On November 13, 2019 Brickell Biotech, Inc. ("Brickell") (Nasdaq: BBI), a clinical-stage pharmaceutical company focused on developing innovative and differentiated prescription therapeutics for the treatment of debilitating skin diseases, reported financial results for the third quarter ended September 30, 2019 and provided a corporate update (Press release, Vical, NOV 13, 2019, View Source [SID1234551140]).

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"Becoming a publicly listed company earlier this year was a transformative milestone for Brickell and we remain confident about the potential of our lead asset, sofpironium bromide, for primary axillary hyperhidrosis," commented Robert Brown, Chief Executive Officer of Brickell. "Ten million people in the United States suffer from this disease which can have a negative impact on a patient’s social life, well-being, emotional and mental health."

Business and Recent Developments

In September 2019, Brickell announced completion of its merger with Vical Incorporated ("Vical"), following approval by Vical’s shareholders, and commenced trading on The Nasdaq Capital Market under the ticker symbol "BBI". Vical contributed approximately $35 million to the combined company in addition to an R&D financing arrangement entered into with NovaQuest Capital Management that provides up to $25 million in funding.

The long-term safety study for sofpironium bromide is fully enrolled with 300 subjects and is on track to be completed in the first quarter of 2020. Earlier this year, Brickell’s development partner, Kaken Pharmaceutical Co. Ltd. ("Kaken"), achieved positive pivotal Phase 3 results in its clinical study conducted in Japan. To date there have been 19 clinical studies conducted by Brickell and Kaken of sofpironium bromide gel that encompass over 1,200 subjects.

On October 23, 2019, Bodor Laboratories, Inc. and Nicholas S. Bodor (collectively, "Bodor") filed a complaint against Brickell disputing certain aspects of the license agreement between the parties with respect to sofpironium bromide ("Complaint"). As a result, NovaQuest notified the Company that additional development funding for sofpironium bromide was suspended temporarily. Subsequently, Brickell filed a motion to dismiss the Complaint, initiated arbitration proceedings against Bodor and asserted claims against Bodor for tortious interference and material breach of the license agreement by Bodor.

The sofpironium bromide pivotal Phase 3 studies in axillary hyperhidrosis are ready to commence in the United States, pending developments in the ongoing dispute resolution process with Bodor.

Financial Results

Cash, cash equivalents, and short-term investments were $25.7 million as of September 30, 2019 compared to $8.1 million as of September 30, 2018.

Revenue was $1.2 million for the third quarter of 2019 compared to $3.0 million for the third quarter of 2018. The decrease is due primarily to the completion of certain research and development activities during the three months ended September 30, 2019 for which funding is provided under a license and collaboration agreement with Kaken.

Research and development expenses were $3.3 million for the third quarter of 2019 compared to $4.1 million for the third quarter of 2018. The decrease in research and development expenses is primarily due to a decrease in clinical studies costs associated with sofpironium bromide following the completion of certain clinical trials.

General and administrative expenses were $3.9 million for the third quarter of 2019 compared to $1.2 million for the third quarter of 2018. The increase in general and administrative expenses is primarily due to an increase in professional fees for legal, accounting, and auditing services, including merger-related costs.

The Company’s net loss was $4.8 million for the third quarter of 2019, and $13.0 million for the nine months ended September 30, 2019, compared to $2.5 million for the third quarter of 2018, and $5.6 million for the nine months ended September 30, 2018.

About Sofpironium Bromide

Sofpironium bromide, is a proprietary new molecular entity that belongs to a class of medications called anticholinergics. Anticholinergics block the action of acetylcholine, a chemical that transmits signals within the nervous system that are responsible for a range of bodily functions, including activation of the sweat glands. Sofpironium bromide was retrometabolically designed. Retrometabolic drugs are designed to exert their action topically and are potentially rapidly metabolized once absorbed into the blood. This proposed mechanism of action may allow for highly effective doses to be used while limiting systemic side effects.

About Hyperhidrosis

Hyperhidrosis is a life-altering medical condition where a person sweats more than the body requires to regulate its temperature. More than 15 million people, or 4.8% of the population of the United States, are believed to suffer from hyperhidrosis. Axillary (underarm) hyperhidrosis is the targeted first indication for sofpironium bromide and is the most common occurrence of hyperhidrosis, affecting an estimated 65% of patients in the United States or 10 million individuals. Doolittle et al. Arch Dermatol Res (2016).

On November 13, 2019 Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), reported a paper published in Nature Communications that describes a proof-of-concept for rewiring the cell pathway to create highly intelligent T-cells that can recognize cancerous tumors and cause a micro secretion of therapeutic proteins onto these tumors, which ultimately reshapes the tumor microenvironment and improves the T-cells ability to fight cancer (Press release, Cellectis, NOV 13, 2019, View Source [SID1234551149]). By utilizing gene editing techniques to rewire the TCRα, CD25 and PD1 genes, the study enabled CAR T-cells to micro secrete the pro-inflammatory cytokine, IL-12, in a tumor and time-dependent manner, paving the way for a next generation of tightly controlled, highly active and potentially safer CAR T-cell treatments.

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"Discussion around the tumor microenvironment has become a popular topic in the CAR T-cell space, and with recent advancements in gene editing technologies, especially TALEN, it is now possible to manipulate the way a T-cell regulates itself to adapt to its environment," said Dr. Philippe Duchateau, Ph.D., Chief Scientific Officer, Cellectis. "With seamless modification of multiple genes, and subsequently rewiring their natural regulatory processes, this approach causes the T-cells to secrete therapeutic proteins of interest in a tightly controlled and localized manner. We have essentially transformed the current T-cells used today into precise and powerful micro-robots that can spray IL-12 specifically onto cancer cells – potentially avoiding the toxicity of a systemic injection of IL-12, while enhancing CAR-T activity."

"Our extensive knowledge in TALEN-based gene editing and DNA donor template design enabled us to develop this groundbreaking proof-of-concept, a milestone that paves the way to the next generation of CAR T-cells," added Dr. Julien Valton, Ph.D., Innovation Team Leader, Cellectis. "These highly intelligent CAR T-cells can sense and remodel their microenvironment in a tailored, highly regulated, and antigen-specific manner, allowing us to have more control over increasingly potent treatments and less risk of general secretion into healthy tissues. This engineering strategy could bring smarter, safer and more effective treatments to the forefront for patients in need."

Julien Valton, Ph.D., Innovation Team Leader, Cellular Engineering & Adoptive CAR T-Cell Immunotherapy

Dr. Julien Valton obtained his Ph.D. at the University Joseph Fourier in Grenoble, France, where he was trained as an enzymologist. He then joined the Yale School of Medicine to apply his knowledge to therapeutic research by investigating the mechanism of inhibition of receptor tyrosine kinases that are involved in the development of gastrointestinal cancer. In 2009, he moved a step further into the field of applied science by joining the Innovation Department of Cellectis, where he actively participated in using and improving TALEN gene editing technology for targeted gene therapy and genome engineering. He is now using TALEN along with protein engineering techniques to develop the next-generation CAR T-cells to treat different malignancies.

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 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.