On November 14, 2019 EpicentRx, Inc., a clinical cancer immunotherapy company targeting both sides of the immune system to deliver cancer treatments with minimal toxicity, reported that it has raised a $35 million Series D financing using the Silicon Valley based life-science focused investment bank, Biotech Alliances International (Press release, EpicentRx, NOV 14, 2019, View Source [SID1234551173]). The purpose of this funding round, which will be split into three tranches, is to progress the late-stage clinical development of EpicentRx’s lead anticancer agent, RRx-001, and proprietary transgene-enhanced oncolytic adenoviral platform technology.

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"We are extremely grateful to Biotech Alliances for supported this financing round, which enables us to advance clinical development of our lead cancer programs, including the Phase 3 REPLATINUM trial which recently began enrolling patients," said Corey A. Carter, M.D., President & CEO of EpicentRx.

"This financing validates the potential and uniqueness of our two complementary anticancer platforms, which reprogram both arms of the immune system, innate and adaptive, to seek out and destroy cancer cells. As a company led by practicing oncologists, it also reinforces our mission to provide hope and additional options to cancer patients by developing new treatments with minimal toxicity," commented Dr. Carter.

"It was a great pleasure working with the EpicentRx team on this Series D financing which will allow the company to advance their clinical-stage portfolio of novel immunotherapies. Biotech Alliances International was pleased to have exclusively attracted European private financing sources for this $35 million deal," said Dr. Franck Brinkhaus, CEO of Biotech Alliances.

RRx-001 is a small molecule immunotherapy targeting the CD47 – SIRPα axis. The drug is currently being evaluated in combination with platinum doublet chemotherapy in the Phase 3 REPLATINUM trial for the treatment of third-line and beyond small cell lung cancer.

EpicentRx is also developing a diverse array of oncolytic adenoviruses that are genetically engineered to replicate selectively in cancer cells. Tony Reid, M.D., Ph.D., EpicentRx’s Chief Scientific Officer and Professor of Medicine at the University of California San Diego Moores Cancer Center, discovered and developed the agents. In addition to oncolysis, these adenoviruses have additional and complementary mechanisms of action through therapeutic "arming" with multiple transgenes, that are expressed selectively in cancer cells during replication. The company has identified a lead candidate, TGF-beta trap, that is currently in preparation for Phase 1 trial.

The Series D brings the total capital raised by EpicentRx to approximately $77 million.

About RRx-001

RRx-001 is a next generation small molecule immunotherapy being developed by EpicentRx. The therapy targets the CD47 – SIRPα axis and repolarizes tumor associated macrophages (TAMs) and other immunosuppressive cells in the tumor microenvironment to an immunostimulatory phenotype. RRx-001 stimulates the immune system and can be used as monotherapy or in combination with chemotherapy, immunotherapy, radiation and targeted agents, giving the therapy the potential to convert "treatment-resistant" tumors into "treatment-sensitive" tumors. RRx-001 is currently in the Phase 3 REPLATINUM trial for the treatment of third-line and beyond small cell lung cancer (SCLC). RRx-001 is also in the Phase 2 QUADRUPLE THREAT trial for the treatment of SCLC, non-small cell lung cancer, neuroendocrine tumors, ovarian and prostate cancer, and the Phase 2 PREVLAR trial for the prevention of oral mucositis. Clinical studies for the drug have also been conducted for the treatment of colorectal cancer, brain metastases and glioblastoma.

On November 13, 2019 Protagonist Therapeutics, Inc. (Nasdaq:PTGX) reported that Dinesh V. Patel, Ph.D., President and Chief Executive Officer, will present at two upcoming healthcare investor conferences in New York (Press release, Protagonist, NOV 13, 2019, View Source [SID1234551233]).

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Presentation details:
Event: Stifel 2019 Healthcare Conference
Date: Wednesday, Nov. 20
Time: 2:25 p.m. EST

Event: Piper Jaffray 31st Annual Healthcare Conference
Date: Thursday, Dec. 5
Time: 1:30 p.m. EST

Live and archived audio webcasts of the Stifel and Piper presentations can be accessed by visiting the Investors page of the Protagonist Therapeutics website at View Source

On November 13, 2019, Exicure, Inc. (the "Company") reported that it has entered into a Collaboration, Option and License Agreement (the "Collaboration Agreement") with a wholly-owned subsidiary of Allergan plc, Allergan Pharmaceuticals International Limited ("Allergan") (Filing, 8-K, Exicure, NOV 13, 2019, View Source [SID1234553517]). Pursuant to the Collaboration Agreement, the Company granted to Allergan exclusive access and options to license spherical nucleic acid ("SNA") based therapeutics arising from two collaboration programs related to the treatment of hair loss disorders. Each such license would grant to Allergan exclusive, royalty-bearing, sublicenseable, nontransferable, worldwide rights to develop, manufacture, use and commercialize such SNA therapeutics. Upon written notice to the Company, Allergan may exercise its option at any time following the effective date of the Collaboration Agreement until the expiration of the corresponding collaboration program’s option exercise period (each, an "Option Exercise Period").

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Under the terms of the Collaboration Agreement, the Company will conduct discovery and development in two collaboration programs for hair loss disorders. The Company shall be solely responsible for all costs and expenses of conducting each collaboration program through the completion of initial preclinical activities, and, if Allergan pays the option extension payment described below, all additional activities that are necessary to enable the first filing of an investigational new drug application ("IND"), except to the extent Allergan elects to conduct any formulation assessment activities or in vivo efficacy models. In the event that Allergan exercises an option, Allergan will be responsible for further development from the license effective date and commercialization of the corresponding licensed product.

Under the terms of the Collaboration Agreement, the Company will receive an upfront payment of $25 million, and, if Allergan exercises any of its option rights, Allergan will pay to the Company an option exercise fee equal to $10 million for each exercised option, if such option is exercised during the initial option exercise period. Should Allergan wish to extend an option exercise period beyond the applicable initial exercise period for a particular program, Allergan must pay the Company a one-time option extension payment in the amount of $10 million for each such option exercise period extension and the option payment to exercise the option during such option period extension shall then be $15 million for each exercised option.

If Allergan exercises an option for a program, development and regulatory milestones will be payable for that program upon the initiation of certain clinical trials, and acceptance of the filing for processing by the FDA in the United States and by 2 additional regulators outside the United States of a marketing application for review, per program, with an aggregate total of up to $195 million if both options are exercised. Commercial milestones will be payable for that program upon first commercial sale of a licensed product in certain jurisdictions and the achievement of specified aggregate sales thresholds for all licensed products from that program, with an aggregate total of up to $530 million if both options are exercised. In the event a therapeutic candidate subject to the collaboration results in commercial sales, the Company is eligible to receive tiered royalties at percentages ranging from the mid-single digits to the mid-teens on future net product sales of such commercialized therapeutic candidates. A percentage of the aforementioned payments will be due to Northwestern University ("Northwestern") upon receipt, pursuant to the Company’s existing license agreements with Northwestern (the "Northwestern License Agreements").

The Collaboration Agreement will remain in effect, unless earlier terminated, until (a) the expiration of the later-to-expire option exercise period, if Allergan does not exercise either option, or (b) the expiration of the last-to-expire royalty term for any licensed product in such country on a licensed product-by-licensed product and country-by-country basis, if Allergan exercises one or both options. Upon expiration of the royalty term with respect to a particular licensed product in a country, the license for such product in such country will convert to a worldwide, fully-paid, irrevocable and perpetual license.

The Collaboration Agreement also contains customary provisions for termination by either party, including in the event of breach of the Collaboration Agreement, subject to cure, by Allergan for convenience and by the Company upon a challenge of the licensed patents, subject, in certain cases, to customary reversion rights. Upon termination of the Collaboration Agreement by Allergan for convenience or by either party for the other’s breach or bankruptcy, all licenses granted by the Company to Allergan will terminate.

The Collaboration Agreement includes customary representations and warranties on behalf of both the Company and Allergan. The Collaboration Agreement also provides for customary mutual indemnities.

On November 13, 2019 Avidity Biosciences, a biotechnology company pioneering Antibody-Oligonucleotide Conjugates (AOCs) for the treatment of rare muscle disorders and other serious diseases, reported the completion of a $100 million Series C financing (Press release, Avidity Biosciences, NOV 13, 2019, View Source [SID1234551124]).

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The financing was led by RTW Investments and included new investors Cormorant Asset Management, LP, CureDuchenne, Logos Capital, Perceptive Advisors LLC and ST Pharm. Existing investors also participated and include Alethea Capital, Alexandria Venture Investments, Boxer Capital of Tavistock Group, Brace Pharma Capital, EcoR1 Capital, Partner Fund Management and Takeda Ventures. Eli Lilly and Company contributed $15 million to the financing in connection with the research collaboration between Lilly and Avidity initiated in April 2019. Roderick T. Wong, M.D., managing partner of RTW Investments, will join Avidity Biosciences’ board of directors.

"Avidity pioneered AOC technology that combines two powerful technologies: monoclonal antibodies for effective delivery and oligonucleotide therapeutic for potent and selective activity," said Sarah Boyce, president and CEO of Avidity Biosciences. "Using our AOC platform, we can unlock new potential for RNA therapeutics by efficiently targeting muscle and immune cells. These funds will enable Avidity to advance our growing proprietary portfolio of multiple programs, including our AOC treatment of myotonic dystrophy type I, to treat serious muscle disorders."

"Our investment team is excited about Avidity’s first-in-class therapy for myotonic dystrophy, and the potential of its AOC platform to deliver RNA-based therapeutics to muscle and create transformative treatments for diseases with high unmet need," said Dr. Wong. "As a member of the board, I look forward to supporting the advancement of their pipeline into the clinic."

Dr. Wong is the founder of RTW Investments and is responsible for managing the firm’s investments focused on innovative drug development. Prior to forming RTW, he was a managing director and sole portfolio manager for the Davidson Kempner Healthcare Funds. Before this, he held various healthcare investment and research roles at Sigma Capital and Cowen & Company. Dr. Wong received his medical doctorate from the University of Pennsylvania Medical School and an MBA from Harvard Business School, as well as a bachelor’s degree in economics from Duke University.

SVB Leerink acted as lead financial advisor for Avidity Biosciences’ Series C financing.

On November 13, 2019 IDEAYA Biosciences, Inc. (Nasdaq:IDYA), an oncology-focused precision medicine company committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics,reported financial results for the third quarter ended September 30, 2019 (Press release, Ideaya Biosciences, NOV 13, 2019, View Source [SID1234551141]).

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"We have made significant progress on our Phase 1/2 tissue-agnostic basket trial evaluating IDE196 in patients with solid tumors harboring GNAQ or GNA11 (GNAQ/11) mutations, including metastatic uveal melanoma (MUM), cutaneous melanoma and colorectal cancer. This clinical trial progress, considered together with recent FDA feedback providing guidance on the regulatory pathway for IDE196 for treatment of MUM, are important enabling steps to advance this clinical-stage program," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

IDEAYA continues to advance its MAT2A synthetic lethality program to treat patients having tumors with MTAP deletion. The Company remains committed to the research and development of a differentiated MAT2A inhibitor that could meaningfully enhance patient outcomes across a broad range of indications. IDEAYA also continues to progress its broad pipeline of synthetic lethality programs, including Pol theta for patients having tumors with BRCA or other homologous recombination deficiency (HRD) mutations, and Werner (WRN) for patients having tumors with high microsatellite instability (MSI).

Key highlights for IDEAYA’s research and development programs include:

Clinical Program IDE196

IDE196

Advanced IDEAYA’s Phase 1/2 tissue-type agnostic basket trial, which was initiated in June 2019 to evaluate IDE196 in solid tumors harboring activating GNAQ/11 mutations, and is entitled "A phase 1/2 study of IDE196 in patients with solid tumors harboring GNAQ/11 mutations or PRKC fusions" (ClinicalTrials.gov Identifier: NCT03947385)

Completed enrollment in the Phase 1 dose escalation portion of the Phase 1/2 clinical trial at sites in the U.S. and Australia

Anticipate selection of the dosing regimen and initiation of the Phase 2 portion of the clinical trial by year-end 2019

Expect to introduce a tablet formulation of IDE196 in Q1 2020 for use in the Phase 2 portion of the clinical trial

Enrolled our first cutaneous melanoma patient harboring a tumor with an activating GNAQ/GNA11 mutation in October 2019, a key milestone for evaluating IDE196 in solid tumors outside of metastatic uveal melanoma (MUM)

Expanded our relationship with Foundation Medicine, participating in their Smart Trials program for identification of non-MUM patients having tumors with activating GNAQ/11 mutations for potential enrollment in our Phase 1/2 basket trial

Anticipate release of interim data from the Phase 1/2 basket trial in Q2/Q3 2020

Obtained FDA feedback in End-of-Phase 1 meeting in Q4 2019, providing guidance on the regulatory pathway for IDE196 for treatment of MUM

Company’s proposed single-arm Phase 2 IDE196‑001 clinical trial may be adequate to support an NDA seeking Accelerated Approval for IDE196 monotherapy in metastatic uveal melanoma (MUM)

The single-arm potentially registration‑enabling Phase 2 clinical trial will target enrollment of 60 evaluable MUM patients

The primary endpoint is overall response rate (ORR) as determined by blinded independent central review (BICR), supported by

BICR‑determined duration of response (DOR) as a secondary endpoint

The 13-week GLP-compliant toxicology studies in 2 species is scheduled to initiate in November 2019, in support of FDA requirement that study results be submitted prior to enrollment of more than approximately 50 patients in the investigational arm of the clinical trial that will support a marketing application

Continued the ongoing Phase 1 clinical trial, entitled "A Phase I, multi-center, open-label, study of LXS196, an oral protein kinase C inhibitor, in patients with metastatic uveal melanoma" (ClinicalTrials.gov Identifier: NCT02601378), being conducted by Novartis

A confirmed Complete Response at the 200 mg BID dose level was observed at month 31 in one of four patients previously reported with confirmed Partial Response out of 30 total (28 evaluable) BID patients in this ongoing monotherapy arm of the Novartis clinical trial

Based on confirmation of the single arm trial design and anticipated rate of enrollment, we are anticipating submission of a New Drug Application (NDA) to the FDA in Q4 2021 to Q1 2022 for IDE196 in MUM.

Synthetic Lethality Preclinical Pipeline

MAT2A

We believe that data presented at the AACR (Free AACR Whitepaper)/NCI/EORTC conference in October 2019 demonstrates clinical activity of MAT2A as a biological target in patients having tumors with MTAP deletion.

Continuing our preclinical evaluation of potential drug candidates, including in the HCT116 engineered in-vivo model, and in multiple endogenous in-vivo models

Goal is to select a MAT2A inhibitor development candidate in the first half of 2020 with differentiated properties relative to Agios’ presented published compounds and Agios’ publicly-disclosed properties of AG270

Expect to file an IND for a differentiated MAT2A inhibitor development candidate in second half of 2020

"We continue to aggressively progress our programs, expand our capabilities and enhance our team. IDEAYA remains committed to its vision of improving lives through transformative precision medicines, and believes that the IDE196 tissue-type agnostic basket trial and our pipeline of synthetic lethality programs are key elements of this goal," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

Corporate Updates

IDEAYA anticipates that existing cash, cash equivalents and short-term marketable securities of $109.4 million (as of September 30, 2019) will be sufficient to fund planned operations into the third quarter of 2021.

Financial Results

As of September 30, 2019, IDEAYA had cash, cash equivalents and short-term marketable securities totaling $109.4 million. This compared to cash, cash equivalents and short-term marketable securities of $90.0 million at December 31, 2018. The increase was primarily due to the receipt of $50.2 million in net proceeds from IDEAYA’s initial public offering, which was completed in May 2019, offset by cash used in operations.

Research and development expenses for the three months ended September 30, 2019 totaled $8.9 million compared to $12.5 million for the same period in 2018. The decrease was primarily due to license fees for our IDE196 license agreement with Novartis during the three months ended September 30, 2018, offset by an increase in costs in connection with IDEAYA’s Phase 1/2 clinical trial to evaluate IDE196 in solid tumors, and costs for personnel, consulting, and laboratory supplies in support of our research programs during the three months ended September 30, 2019.

General and administrative expenses for the three months ended September 30, 2019 totaled $2.7 million compared to $1.1 million for the same period in 2018. The increase was primarily due to an increase in costs for personnel, directors’ and officers’ liability insurance premiums, and professional fees in connection with becoming a publicly traded company as well as external legal patent expenses for our product candidates.

The net loss for the three months ended September 30, 2019 was $11.0 million compared to $13.0 million for the same period in 2018. Total stock compensation expense for the three months ended September 30, 2019 was $0.5 million compared to $0.3 million for the same period in 2018.