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SpringWorks Therapeutics Launches with $103M in Series A Funding and Rights to Four Clinical Programs

On September 25, 2017 SpringWorks Therapeutics, a mission-driven medicines company dedicated to developing innovative potential new treatments for underserved patient communities, reported its launch with a completed $103 million Series A financing funded by Bain Capital Life Sciences, Bain Capital Double Impact, OrbiMed, Pfizer (NYSE:PFE) and LifeArc (formerly known as MRC Technology) (Press release, SpringWorks Therapeutics, SEP 25, 2017, View Source [SID1234529339]). SpringWorks Therapeutics also has rights to four clinical-stage experimental therapies from Pfizer.

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"SpringWorks Therapeutics will pursue the development of medicines across therapeutic areas, focused on diseases where there is an urgent need and the potential for the greatest impact for patients," said Lara S. Sullivan, M.D., MBA, Founder and President of SpringWorks Therapeutics and a former Vice President at Pfizer. "We initially have rights to four very promising experimental therapies and, over time, plan to expand our pipeline by partnering with other life science companies and academic institutions who share in our mission."

New Approach to Drug Development

SpringWorks Therapeutics was originally conceived by Pfizer as an innovative way to advance investigational therapies that may hold significant promise for underserved patients. SpringWorks Therapeutics’ collaborative business model is designed to deliver both social and financial returns via partnerships with a variety of stakeholders, including scientists, biopharmaceutical partners, patient groups, funders and philanthropists. Pfizer’s contribution consists of both equity capital and royalty- and milestone-bearing licenses to experimental therapies.

"Pfizer sees SpringWorks Therapeutics as a groundbreaking new model for collaboration to deliver on the promise of medical research and development, so that more people have the potential to overcome disease. We hope that our investment in SpringWorks Therapeutics will, over time, enable us to realize even more value for patients and society," said Freda Lewis-Hall, M.D., DFAPA, Executive Vice President and Chief Medical Officer, Pfizer. "SpringWorks Therapeutics started as an idea about a new way to get things done with—and for—patients, it’s been a tremendous team effort, and we and our partners are excited to see it become a reality."

Promising Pipeline with Four Clinical-Stage Experimental Therapies

SpringWorks Therapeutics is focused on underserved patient populations where there is great medical need. The company plans to move forward potential programs for four diseases, all of which currently have no cure. SpringWorks Therapeutics plans to expand its pipeline by partnering with other life science companies and academic institutions who share in the company’s mission.

DESMOID TUMOR

A desmoid tumor is a rare, non-metastatic tumor of connective tissue cells, which can cause severe morbidity, pain and loss of function in children and adults. Desmoid tumors can show up in almost any part of the body, and desmoids that are faster growing or located near vital organs can cause life-threatening problems. Approximately 900-1,200 people are diagnosed with desmoid tumors each year in the U.S.1 Currently available treatments include unapproved medical therapy, radiation therapy, thermal ablation and surgery, which can be dangerous, costly and offer limited effectiveness. SpringWorks Therapeutics is planning to initiate a Phase 3 program to establish safety and efficacy of nirogacestat (PF-03084014), its gamma-secretase inhibitor, and will work collaboratively with the Desmoid Tumor Research Foundation to enable the needs of the patient community to be addressed.

NEUROFIBROMATOSIS

Neurofibromatosis (NF) refers to three genetic disorders—NF1, NF2 and schwannomatosis—which cause tumors to grow on nerves throughout the body and can lead to blindness, deafness, disfigurement, cancer, bone abnormalities, learning disabilities and severe pain. NF1 affects one in 3,000 individuals and usually is diagnosed in childhood when symptoms begin to appear.2 MEK inhibitors have shown encouraging activity in reducing tumor size in clinical Phase 1-2 studies in patients with plexiform neurofibromatosis, one of the many manifestations of NF1. SpringWorks Therapeutics is planning to initiate a Phase 3 program to establish safety and efficacy of its MEK 1/2 inhibitor (PD-0325901) in the NF1 population and will work collaboratively with the Children’s Tumor Foundation to enable the needs of the patient community to be addressed.

HEREDITARY XEROCYTOSIS

Hereditary xerocytosis (HX) is a genetic disorder in which red blood cells become dehydrated due to loss of potassium and cell water. The fragility of the dehydrated red cells can lead to a ranging severity of anemia and can cause complications including jaundice, fatigue, splenomegaly and gallstones. In some cases, it will lead to severe anemia that requires frequent blood transfusions. HX affects an estimated one in 10,000 people, and symptoms begin shortly after birth.3 There is no approved therapy for this disease. Senicapoc (PF-05416266) has demonstrated a good safety/tolerability profile in previous Phase 1-3 studies in other indications. SpringWorks Therapeutics plans to assess the potential activity of senicapoc in hereditary xerocytosis.

POST-TRAUMATIC STRESS DISORDER

Post-traumatic stress disorder (PTSD) is a chronic condition that some people develop after experiencing traumatic or life-threatening events, serious injury or sexual violence. PTSD involves the persistent re-experiencing of the traumatic event, which results in avoidance of trauma-related stimuli, as well as negative feelings and heightened anxiety-like symptoms. PTSD is often seen in military veterans, first responders, rape and battery victims, and abused children. Around 8.6 million people in the U.S. between the ages of 18-64 have been diagnosed with the disease.4 Over 200,000 veterans in the U.S. live with PTSD, which is currently treated with antidepressants such as SSRIs and trauma-focused psychotherapy; however, the disease can result in suicide even with treatment. SpringWorks Therapeutics’ FAAH inhibitor (PF-0445784) has demonstrated a good safety/tolerability profile in previous Phase 1 studies. The effectiveness of PF-0445784 in PTSD is to be determined. Cohen Veterans Bioscience (CVB) and SpringWorks Therapeutics plan to assess patient populations that could benefit from this mechanism.

Experienced Leadership Team

SpringWorks Therapeutics is led by a preeminent team of industry veterans, including:

Daniel S. Lynch, Executive Chairman, who has over 25 years of industry experience serving in management and board positions for a number of top-tier biotechnology and pharmaceutical companies.
Lara S. Sullivan, M.D., Founder and President, who brings more than two decades of senior leadership experience in biopharmaceuticals, healthcare and life sciences, most recently at Pfizer, where she had previously led the portfolio strategy for the company’s early stage pipeline and its Medical collaboration funding platform.
Stephen Squinto, Ph.D., Acting Head of Research & Development, Member of the Board of Directors, who brings over 25 years of biotechnology industry experience as both a scientist and senior executive.
Saqib Islam, JD, Chief Financial Officer and Chief Business Officer, who brings over 25 years in international business management with a focus on business development, global strategic planning and capital markets in the healthcare sector. Most recently, within the biotechnology industry, Saqib was an Executive Vice President and Chief Strategy Officer at Alexion Pharmaceuticals and Chief Business Officer at Moderna Therapeutics.
L. Mary Smith, Ph.D., Vice President, Clinical Research and Development, who brings more than 20 years of research and clinical development experience from both pharmaceutical and biotech companies. Most recently, Smith was the Vice President of Product Development at United Therapeutics and led the development and approval of Unituxin for high-risk neuroblastoma, a rare pediatric cancer.
In addition to Lynch, Sullivan and Squinto, SpringWorks Therapeutics has appointed the following seasoned directors to the board:

Carl L. Gordon, Ph.D., CFA – Partner, OrbiMed
Peter Keen – Trustee, LifeArc
Freda Lewis-Hall, M.D., DFAPA – Executive Vice President and Chief Medical Officer, Pfizer
Deval Patrick – Managing Director, Bain Capital Double Impact
Jeffrey Schwartz – Managing Director, Bain Capital Life Sciences

10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

(Filing, 10-K, Palatin Technologies, 2017, SEP 25, 2017, View Source [SID1234520832])

Salubris Pharmaceuticals Invests $5 Million in GO Therapeutics

On September 25, 2017 GO Therapeutics reported that Salubris Pharmaceutical Limited has made a $5M investment in GO Therapeutics, a Cambridge, MA-based company exploiting new advances in glycoproteomics to develop novel, multimodal first-in-class cancer therapeutics against intractable targets (Press release, GO Therapeutics, OCT 25, 2017, View Source [SID1234521293]).

The generation of solid tumor targeting domains that are specific to cancer cells remains a significant challenge in the drug development industry. GO Therapeutics’ targeting platforms and technologies open a novel class of tumor-specific antigens that promise to realize the full potential of antibody-drug conjugates, bi-specific T-cell engagers, and immune-based cell therapies.

"This investment from Salubris will accelerate the development of our pre-clinical antibody programs, and help support the discovery of new antibodies against prioritized targets of interest. Additionally, Salubris offers future downstream market access to China, which is expected to become a significant consumer of innovative cancer therapies," said Constantine Theodoropulos, Chief Executive Officer, GO Therapeutics.

"Novel, cancer-specific targets are imperative to widening the therapeutic window for powerful, cutting-edge cancer therapeutic modalities such as T-cell engagement and ADCs that offer tremendous promise for patients around the world. GO Therapeutics has the opportunity to generate significant value by opening an exciting new class of cancer-specific targets that will underpin transformative cancer therapies," said Sam Murphy, VP and Head of International Business Development for Salubris, who will join GO Therapeutics’ board.

Salubris’ strategic investment extends the company’s expansion to the U.S. and evolution towards leading-edge innovations in oncology and cardiovascular disease. Earlier this year Salubris announced the opening of the SalubrisBio research facility outside of Washington D.C. where the focus is building the company’s internal portfolio of NME biologic product candidates (www.salubrisbio.com).

BioTime Board of Directors Approves Distribution of AgeX Therapeutics Shares to BioTime Shareholders

On September 25, 2017– BioTime, Inc. (NYSE American: BTX), a clinical-stage biotechnology company focused on developing and commercializing products addressing degenerative diseases, reported that its Board of Directors has approved a distribution of some or all of the shares of AgeX Therapeutics, Inc. owned by BioTime to BioTime’s shareholders (Press release, BioTime, SEP 25, 2017, View Source;p=RssLanding&cat=news&id=2302486 [SID1234520622]). The Board also authorized management to work with investment banks and other financial institutions to finalize and implement the strategy for taking AgeX public, which may include a tax-free distribution.

"Asterias BioTherapeutics and OncoCyte Corporation, two companies founded by BioTime, were successfully transitioned into independent, publicly-traded companies that created significant value for BioTime and its shareholders," said Alfred Kingsley, BioTime’s Chairman of the Board. "We believe the formation and independent funding of AgeX, and a subsequent distribution to BioTime shareholders, will similarly unlock the significant value of the previously embedded BioTime assets related to the treatment of aging and age-related diseases, such as diabetes, obesity, heart disease, stroke and cancer."

AgeX Therapeutics focuses on technologies relating to cellular immortality and the regenerative biology of aging. Aging and age-related diseases have recently garnered significant investor interest, as evidenced by the formation of companies such as Calico, Human Longevity Inc., Unity Biotechnology, and Samumed, as well as others. These companies have attracted major financial supporters, many of whose investments were made at multibillion-dollar market valuations. AgeX’s initial investors similarly include institutions and accomplished business leaders, whose support creates a foundation for the company’s potential future success.

AgeX closed its initial $10 million equity financing in August of 2017 with a post-money valuation of approximately $68 million. BioTime currently owns approximately 85% of the outstanding shares of AgeX, with a post-money valuation of approximately $58 million, or 50 cents per BioTime share. The equity financing is expected to fund AgeX’s general operations and product development well into 2019, while saving BioTime more than $5 million annually on these programs and associated operational expenses.

Building on the recent success of the formation, funding and launch of AgeX, BioTime’s management and Board are now exploring all options for making AgeX a publicly-traded company, including a potential tax-free distribution of all AgeX shares to BioTime shareholders. Once BioTime’s management completes its discussions with investment banks and other financial firms, and its analysis of remaining tax, legal, commercial and regulatory issues, BioTime will announce further details of the resulting plan.

SignalRx Presents in silico Design of Dual PI3K/BRD4 Inhibitors for Combinatorial Activation of Anti-tumor Immunity in Treating Cancer

On September 25, 2017 SignalRx Pharmaceuticals Inc. reported the presentation of scientific data on the company’s in silico platform technology for the rational design of dual small-molecule PI3K/BRD4 inhibitor for immune-oncology relative to activating anti-tumor immunity (Press release, SignalRx, SEPT 25, 2017, http://www.ireachcontent.com/news-releases/signalrx-presents-in-silico-design-of-dual-pi3kbrd4-inhibitors-for-combinatorial-activation-of-anti-tumor-immunity-in-treating-cancer-647615323.html [SID1234527320]). The presentation by Dr. Donald L. Durden, MD, PhD, senior scientific advisor for SignalRx, was made at the Immunomodulatory Small Molecules section of the 15th Annual Discovery on Target meeting in Boston, MA on September 25, 2017 at 8:40 a.m.

The presentation was entitled "A Novel Dual PI3K/BRD4 Inhibitor, SF2523 for Combinatorial Activation of Anti-Tumor Immunity in Cancer via the Orthogonal Inhibition of MYCN and MYC" and highlighted advancements in the development of single small-molecules that simultaneously inhibit both PI3 kinase (PI3K) and the new epigenetic cancer target BRD4 in vivo.

Key highlights presented are:

Dual inhibitory chemotype disclosed which blocks MYC via two orthogonal independent pathways.
Synergistic anticancer effects of dual inhibition: PI3K inhibition induces MYCN degradation and BRD4 inhibition blocks MYCN transcription.
Dual PI3K/BRD4 inhibitor SF2523 blocks MYCN transcription and induces MYCN degradation
SF2523 shown to block tumor growth, metastasis, and PI3K/BRD4 signaling in vivo.
Demonstrated that SF2523 abrogates the macrophage immunosuppressive effects on tumor immunity via the blockade of the M1- M2 transition in vivo, and activates the adaptive T cell immune response against the tumor.
Data also was also presented related to the recent discovery of SRX3207, a novel dual inhibitor which inhibits PI3K and a recently discovered new immune checkpoint kinase. This inhibitor has potent immunostimulatory properties and blocks the immunosuppressive macrophage compartment.
SignalRx, focused on developing more effective oncology drugs through molecular design imparting multiple target-selected inhibition, is also announcing that it is seeking partnerships to accelerate the development of their novel small molecules into first-in-man clinical trials. These molecules include single-targeted novel BRD4 inhibitors and CDK inhibitors with picomolar potencies.