IPI-549, our first-in-class, oral, immuno-oncology development candidate that selectively inhibits phosphoinositide-3-kinase-gamma (PI3K-gamma), including in doublet and triplet combination trials to identify the best combination regimens to treat patients with specific types of cancer."

In 2016, Infinity entered into a license agreement granting Verastem an exclusive worldwide license for the research, development, commercialization, and manufacture of duvelisib and products containing duvelisib in oncology. Pursuant to the terms of the license agreement, Verastem has notified Infinity of its election to make the $22 million payment in cash, which Infinity expects to receive later this year. Infinity also is eligible for royalties on worldwide net sales of duvelisib ranging from the mid-to-high single digits, shared equally with Takeda.

Infinity’s updated 2018 financial guidance is:

Net Loss: Infinity expects net loss for 2018 to range from $10 million to $20 million.

Cash and Investments: Infinity expects to end 2018 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $50 million to $60 million.

Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities will be adequate to satisfy the company’s capital needs into 2020. Infinity’s financial guidance excludes additional funding or business development activities and does not include a potential $2 million payment from PellePharm, a private company, upon initiation of a Phase 3 study for the hedgehog inhibitor program, which Infinity licensed to PellePharm in 2013.

On October 1, 2018 Aduro Biotech, Inc. (NASDAQ: ADRO) reported that four separate abstracts from its research and development portfolio will be presented at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in Washington, D.C. from November 7-11, 2018 (Press release, Aduro Biotech, OCT 1, 2018, View Source;p=RssLanding&cat=news&id=2369686 [SID1234530081]). Preliminary clinical data from the ongoing Phase 1 dose-finding study evaluating ADU-S100 (MIW815), an intratumoral STING agonist in patients with advanced solid tumors or lymphomas, were accepted for presentation on November 9, 2018.

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"We look forward to sharing preliminary clinical data from the dose escalation portion of the monotherapy trial which provides an initial understanding of the potential role of ADU-S100 in the treatment of cancer and which contribute to the broader scientific understanding of the STING pathway," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro Biotech. "In collaboration with our partner Novartis, our objective is to characterize the safety and mechanism of action of ADU-S100 across a wide array of solid tumors and lymphomas and provide a basis for continued development of ADU-S100 in combination with checkpoint inhibitors targeting PD-1 and CTLA-4."

Researchers will present additional preclinical data for ADU-S100 in combination with immune checkpoint inhibitors. They will also present the results of early research to identify and characterize an anti-SIRPa antibody ADU-1805.

Details of the poster and oral presentations are as follows:

Abstract 10763: Phase I dose-finding study of MIW815 (ADU-S100), an intratumoral STING agonist, in patients with advanced solid tumors or lymphomas
Date: November 9-10, 2018
Location: Poster Hall E, Walter E. Washington Convention Center

Abstract 10938: ADU-S100 (MIW815) Synergizes with Checkpoint Inhibition to Elicit an Anti-Tumor CD8+ T Cell Response to Control Distal Tumors
Date: November 9-10, 2018
Location: Poster Hall E, Walter E. Washington Convention Center

Abstract 10923: SIRPα blockade increases the activity of multiple myeloid lineage cells, enhances dendritic cell cross-presentation, and aids in remodeling the tumor microenvironment
Session: Rapid Oral Abstracts
Date/Time: November 9, 2018, 1:00 – 2:00 p.m. ET
Location: Poster Hall E, Walter E. Washington Convention Center

Abstract 10960: Pan-allele anti-SIRPα antibodies that block the SIRPα–CD47 innate immune checkpoint
Date: November 9-10, 2018
Location: Poster Hall E, Walter E. Washington Convention Center
About STING Pathway Activator Platform
The Aduro-proprietary STING pathway activator product candidates, including ADU-S100 (MIW815), are synthetic small molecule immune modulators that are designed to target and activate human STING. STING is generally expressed at high levels in immune cells, including dendritic cells. Natural activation of STING is not always sufficient to prevent the growth and spread of cancer cells. In preclinical models, ADU-S100 directly activates STING to further amplify the natural anti-tumor response. Once activated, the STING receptor initiates a profound innate immune response through multiple pathways, inducing the expression of a broad profile of cytokines, including interferons and chemokines. This subsequently leads to the development of a systemic tumor antigen-specific T cell adaptive immune response.

Aduro’s lead molecule, ADU-S100, is the first therapeutic in development specifically targeting STING. In collaboration with Novartis, it is being tested in a Phase 1 clinical trial as a single agent and in combination with ipilimumab, and in a Phase 1b combination trial with spartalizumab (PDR001), an investigational anti-PD-1 compound. These studies are enrolling patients with cutaneously accessible, advanced/metastatic solid tumors or lymphomas. The trials are evaluating the ability of ADU-S100 to activate the immune system and recruit specialized immune cells to attack the injected tumor, leading to a broad immune response that seeks out and kills distant metastases.

On October 1, 2018 NewLink Genetics Corporation (NASDAQ:NLNK) reported that two abstracts were accepted for presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 33rd Annual Meeting being held November 7-11, 2018 in Washington, D.C (Press release, NewLink Genetics, OCT 1, 2018, View Source [SID1234529909]). These poster presentations will feature pharmacokinetic (PK) data from the Company’s Phase 1a study of NLG802, a prodrug of indoximod, in patients with advanced solid tumors and biomarker data from its Phase 2 study of indoximod in combination with checkpoint inhibition for patients with advanced melanoma. These data will be available during poster sessions on November 9 and 10, 2018.

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Abstracts to be Presented at SITC (Free SITC Whitepaper) 2018:

Abstract 11213: A phase 1a clinical trial of NLG802, a prodrug of indoximod with enhanced pharmacokinetic properties, Rixe, O., et al.

Abstract 10294: The immunogenomic impact of indoximod on the tumor microenvironment of melanoma patients, Yu, J., et al.

About Indoximod

Indoximod is an investigational, orally available small molecule targeting the IDO pathway. The IDO pathway is a key immuno-oncology target involved in regulating the tumor microenvironment and immune escape. Indoximod is being evaluated in combination with treatment regimens including chemotherapy, radiation, checkpoint blockade and cancer vaccines across multiple indications such as acute myeloid leukemia (AML), diffuse intrinsic pontine glioma (DIPG) and melanoma.

About NLG802

NLG802 is a prodrug of indoximod. NLG802 has been shown in preclinical trials to increase bioavailability and exposure to indoximod above the levels achievable by direct administration of indoximod. NLG802 is currently being evaluated in clinical trials.

On October 1, 2018 RXi Pharmaceuticals Corporation (NASDAQ: RXII), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported the pricing of an underwritten public offering of 21,428,572 units at a price to the public of $0.70 per unit (Press release, RXi Pharmaceuticals, OCT 1, 2018, View Source [SID1234529890]). Each unit contains one share of common stock (or common stock equivalent) and one warrant to purchase one share of common stock. This offering is expected to close on or about October 3, 2018, subject to customary closing conditions.

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H.C. Wainwright & Co. is acting as the sole book-running manager for the offering.

Each warrant has an exercise price of $0.70 per share, is exercisable immediately and will expire seven years from the date of issuance. The shares of common stock (or common stock equivalents) and the accompanying warrants included in the units can only be purchased together in this offering but will be issued separately and will be immediately separable upon issuance.

RXi expects to receive aggregate gross proceeds of approximately $15 million from the offering, prior to deducting underwriting discounts and commissions and estimated offering expenses. RXi intends to use the net proceeds of this offering towards the development of the Company’s immuno-oncology program, for other research and development activities and for general working capital.

A registration statement on Form S-1 relating to the public offering of the securities described above was filed with the Securities and Exchange Commission ("SEC") and was declared effective on September 28, 2018, and an additional registration statement on Form S-1 filed pursuant to Rule 462(b), which became automatically effective on October 1, 2018. The offering is being made only by means of a prospectus forming part of the effective registration statement. A preliminary prospectus relating to and describing the terms of the offering has been filed with the SEC and a final prospectus relating to the offering will be filed with the SEC, and will be available on the SEC’s website at www.sec.gov. Copies of the final prospectus, when available, may also be obtained from H.C. Wainwright & Co., LLC, 430 Park Avenue, 3rd Floor, New York, NY 10022, by calling (646) 975-6996 or by emailing [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any 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 any such state or jurisdiction.