KSQ Therapeutics Emerges with $76 Million in Financing to Pioneer High-Confidence Drug Development

On October 2, 2017 KSQ Therapeutics reported that they emerged with $76 million in financing and a world-class executive team to advance the development of drugs empowered by the unique biological insights of its proprietary CRISPRomics drug discovery engine (Press release, KSQ Therapeutics, OCT 2, 2017, View Source [SID1234520709]). With CRISPRomics, KSQ is pioneering high-confidence drug development utilizing a suite of CRISPR-based technologies that industrialize, for the first time, a high-throughput, systematic way to elucidate the precise function that each human gene plays across a multitude of diseases. Based on this deep and comprehensive approach, KSQ is advancing a pipeline of oncology and immunology drug development programs against novel therapeutic nodes to create a new set of disease-tailored medicines. KSQ was founded in 2015 by David Sabatini, MD, PhD, of the Whitehead Institute and MIT, William Hahn, MD, PhD, of the Broad Institute and Dana-Farber Cancer Institute, Jonathan Weissman, PhD, of UCSF, and Tim Wang, PhD, of MIT, along with founding investors Flagship Pioneering and Polaris Partners.

KSQ also announced today the appointment of David Meeker, MD, as Chief Executive Officer. Dr. Meeker most recently served as Head of the Specialty Care Business Unit of Sanofi-Genzyme and previously was Chief Executive Officer and President of Genzyme, a Sanofi Company. He brings more than 20 years of industry leadership experience, from leading-edge R&D programs to global operational roles.

"We have a clear goal with CRISPRomics: empower the drug development process to strategically focus on high-confidence, patient-tailored, novel drug candidates," said Dr. Meeker. "There is a compelling need to improve the quality of drug targets and to identify patients most likely to respond because our industry and our health care systems are challenged by the sheer volume of potential new medicines. With our proprietary CRISPRomics engine, KSQ is positioned to play a leading role in shortening drug development timelines, increasing the rate at which meaningful medicines can reach patients, and ultimately, improving the sustainability of our health care systems."

KSQ also announced Frank Stegmeier, PhD, as Chief Scientific Officer and George Golumbeski, PhD, as Executive Advisor and Board Member. Dr. Stegmeier is a leading expert in industrialized functional genomics who led Oncology Target Discovery at Novartis prior to joining KSQ. He has led KSQ’s platform development and R&D efforts since the company’s formation in 2015, demonstrating the early productivity of CRISPRomics. Dr. Golumbeski is a business development executive with an outstanding track record of success in the pharmaceutical industry. During his more than 25-year career, he has identified and evaluated innovative technologies and drug candidates from early-stage biotech companies and accelerated their drug innovation potential through creative partnerships.

"The human genome harbors more than 20,000 genes, however for most diseases we have yet to discern which genes represent the best therapeutic targets. For the first time, CRISPRomics allows us to systematically pinpoint the optimal nodal targets of disease with extraordinary precision and speed," said Dr. Stegmeier. "The genome-scale insights that are possible with CRISPRomics are enabling KSQ to most efficiently focus our drug discovery efforts on the development of medicines with the greatest potential to intervene in disease and, therefore, impact the lives of patients."

KSQ’s financing is led by Flagship Pioneering; Polaris Partners, ARCH Venture Partners, and Alexandria Equities also participated. Since the company’s formation in 2015, KSQ has deployed its CRISPRomics engine to complete the functional assessment of each gene in more than 600 tumor and immune models. The company has compiled new insights in disease biology to identify novel drug targets and new drug combinations designed to elicit maximal impact on disease. This approach has produced KSQ’s initial pipeline of drug development programs, and will serve as a product engine across a wide range of disease areas for the company and future collaborators. KSQ has grown to a team of more than 40 employees.

"KSQ is a pioneering company, unleashing high-confidence drug development for the first time — a step change in the way drugs are discovered and developed," said David Berry, General Partner, Flagship Pioneering. "Led by proven industry leaders, KSQ is uniquely positioned to translate its breakthrough biological insights into therapeutics that can impact the core of many important diseases."

High-Confidence Drug Development
Powered by CRISPRomics, KSQ has elucidated the function that each human gene plays in a multitude of diseases providing a unique and more comprehensive understanding of disease biology. The quality of these insights enables KSQ to identify a multitude of high-confidence, patient-tailored, novel programs for drug development and rapidly rule-out thousands of less relevant targets from the outset; thereby, focusing R&D investment on the development of medicines with the greatest potential to meaningfully impact the treatment of human disease.

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Regulation FD Disclosure

Soligenix, Inc. (the "Company") has updated its corporate presentation (the "Presentation") to include updates to the Presentation since it was last included as an exhibit to the Company’s Form 8-K filed with the U.S. Securities and Exchange on June 8, 2017 (Filing, 8-K, Soligenix, SEP 29, 2017, View Source [SID1234520714]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The Company updated the Presentation to reflect the receipt of approximately $3.7 million in non-dilutive funding. The National Institutes of Health (the "NIH") awarded the Company approximately $1.5 million over two years to support the conduct of its pivotal Phase 3 clinical trial evaluating SGX301 (synthetic hypericin) as a treatment for cutaneous T-cell lymphoma. Further, the NIH granted the Company an additional award of approximately $1.5 million over two years to support the conduct of its pivotal Phase 3 clinical trial of SGX942 (dusquetide) as a treatment for severe oral mucositis in patients with head and neck cancer receiving chemoradiation therapy. Finally, the Company will be participating in a grant awarded to the University of Hawai’i at Manoa for the development of a thermostabilized Ebola vaccine, with the Company awarded funding of approximately $700,000 over five years.

Additionally, the Company updated the estimated timeframe for the Phase 3 clinical trial of SGX301 development milestone, as its achievement may have the potential to occur later in the year.

Mylan Launches Generic Gleevec® Tablets

On September 29, 2017 Mylan N.V. (NASDAQ, TASE: MYL) reported that the U.S. launch of Imatinib Mesylate Tablets, 100 mg and 400 mg, a generic version of Novartis’s Gleevec Tablets. Mylan received final approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA) for this product, which has multiple indications, including for several blood cancers (Press release, Mylan, SEP 29, 2017, View Source [SID1234520708]).

Imatinib Mesylate Tablets, 100 mg and 400 mg, had U.S. sales of approximately $1.7 billion for the 12 months ending July 31, 2017, according to QuintilesIMS.

Mylan is one of the largest suppliers of cancer medicines by volume in the U.S., with a robust oncology portfolio of more than 40 products.

To enhance access for eligible commercially insured patients, Mylan will offer a Savings Card for Imatinib Mesylate Tablets,* which will help reduce a patient’s out-of-pocket costs. The card provides up to $700 off the monthly out-of-pocket costs for the product and is reusable up to 12 times per calendar year. Eligible patients can participate in Mylan’s Savings Card for Imatinib Mesylate Tablets program by registering online at activatethecard.com/imatinib.

Currently, Mylan has 227 ANDAs pending FDA approval, representing approximately $96.2 billion in annual brand sales, according to QuintilesIMS. Forty-five of these pending ANDAs are potential first-to-file opportunities, representing $45.5 billion in annual brand sales, for the 12 months ending July 31, 2017, according to QuintilesIMS. Currently, one out of every 13 prescriptions filled in the U.S. – brand-name or generic – is a Mylan product.

Imatinib Mesylate Tablets are a kinase inhibitor indicated for the treatment of:

Newly diagnosed adult and pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase

Patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in blast crisis (BC), accelerated phase (AP) or in chronic phase (CP) after failure of interferon-alpha therapy

Adult patients with relapsed or refractory Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL)

Adult patients with myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with PDGFR (platelet-derived growth factor receptor) gene re-arrangements as determined with an FDA-approved test

Adult patients with aggressive systemic mastocytosis (ASM) without the D816V c-Kit mutation as determined with an FDA-approved test or with c-Kit mutational status unknown

Adult patients with hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukemia (CEL) who have the FIP1L1-PDGFRα fusion kinase (mutational analysis or FISH demonstration of CHIC2 allele deletion) and for patients with HES and/or CEL who are FIP1L1-PDGFRα fusion kinase negative or unknown

Adult patients with unresectable, recurrent and/or metastatic dermatofibrosarcoma protuberans (DFSP)

Mylan Launches Generic Gleevec® Tablets

On September 29, 2017 Mylan N.V. (NASDAQ, TASE: MYL) reported that the U.S. launch of Imatinib Mesylate Tablets, 100 mg and 400 mg, a generic version of Novartis’s Gleevec Tablets. Mylan received final approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA) for this product, which has multiple indications, including for several blood cancers (Press release, Mylan, SEP 29, 2017, View Source [SID1234520708]).

Mylan (PRNewsfoto/Mylan N.V.)

Imatinib Mesylate Tablets, 100 mg and 400 mg, had U.S. sales of approximately $1.7 billion for the 12 months ending July 31, 2017, according to QuintilesIMS.

Mylan is one of the largest suppliers of cancer medicines by volume in the U.S., with a robust oncology portfolio of more than 40 products.

To enhance access for eligible commercially insured patients, Mylan will offer a Savings Card for Imatinib Mesylate Tablets,* which will help reduce a patient’s out-of-pocket costs. The card provides up to $700 off the monthly out-of-pocket costs for the product and is reusable up to 12 times per calendar year. Eligible patients can participate in Mylan’s Savings Card for Imatinib Mesylate Tablets program by registering online at activatethecard.com/imatinib.

Currently, Mylan has 227 ANDAs pending FDA approval, representing approximately $96.2 billion in annual brand sales, according to QuintilesIMS. Forty-five of these pending ANDAs are potential first-to-file opportunities, representing $45.5 billion in annual brand sales, for the 12 months ending July 31, 2017, according to QuintilesIMS. Currently, one out of every 13 prescriptions filled in the U.S. – brand-name or generic – is a Mylan product.

Imatinib Mesylate Tablets are a kinase inhibitor indicated for the treatment of:

Newly diagnosed adult and pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase

Patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in blast crisis (BC), accelerated phase (AP) or in chronic phase (CP) after failure of interferon-alpha therapy

Adult patients with relapsed or refractory Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL)

Adult patients with myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with PDGFR (platelet-derived growth factor receptor) gene re-arrangements as determined with an FDA-approved test

Adult patients with aggressive systemic mastocytosis (ASM) without the D816V c-Kit mutation as determined with an FDA-approved test or with c-Kit mutational status unknown

Adult patients with hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukemia (CEL) who have the FIP1L1-PDGFRα fusion kinase (mutational analysis or FISH demonstration of CHIC2 allele deletion) and for patients with HES and/or CEL who are FIP1L1-PDGFRα fusion kinase negative or unknown

Adult patients with unresectable, recurrent and/or metastatic dermatofibrosarcoma protuberans (DFSP)

Syros to Present Biomarker Data from Ongoing Phase 2 Clinical Trial of SY-1425 at ESH Conference on AML

On September 29, 2017 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of disease-driving genes, reported that the Company will present biomarker data from its ongoing Phase 2 clinical trial of SY-1425, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) at the European School of Haematology’s 4th International Conference on Acute Myeloid Leukemia “Molecular and Translational”: Advances in Biology and Treatment taking place October 5-7 in Estoril, Portugal (Press release, Syros Pharmaceuticals, SEP 29, 2017, View Source [SID1234520706]).

The presentation will describe Syros’ proprietary RARA and IRF8 biomarkers, including their prevalence in AML and MDS patients screened for enrollment in the Phase 2 clinical trial to date. The presentation will also include data correlating biomarker status with myeloid differentiation in cells taken from patients upon screening for the trial and then treated ex vivo with SY-1425.

Details on the presentations are as follows:

Date & Time: Friday, October 6, from 7:05 – 8:30 p.m. WEST (2:05 -3:30 p.m. EDT)
Presentation Title: Novel RARA Pathway Activation Biomarkers in Study SY-1425-201 Define a New Subset of AML and MDS Patients and Correlate with Myeloid Differentiation Following Ex Vivo SY-1425 Treatment
Session Title: Molecular Therapeutics
Presenter: Emmanuelle di Tomaso, Ph.D., Vice President, Translational Medicine, Syros
Abstract Number: 8882
Location: Estoril Congress Centre