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

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]).

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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).

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.

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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.

CHIME’s biologic products are designed to introduce specific epitopes from childhood vaccines into tumors after administration with a delivery system (e.g., viral vector) to rapidly stimulate and expand existing effector memory T cells that patrol the body to destroy such ‘infected’ cells (Fact Sheet, JLABS, SEP 22, 2017, View Source [SID1234520598]). The recall and redirection of pre-existing memory T-cells against tumor cells should present a number of advantages compared to other immunotherapies: it does not require the discovery of cancer antigens and it does not need to break tolerance since it is based upon a non-self approach; it also does not require ex vivo manipulation of cells and it can be administered directly to patients as a biologic. CHIME’s product should be applicable to all types of tumors.

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CHIME’s unique approach does not require to modify or educate naive T cells but rather promotes the recruitment and activation of pre-existing vaccine-derived memory T cells to the cancerous tissue. The unique advantages of CHIME’s approach based on memory responses is expected to induce a strong and rapid direct tumor killing, decrease the tumor-mediated immune suppression, and drive the development of a systemic tumor-specific immunity through the release of tumor-associated antigens that are taken up by antigen presenting cells and presented to T cells to generate tumor-specific effector cells and tumor-specific immune memory.

On September 21, 2017 Cellectar Biosciences, Inc. (Nasdaq: CLRB), an oncology-focused, clinical stage biotechnology company (the "company"), and Onconova Therapeutics (Nasdaq:ONTX) reported that they have entered into a strategic collaboration to develop new phospholipid drug conjugates (PDCs) combining Cellectar’s patented phospholipid ether delivery platform with select proprietary compounds or payloads from Onconova’s early stage product pipeline (Press release, Cellectar Biosciences, SEP 22, 2017, View Source [SID1234520607]). Newtown, Pa.-based Onconova is a late-stage biopharmaceutical company focused on the discovery and development of novel small molecule drug candidates to treat cancer.

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"Access to novel anti-tumor payloads is key to leveraging our next generation PDC delivery platform technology for the discovery of novel, proprietary targeted anti-cancer therapeutics," said Jim Caruso, president and CEO of Cellectar Biosciences. "Onconova is an established player in developing small molecule anti-cancer compounds. Their unique early stage assets, development experience and ability to successfully advance compounds into Phase 3 clinical trials makes them an excellent partner for Cellectar."

Under the terms of the collaboration, Onconova will provide Cellectar with several compounds, including some from the family of molecules that contains Briciclib, which is an EIF4E targeting small molecule with early Phase 1 data. Cellectar will leverage its expertise in early development and chemical conjugation to link the molecules to its phospholipid ether (PDC platform) to create new, more precisely targeted antitumor agents. Both companies will have the option to advance the development of any of the newly conjugated PDC molecules. Financial terms of the collaboration have not been disclosed.

"We are focused on optimizing the delivery of our therapeutic compounds in the battle against Myelodysplastic Syndrome and a variety of cancers. As such, we are excited to collaborate with Cellectar and leverage their PDC platform, which we believe can improve the targeting of our molecules directly to the cancer, in addition to extending the patent coverage of these drug candidates," said Ramesh Kumar, Ph.D., president and CEO of Onconova Therapeutics.

About Phospholipid Drug Conjugates (PDCs)
Cellectar’s product candidates are built upon its patented cancer cell-targeting delivery and retention platform of optimized phospholipid ether-drug conjugates (PDCs). The company deliberately designed its phospholipid ether (PLE) carrier platform to be coupled with a variety of payloads to facilitate both therapeutic and diagnostic applications. The basis for selective tumor targeting of our PDC compounds lies in the differences between the plasma membranes of cancer cells compared to those of normal cells. Cancer cell membranes are highly enriched in lipid rafts, which are glycolipoprotein microdomains of the plasma membrane of cells that contain high concentrations of cholesterol and sphingolipids, and serve to organize cell surface and intracellular signaling molecules. PDCs have been tested in more than 80 different xenograft models of cancer.