AgeX Therapeutics to Present at Biotech Showcase Annual Conference

On January 5, 2018 AgeX Therapeutics, Inc., a subsidiary of BioTime, Inc. (NYSE American: BTX), reported that it will present at Biotech Showcase, January 8-10, 2018 at the Hilton San Francisco Union Square (Press release, BioTime, JAN 5, 2018, View Source;p=RssLanding&cat=news&id=2325217 [SID1234522928]).

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Michael D. West, Ph.D., Chief Executive Officer, will present at the conference as follows:

Date: Tuesday, January 9
Time: 2:30 PM PST
Track: Yosemite-B (Ballroom Level)
Venue: Hilton San Francisco Union Square, 333 O’Farrell Street

According to the event organizers, Biotech Showcase features more than 3,500 decision makers from 2,100 life sciences companies in a variety of plenary sessions, fireside chats, and panels. This year’s event is expected to draw over 900 investors from 50-plus countries, in anticipation of 400-plus public and private company presentations.

A copy of Dr. West’s presentation will be available on the AgeX website.

NantKwest to Host 3rd Annual Investor/Analyst Day Highlighting a Record Number of 15 INDs Authorized in 2017 Covering 12 Tumor Types with First In-Human Clinical Data of Safety and Early Signs of Clinical Activity of the NANT Cancer Vaccine

On January 5, 2018 NantKwest (NASDAQ: NK), a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of the human immune system using natural killer (NK) cells to treat cancer, reported that the company will be hosting an Investor/Analyst Day on Tuesday, January 9th in San Francisco, California (Press release, NantKwest, JAN 5, 2018, http://ir.nantkwest.com/phoenix.zhtml?c=254059&p=RssLanding&cat=news&id=2325070 [SID1234522918]).

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NantKwest will be sharing with analysts and investors a review of the company’s activities in 2017 and provide a detailed overview for 2018. The record number of 15 INDs authorized in 2017 (aNK, haNK, Cryopreserved haNK and taNK) will be announced, detailing the multiple tumor types to be studied with combination chemo-radiation therapy and activators of adaptive immune system through adenovirus, yeast and super agonist IL-15 platforms. In addition, details will be presented regarding authorization in Europe to treat patients with glioblastoma and authorization by the FDA to initiate high-affinity natural killer (haNK) cell trials in cancer patients with HIV.

First in-human clinical data will be revealed including safety and early signs of clinical activity in patients receiving freshly cultured haNK, cryopreserved haNK and combinations of NK cells (aNK and haNK) with adenovirus, yeast, and super agonist IL-15 platforms. These studies form the basis for multiple Phase II and registration trials anticipated to be initiated in 2018.

At this event, senior management of NantKwest will provide a review of its ongoing R&D program, as well as provide an in-depth clinical trial roadmap for the company’s clinical programs for 2018 and the current status of the GMP manufacturing plants.

A webcast of the event will begin at 7:00 PM PT on Tuesday, January 9, 2018 and is expected to conclude at 9:00 PT. A link to the live webcast can be accessed by visiting the Investors page of the company’s website. In addition, a replay of the event will be posted on the website after the event and will be available for 30 days following the event.

Arvinas Announces Research Collaboration and License Agreement with Pfizer Inc. for the Discovery and Development of Protein Degradation Drug Candidates

On January 4, 2018 Arvinas LLC, a private biotechnology company focused on creating a new class of drugs based on protein degradation, reported a research collaboration and license agreement with Pfizer Inc. (NYSE: PFE) for the discovery and development of drug candidates using Arvinas’ proprietary PROTAC (PROteolysis TArgeting Chimeras) Platform, a novel technology used to create small molecule therapeutics aimed at degrading disease-causing cellular proteins (Press release, Arvinas, JAN 4, 2018, View Source [SID1234585092]).

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The multi-year agreement covers the discovery and development of potential PROTAC clinical candidates designed to degrade several key disease-causing proteins in multiple therapeutic areas. Arvinas will drive discovery efforts, and Pfizer will be accountable for clinical development and commercialization of any products that may result from this collaboration. Under the terms of the agreement, Arvinas may receive up to $830 million in upfront and potential development and commercialization milestone payments upon achievement of specified preclinical, clinical and commercial milestones. In addition, Arvinas may be entitled to receive tiered royalties based on global product sales on any products that may result from this collaboration.

"As a global industry leader, Pfizer is uniquely positioned to partner with us as we exploit the potential of PROTACs in multiple disease areas," stated John Houston, Ph.D., President and Chief Executive Officer of Arvinas. "This marks another key milestone as we continue to expand the use of our targeted protein degradation platform and advance Arvinas’s first candidates into the clinic."

"Protein degradation is an area of considerable interest for us, and we look forward to working with Arvinas to determine the potential applicability of this approach across multiple therapeutic areas," said John Ludwig, Ph.D., Head of Medicinal Sciences, Pfizer.

The PROTAC Platform offers potential improvements over traditional small molecule inhibitors by using the cell’s natural and selective ubiquitin- proteasome system to degrade disease-causing proteins. By removing target proteins directly rather than simply inhibiting them, PROTACs can provide multiple advantages over small molecule inhibitors which can require high systemic exposure to achieve sufficient inhibition, often resulting in toxic side effects and eventual drug resistance. With multiple protein targets, Arvinas’ PROTAC platform has demonstrated that a transient binding event at a range of binding sites and affinities can translate into very potent degradation of the target protein.

pellepharm appoints sanuj k. ravindran, m.d., as president and chief executive officer

On January 4, 2018 PellePharm, a clinical-stage biopharmaceutical company committed to targeting rare genetic dermatological conditions at the source, reported the appointment of Sanuj K. Ravindran, M.D., to the position of president and chief executive officer (Press release, PellePharm, JAN 4, 2018, View Source [SID1234576282]). In parallel, Dr. Ravindran will join BridgeBio Pharma, PellePharm’s lead investor, as CEO-in-Residence, to advance its broader orphan dermatology portfolio.

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"Dr. Ravindran’s biopharma industry experience and rare disease expertise will offer enormous strategic benefit to PellePharm as we move into our next stage of development," said Ervin Epstein, M.D., chief medical officer and co-founder of PellePharm. "Having been successful with the management and growth of multiple biopharma companies, Dr. Ravindran brings the right experience to PellePharm so that we may offer topical patidegib to patients as swiftly as possible."

Dr. Ravindran brings more than 15 years of strategic and operational biopharma experience to PellePharm. Most recently, he was chief business officer at aTyr Pharma ("LIFE"), a clinical stage rare disease-focused biotechnology company, where he led corporate and financial strategy, business development, and investor relations. Prior to that, Dr. Ravindran was senior vice president of corporate development for The Medicines Company ("MDCO"), where he worked to execute multiple transactions totaling more than $2 billion in potential aggregate value. Previously a practicing physician, Dr. Ravindran began his industry career as a venture capitalist for 10 years with Burrill & Company, Radius Ventures, and Asian Healthcare Fund. Dr. Ravindran is trained in Internal Medicine and completed his residency training at Thomas Jefferson University Hospital. Dr. Ravindran received his B.A. from Northwestern University, his M.D. from Jefferson Medical College and his MBA from the Kellogg School of Management.

"I am thrilled to join PellePharm at such an important juncture, as the Company prepares to advance topical patidegib one step closer to patients with Gorlin Syndrome. With PellePharm’s scientific premise, clinical progress, and recently strengthened leadership team, the company is well on its way to meeting its mission of delivering therapies for rare genetic dermatological conditions," said Dr. Ravindran.

PellePharm today also announced that it has expanded its executive team to enhance regulatory and operational capabilities. Alix Alderman is now vice president of regulatory affairs at PellePharm, and Gerd Kochendoerfer, Ph.D., is vice president of technical operations and program management. Both Ms. Alderman and Dr. Kochendoerfer bring many years of experience in drug development, quality management and global regulatory affairs.

"We are pleased to welcome Dr. Ravindran, Dr. Kochendoerfer and Ms. Alderman," said Neil Kumar, CEO and co-founder of BridgeBio Pharma. "At this inflection point, having the right team in place, with the collective experience this group brings, enables PellePharm to more ably achieve its goal of helping patients with serious unmet dermatologic conditions."

About Patidegib

Topical patidegib gel has shown early promise in a Phase 2 clinical study in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. Topical patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials, but without the adverse systemic side effects of oral hedgehog inhibitors. Patidegib’s gel formulation is stable at room temperature for at least two years, making it a viable potential therapy for ongoing, at-home management of Gorlin Syndrome. Topical patidegib currently is being studied in a United States-based Phase 2 clinical trial for the treatment of sporadic basal cell carcinomas (BCCs).

About Gorlin Syndrome

Gorlin Syndrome is a rare, genetic disease characterized by mutations in the tumor suppressor gene encoding Patched1 (PTCH1), which acts as the primary inhibitor of the hedgehog signaling pathway. This leads to hundreds of basal cell carcinomas, especially on the face and sun-exposed areas.

With no FDA-approved drugs available for Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome (BCCNS), the standard of care is surgery. People with severe Gorlin Syndrome may have as many as 30 surgeries per year, which can be repetitive and scarring. Approximately 10,000 people in the United States, or one in 31,000, are believed to be affected by Gorlin Syndrome. Gorlin Syndrome is known by several names, including BCCNS, Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome, or Nevoid Basal Cell Carcinoma Syndrome.

CureVac and Arcturus Therapeutics Announce Broad Strategic Collaboration to Advance Next Generation of Lipid-Mediated mRNA Therapeutics

On January 4, 2018 CureVac AG, a leading clinical-stage biopharmaceutical company focused on the development of pioneering mRNA therapeutics, and Arcturus Therapeutics Ltd. (NASDAQ:ARCT), an RNA medicines company, reported they have entered into a broad strategic collaboration to jointly discover, develop and commercialize novel messenger RNA (mRNA) therapeutics (Press release, CureVac, JAN 4, 2018, View Source [SID1234522914]).

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Under the agreement, the companies will collaborate to develop up to four molecular therapy products for rare diseases using Curevac’s optimized natural mRNA sequence (RNAoptimizer) and Arcturus’s lipid-mediated nucleic acid delivery system (LUNAR). The agreement focuses on developing mRNA therapeutics for enzyme replacement and antibody generation. Development costs will be shared between the companies, with plans to co-commercialize products in the future under a profit sharing arrangement. The first mRNA therapy to be jointly developed and potentially commercialized by the companies will target ornithine transcarbamylase (OTC) deficiency, a genetic disease characterized by the accumulation of ammonia in the blood. The collaboration also grants CureVac access to the full suite of Arcturus’s lipid-mediated delivery intellectual property to enable the development of additional mRNA product candidates.

"This collaboration for up to four products establishes a sound relationship with Arcturus, which we believe is one of the leaders in developing lipid-mediated delivery systems for mRNA molecules," said Ingmar Hoerr, Ph.D., co-founder and CEO of CureVac. "Just as important, we are excited to have secured access to Arcturus’s leading intellectual property rights for future product development in molecular therapies. This partnership combines both companies’ technology platforms with the expertise necessary to develop the next generation of therapeutics based on the considerable potential of mRNA."

"We are thrilled to combine Arcturus’s platform technologies and expertise with CureVac’s recognized capabilities in mRNA construct optimization and GMP manufacturing to co-develop messenger RNA medicines for patients in need," said Joseph Payne, President and CEO of Arcturus. "We believe our collaboration with CureVac has the potential to help reduce costs, mitigate manufacturing risks, and accelerate our timelines for ushering quality mRNA medicines into the clinic."