On June 11, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that the Company has confirmed its path to submit umbralisib for accelerated approval based on data from the marginal zone lymphoma (MZL) cohort of the UNITY-NHL Phase 2b trial (Press release, TG Therapeutics, JUN 11, 2019, View Source [SID1234536991]). The Company recently had a productive Breakthrough Therapy Designation (BTD) meeting with the U.S. Food and Drug Administration (FDA) to discuss the MZL submission strategy. Based on this meeting, the Company anticipates initiating a New Drug Application (NDA) submission for patients with previously treated MZL by year-end 2019.

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Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics stated, "Marginal zone lymphoma is an incurable disease for which there are limited available therapies. As a non-chemotherapy, orally active, once daily medication, we believe umbralisib could represent a meaningful new treatment option for patients with MZL. Defining a path to a regulatory submission for umbralisib for this patient population marks an important step forward for the Company and we appreciate the guidance we have received from the FDA. We look forward to beginning the NDA process later this year, as we work expeditiously to bring umbralisib to the patients who need it." Mr. Weiss continued, "With our MZL NDA moving forward, and Phase 3 read-outs for CLL and MS targeted over approximately the next six to twelve months, we believe TG is well positioned for success."

ABOUT THE UNITY-NHL PHASE 2b STUDY—MARGINAL ZONE LYMPHOMA COHORT
The multicenter, open-label, UNITY-NHL Phase 2b study – Marginal Zone Lymphoma (MZL) cohort was designed to evaluate the safety and efficacy of single agent umbralisib, in patients with MZL who have received at least one prior anti-CD20 regimen. The primary endpoint is overall response rate (ORR) as determined by central Independent Review Committee (IRC) assessment.

The MZL cohort completed enrollment in August 2018 with a total of 69 patients enrolled and receiving at least one dose of umbralisib. In February of 2019, the Company announced that the MZL cohort met its primary endpoint of ORR as determined by central IRC for all treated patients (n=69). While the study has already met the Company’s target guidance of 40-50% ORR, the final analysis of ORR will be conducted when all treated patients have had at least 9 cycles (cycle = 28 days) of follow-up. Secondary endpoints include safety, duration of response, and progression-free survival (PFS).

ABOUT BREAKTHROUGH THERAPY DESIGNATION
The Company announced in January of 2019 that the U. S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for umbralisib for the treatment of adult patients with MZL who have received at least one prior anti-CD20 regimen.

The FDA’s Breakthrough Therapy Designation is intended to expedite the development and review of a drug candidate that is planned to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies.

On June 10, 2019 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported the addition of a new candidate to its pipeline, DCC-3116, a potential first-in-class small molecule designed to inhibit cancer autophagy, a key tumor survival mechanism. DCC-3116, discovered using the Company’s novel switch control inhibitor platform, is designed to inhibit autophagy by inhibiting the ULK kinase (Press release, Deciphera Pharmaceuticals, JUN 10, 2019, View Source [SID1234536973]). Autophagy is a cellular pathway that has been shown to be upregulated in mutant RAS cancers and that also mediates resistance to inhibitors of the RAS signaling pathway. Subject to favorable investigational new drug (IND)-enabling studies and filing and activation of an IND, Deciphera intends to develop DCC-3116 for the potential treatment of mutant RAS cancers in combination with inhibitors of downstream effector targets including RAF, MEK, or ERK inhibitors as well as with direct inhibitors of mutant RAS.

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Based on pre-clinical studies, DCC-3116 selectively inhibits ULK kinase, believed to be the initiating factor that activates autophagy. Autophagy is a cell survival pathway in which cells respond to stress by recycling their own components and/or clearing damaged organelles and proteins from the cell. Mutant RAS cancers, including KRAS, NRAS, and HRAS cancers, are reported to have high basal levels of autophagy, which they use to maintain nutrient supply, regulate cancer cell metabolism, and mitochondria surveillance.1 In multiple in vitro and in vivo models of mutant RAS cancers, autophagy inhibition combined with inhibition of MAPK signaling using MEK inhibitors or ERK inhibitors has demonstrated synergistic anti-tumor effects.2,3 When used in pre-clinical in vitro and in vivo studies in combination with inhibitors of the MAPK pathway, DCC-3116 synergized with these inhibitors to inhibit mutant RAS cancer growth. Cellular studies in mutant RAS cancers have demonstrated that MAPK pathway inhibitors further activate autophagy as a compensatory survival mechanism. Such activation of autophagy is seen with RAF, MEK, and ERK inhibitors as well as with direct inhibitors of mutant KRAS G12C. As an inhibitor of ULK, DCC-3116 is designed to address mutant RAS cancers by inhibiting the basal and compensatory autophagy that mutant RAS cancer cells use for their survival.

"We are very excited to announce our new development candidate, DCC-3116, a potential first-in-class agent aimed at treating mutant RAS cancers through the inhibition of autophagy," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "Recent efforts in the fight against cancer have focused on direct approaches targeting mutant RAS, which comprise approximately 30% of all cancers and that we believe represents one of largest unmet medical needs in oncology. We believe that as a highly selective inhibitor of ULK kinase, DCC-3116 may offer a new and complementary approach to targeting mutant RAS cancer through suppression of autophagy."

"Our new clinical candidate, DCC-3116, is a potent and selective inhibitor of ULK kinase generated using our proprietary switch control inhibitor platform. Inhibition of ULK has potential application in a very wide range of cancers and is an exciting addition to our pipeline," said Daniel Flynn, Executive Vice President, Chief Scientific Officer and Founder of Deciphera.

Deciphera is currently conducting IND-enabling studies for DCC-3116 and, pending favorable results, expects to file an IND in mid-2020.

DCC-3116 Event and Webcast Information

Deciphera will host a live event and webcast to discuss the new program on Tuesday, June 18, 2019 at 8 a.m. ET. The event will feature members of the Deciphera management team and Channing Der, Ph.D., Sarah Graham Kenan Distinguished Professor, Department of Pharmacology, UNC School of Medicine, who is a leading expert in mutant RAS cancers and autophagy.

A live audio webcast of the event and accompanying slides may be accessed through the Investors section of Deciphera’s website at www.deciphera.com. A replay of the webcast will be available for 30 days following the event.

On June 10, 2019 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with an initial focus on myelodysplastic syndromes (MDS), reported two presentations at the 24th Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) being held June 13-16, 2019 in Amsterdam, Netherlands (Press release, Onconova, JUN 10, 2019, View Source [SID1234536974]). Dr. Steven Fruchtman, President & CEO, Dr. Ric Woodman, CMO, and Dr. Ronnee Adesanya, VP Medical Affairs, will be attending the conference.

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DETAILS OF THE PRESENTATIONS:

Title: PHASE II STUDY OF ORAL RIGOSERTIB COMBINED WITH AZACITIDINE IN PATIENTS WITH HIGHER-RISK MYELODYSPLASTIC SYNDROMES (MDS)

Format: ORAL PRESENTATION: Abstract S839
Location – Elicium 1

Date/Time: Saturday, June 15 / 12:00 – 12:15

Presenter: Shyamala C. Navada, MD
Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY

Title: RIGOSERTIB MODULATES HEMATOPOIESIS GENE SIGNALING PATHWAYS ALONE OR IN SEQUENCES
COMBINATION WITH AZACITIDINE ON MDS CELLS IN VITRO

Format: POSTER PRESENTATION: Abstract PS1332
Location – Poster area

Date/Time: Saturday, June 15 / 17:30 – 19:00

Presenter: Lewis R. Silverman, MD
Tisch Cancer Institute, Division of Hematology/Oncology, Icahn School of Medicine at Mount Sinai, New York, NY
A copy of the poster and oral presentation slides will be available immediately following the event by visiting the Scientific Presentations section of Onconova’s website: View Source

On June 10, 2019 Sesen Bio (Nasdaq:SESN), a late-stage clinical company developing targeted fusion protein therapeutics for patients with cancer, reported that it has completed a successful Type B Pre-Biologics License Application (BLA) meeting regarding the approval path for Vicinium for the treatment of patients with high-risk,Bacillus Calmette-Guérin(BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC) (Press release, Xoma, JUN 10, 2019, View Source [SID1234539296]). The Company has reached alignment with the U.S. Food and Drug Administration (FDA) on an Accelerated Approval Pathway for Vicinium along with Rolling Review, and the Company expects to initiate submission of the BLA in the fourth quarter of 2019. The FDA also indicated that the nonclinical data, the clinical pharmacology data, and the safety database are sufficient to support a BLA submission, and that no additional clinical trials are necessary for a BLA submission.

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Rolling Review of the BLA enables individual modules to be submitted and reviewed on an ongoing basis, rather than waiting for all sections to be completed before submission. The final module submission for the BLA will be CMC, and Sesen Bio plans to meet with the FDA in the second half of 2019 to discuss the content and timing of that module.

"We have now had two successful meetings with the FDA over the last three weeks, which build on our long-term relationship with the Agency and make our regulatory path forward for Vicinium even more clear," said Dr. Thomas Cannell, president and chief executive officer of Sesen Bio. "Gaining alignment with the FDA on an Accelerated Approval Pathway, in addition to a rolling review of the BLA, significantly increases our confidence in our regulatory pathway and our ability to bring a product to market that has the potential to save and improve the lives of patients."

On May 21, 2019Sesen Bio announced a positive outcome from its previous meeting with the FDA, a Type C CMC meeting, where Sesen Bio reached agreement with the FDA on the Analytical Comparability Plan, and confirmed, subject to final comparability data to be provided in the BLA submission, that no additional clinical trials were deemed necessary for comparability.

Sesen Bio plans to schedule two additional meetings with the FDA in the second half of 2019, a Type C meeting to discuss the details of a post-marketing confirmatory trial in support of the Accelerated Approval Pathway for Vicinium, and a Type B CMC meeting to discuss the submission strategy of the CMC module.

Conference Call Information

To participate in the conference call, please dial (844) 831-3025 (domestic) or (315) 625-6887 (international) and refer to conference ID 2958515. The webcast can be accessed in the Investor Relations section of the company’s website at www.sesenbio.com. The replay of the webcast will be available in the investor section of the company’s website at www.sesenbio.com for 60 days following the call.

About Vicinium

Vicinium, a locally-administered fusion protein, is Sesen Bio’s lead product candidate being developed for the treatment of high-risk non-muscle invasive bladder cancer (NMIBC). Vicinium is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A. Vicinium is constructed with a stable, genetically engineered peptide tether to ensure the payload remains attached until it is internalized by the cancer cell, which is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical trials conducted by Sesen Bio, EpCAM has been shown to be overexpressed in NMIBC cells with minimal to no EpCAM expression observed on normal bladder cells. Sesen Bio is currently conducting the Phase 3 VISTA trial, designed to support the registration of Vicinium for the treatment of high-risk NMIBC in patients who have previously received a minimum of two courses of Bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive. Additionally, Sesen Bio believes that Vicinium’s cancer cell-killing properties promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. The activity of Vicinium in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.

On June 10, 2019 FibroGen, Inc. (NASDAQ: FGEN), a leading biopharmaceutical company discovering and developing a pipeline of first-in-class therapeutics, reported that Chief Executive Officer Thomas B. Neff will participate in a Fireside Chat at the Goldman Sachs 40th Annual Global Healthcare Conference in Rancho Palos Verdes, California, on Wednesday, June 12, at 10:00 a.m. Pacific Time (Press release, FibroGen, JUN 10, 2019, View Source;p=irol-newsArticle&ID=2401014 [SID1234536975]). A live audio webcast of the presentation will be available under the Investors section of the FibroGen website at www.fibrogen.com. The replay of the webcast will be available for at least 14 days following the webcast.

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