On November 14, 2019 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that Scott Wolchko, President and Chief Executive Officer, will present at the Jefferies 2019 London Healthcare Conference in London on Thursday, November 21, 2019, at 4:40 p.m. GMT (Press release, Fate Therapeutics, NOV 14, 2019, View Source [SID1234551285]).

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A live webcast of the presentation will be available through the investor relations section of the Company’s website at www.fatetherapeutics.com. Following the live webcast, an archived replay will be available on the Company’s website.

On November 14, 2019 ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, reported its operational and financial results for the three and nine months ended September 30, 2019 (Press release, ProMIS Neurosciences, NOV 14, 2019, View Source [SID1234551302]).

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"Over the course of 2019, the breadth and depth of our unique discovery and development platform was further evidenced as ProMIS made considerable progress in expanding its portfolio of opportunities in neurodegenerative diseases," stated Dr. Elliot Goldstein, ProMIS President and CEO. "In the third quarter of this year, we continued to generate further data supporting our antibody candidates for Parkinson’s disease (targeting toxic forms of alpha-synuclein), amyotrophic lateral sclerosis (targeting toxic aggregates of TDP-43) and for Alzheimer’s disease (targeting toxic forms of tau)."

Narrated updates relating to ProMIS’ unique approach and capabilities can be found on the ProMIS website by clicking on the links below:

Click here for strategy overview memorandum from Executive Chairman Eugene Williams: http://bit.ly/ProMIS102319
Click here for Chief Scientific Officer Dr. Neil Cashman’s overview of ProMIS’ unique capability to design and develop antibodies selectively targeting toxic mis-folded proteins that are root causes of neurodegenerative diseases: http://bit.ly/ProMIS2KQ88la
Click here for Chief Development Officer, Dr. Johanne Kaplan’s narrated overview of "Best in Class" therapy for misfolded protein diseases: http://bit.ly/ProMIS110719
Corporate Highlights

On November 13, 2019, subsequent to period-end, the company announced a non-brokered private placement for gross proceeds of up to $6.5 million. A first closing in the amount of $2,055,000 gross proceeds will occur on November 15, 2019.
In July 2019, the Company announced the voting results of the Corporation’s annual meeting of shareholders held on June 27, 2019 in Toronto, Ontario. All of the resolutions announced in the Management Proxy Circular and placed before the Meeting were overwhelmingly approved by the shareholders. All Directors were elected, with each nominee receiving more than 75% of the votes cast.
In July 2019, supporting data for PMN310, the Company’s lead antibody candidate for Alzheimer’s disease (AD) were published in Scientific Reports, a journal of the Nature Research family. In the manuscript, "A Rationally Designed Humanized Antibody Selective for Amyloid Beta Oligomers in Alzheimer’s Disease," the authors demonstrate PMN310’s selectivity for toxic amyloid-beta oligomers (AßOs), a root cause of AD. The results of activity assays show that PMN310 inhibits both the spread and toxicity of AßOs in vitro, and, in mouse studies, that PMN310 prevents AßO-induced loss of memory formation and reduces both synaptic loss and inflammation. PMN310 compared favorably to other Aß-directed antibodies, showing a lack of adverse event-associated binding to Aß plaque.
In July 2019, the Company presented an update on PMN310 for Alzheimer’s disease at the annual Alzheimer’s Association International Conference (AAIC) in Los Angeles. In an oral presentation, Chief Development Officer, Dr. Johanne Kaplan highlighted the therapeutic potential of PMN310 against the toxic oligomer form of AßO, a root cause of AD. Dr. Neil Cashman, ProMIS Chief Scientific Officer delivered data derived from ProMIS’ preclinical program for ALS in a poster presentation demonstrating the role of toxic, misfolded TAR-DNA binding protein 43 (TDP-43) as a root cause of neurogenerative diseases such as ALS and frontotemporal dementia (FTD).
Scientific Advisory Board Appointment

In August 2019, the Company appointed Dr. Andre Strydom to its scientific advisory board (SAB). Dr. Strydom is a world-recognized expert in ageing-related issues in Down syndrome and his research has advanced understanding of AD in Down syndrome patients. His expertise and advocacy will help guide ProMIS development plans relating to treatment of AD in Down syndrome. He is a professor in the Institute of Psychiatry, Psychology and Neuroscience at King’s College London, and Honorary Consultant psychiatrist, South London and the Maudsley NHS Trust.

Financial Results

Results of Operations – Three months ended September 30, 2019 and 2018

Net loss for the three months ended September 30, 2019 was $1,637,714 compared to a net loss of $2,911,981 for the three months ended September 30, 2018, respectively. Included in the net loss amount for the three months ended September 30, 2019 were non-cash expenses of $134,634, representing share-based compensation and amortization of an intangible asset, compared to $385,951 for the three months ended September 30, 2018. The decrease in the net loss in the three months ended September 30, 2019 reflects decreased costs associated with external contract research organizations for internal programs, consultant salaries and associated costs, patent costs and share-based compensation offset by increased general corporate expenditures.

Research and development expenses for the three months ended September 30, 2019 were $1,053,123, as compared to $1,867,648 in the three months ended September 30, 2018. The decrease in the research and development expenses for the three months ended September 30, 2019, compared to the same periods ended September 30, 2018, is primarily attributed to decreased costs associated external contract research organizations for internal programs and patent costs offset by increased external consulting costs and share-based compensation.

General and administrative expenses for the three months ended September 30, 2019 were $584,602, as compared to $1,044,596 in the three months ended September 30, 2018. The decrease for the three months ended September 30, 2019, compared to the same periods ended September 30, 2018, is primarily attributable to decreased consultant salaries and associated costs, general corporate expenditures and share-based compensation.

Results of Operations – Nine months ended September 30, 2019 and 2018

Net loss for the nine months ended September 30, 2019 was $5,942,821, compared to a net loss of $6,683,714 for the nine months ended September 30, 2018, respectively. Included in the net loss amount for the nine months ended September 30, 2019 were non-cash expenses of $551,968, representing share-based compensation and amortization of an intangible asset, compared to $888,506 for the nine months ended September 30, 2018. The decrease in the net loss in the nine months ended September 30, 2019 reflects decreased costs associated with external contract research organizations for internal programs, patent costs and share-based compensation offset by increased consultant salaries and associated costs and general corporate expenditures.

Research and development expenses for the nine months ended September 30, 2019 were $3,866,394, as compared to $4,096,729 in the nine months ended September 30, 2018. The decrease in the research and development expenses for the nine months ended September 30, 2019, compared to the same periods ended September 30, 2018, is primarily attributed to decreased spending on external contract research organizations for internal programs and reduced patent expense offset by increased contracted research salaries and associated costs, external consulting expense and share-based compensation.

General and administrative expenses for the nine months ended September 30, 2019 were $2,076,463, as compared to $2,587,253 in the nine months ended September 30, 2018. The decrease for the nine months ended September 30, 2019, compared to the same periods ended September 30, 2018, is primarily attributable to decreased share-based compensation offset by increased consultant salaries and associated costs, general corporate expenditures and foreign exchange.

Outlook

The Company will continue to build on its unique, proprietary discovery and development platform to further characterize the potential benefits of its programs selectively targeting toxic aggregates of TDP-43 and SOD1 in ALS, toxic forms of alpha-synuclein ( in PD and other related disorders, and toxic forms of tau and amyloid beta in AD and other dementias to further support ongoing pharmaceutical partnering discussions.

On November 14, 2019 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported the presentation of interim clinical data from its ongoing Phase 1 clinical trial evaluating TK216, a first-in-class, targeted, investigational small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins, in patients with relapsed or refractory Ewing sarcoma (Press release, Oncternal Therapeutics, NOV 14, 2019, View Source [SID1234551319]). Paul A. Meyers, M.D., Chief, Pediatric Sarcoma Service and Vice Chair for Clinical Affairs of Memorial Sloan Kettering Cancer Center, reported at the Connective Tissue Oncology Society (CTOS) 2019 Annual Meeting in Tokyo, Japan that one of two patients treated in the current, highest exposure dose cohort of Study TK216-01 is without evidence of Ewing sarcoma after eight months on study, and has tolerated treatments with TK216 alone or combined with vincristine well.

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"We are encouraged by this first reported deep and sustained clinical response to TK216 in a patient with Ewing sarcoma in the dose-finding portion of our clinical trial and look forward to further evaluating the recommended Phase 2 dose regimen of TK216 in a larger number of patients with this devastating disease," said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. "This first-in-class investigational agent may also be applicable in other malignancies driven by ETS alterations including AML and prostate cancer, which we continue to explore in preclinical studies."

Study TK216-01, Oncternal’s ongoing Phase 1 study of TK216 in patients with relapsed or refractory Ewing sarcoma, is a first-in-human, multicenter clinical trial. Trial objectives include the evaluation of safety, tolerability, pharmacokinetics and tumor response. Thirty-two patients have been treated in the dose-finding part of the trial. Patients entering the trial had previously been treated with a median of four, and up to nine prior lines of systemic therapy. TK216 has been generally well tolerated in this trial, with common side effects including myelosuppression, fatigue, nausea and alopecia. The maximum tolerated dose has been identified as 220 mg/m2/day for a seven-day treatment regimen, and 200 mg/m2/day for a 10-day regimen. Dose limiting toxicities consisted of transient and manageable myelosuppression, primarily neutropenia. No unexpected off-target toxicities have been observed.

One of two patients treated at the current, highest exposure dose regimen (200 mg/m2/day for 14 days) has experienced a deep and sustained clinical response to TK216. Multiple lung nodules regressed following two cycles of TK216 alone. After six months of treatment that included concomitant vincristine starting in the third cycle, a single 7 mm lung nodule was resected, resulting in a surgical complete remission. The patient remains with no evidence of disease after eight months of treatment. TK216, with or without vincristine, has been well tolerated by this patient, with only minimal myelosuppression. Clinical pharmacology data suggest that this dosing regimen may result in drug levels that meet or exceed those that killed tumor cells in vitro and inhibited tumor growth in animal models.

Enrollment of the final dose-finding cohort of the study is nearing completion, after which Oncternal intends to begin enrolling patients using the recommended Phase 2 dosing regimen in an expansion cohort to further evaluate the clinical response to treatment.

"I am encouraged by the depth and duration of response to TK216 in this heavily pre-treated patient with Ewing sarcoma, and that the patient tolerated his treatments with TK216 and vincristine well," said Dr. Meyers. "Advanced, refractory Ewing sarcoma is a serious and devastating condition, and novel therapies are desperately needed. These interim results of Study TK216-01, along with evolving preclinical data, suggest that this agent warrants further clinical testing in Ewing sarcoma as well as in other cancer indications."

About TK216

TK216 is an investigational, potentially first-in-class, targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins including fusion proteins. Tumorigenic fusion proteins involving the EWS protein and an ETS protein can be found in most cases of Ewing sarcoma. ETS-related translocations or overexpression are also found in many other tumors such as prostate cancer and acute myeloid leukemia (AML). TK216 was developed based on discoveries in the laboratory of Jeffrey Toretsky, M.D., at Georgetown Lombardi Comprehensive Cancer Center, who discovered inhibitors of EWS-FLI1 using a novel chemical screening assay. In preclinical models, TK216 was observed to bind to EWS-FLI1, blocking the interaction between this fusion protein and other transcriptome proteins such as RNA helicase A, leading to tumor cell apoptosis and inhibiting tumor growth in animal models. The U.S. Food and Drug Administration (FDA) has granted Orphan Designation and Fast Track designation to TK216 for the treatment of Ewing sarcoma. TK216 is an investigational medication that has not been approved by the FDA for any indication.

About the Study

TK216 is being evaluated in a Phase 1 clinical study as a single agent and in combination with vincristine in patients with relapsed or refractory Ewing sarcoma, a rare pediatric cancer with no standard treatment available after first-line chemotherapy. The dose-finding portion of the study is nearing completion, after which Oncternal intends to begin enrolling patients in an expansion cohort to evaluate the clinical response of treatment with TK216 in combination with vincristine using the recommended Phase 2 dosing regimen. This multi-center study is actively enrolling patients at seven clinical trial centers across the U.S. Additional information about the TK216 study may be accessed at ClinicalTrials.gov (NCT02657005).

On November 14, 2019 Leap Therapeutics, Inc. (NASDAQ:LPTX) reported financial results for the third quarter ended September 30, 2019 (Press release, Leap Therapeutics, NOV 14, 2019, View Source [SID1234551338]).

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"The body of clinical data we presented in the third quarter for both DKN-01 monotherapy and combination treatment for cancer patients continues to demonstrate impressive activity. Patients with advanced gastroesophageal junction and gastric cancer whose tumors expressed high levels of DKK1 (DKK1-high) achieved higher survival and objective response outcomes to the combination of DKN-01 and KEYTRUDA," commented Christopher K. Mirabelli, Ph.D., President and Chief Executive Officer of Leap. "DKN-01 also showed durable benefit in patients with endometrial cancer with Wnt pathway alterations, including a monotherapy complete response, highlighting the potential utility of DKN-01 for biomarker-targeted patient populations."

Dr. Mirabelli continued: "We also completed enrollment in the dose escalation phase of our clinical trial evaluating TRX518 in combination with BAVENCIO and cyclophosphamide; however, we’ve made the strategic decision to deprioritize further development of TRX518 at this time in order to focus our resources on our more advanced DKN-01 program. The safety profile observed to date was acceptable, and patients who are benefiting from treatment in the TRX518 program will continue to be treated."

DKN-01 Development Program Update

DKN-01 in ESOPHAGOGASTRIC CANCER: Leap presented data from the KEYNOTE-731 clinical study evaluating DKN-01 in combination with KEYTRUDA (pembrolizumab) in patients with advanced esophagogastric cancer. Study results demonstrated that patients with DKK1-high status had improved outcomes, including longer progression free survival (PFS) independent of PD-L1 Combined Positive Scores (CPS). In ten evaluable gastroesophageal junction and gastric cancer patients who had not received prior PD-1/PD-L1 therapy, DKK1-high patients experienced 22.1 weeks median progression free survival (PFS) and 31.6 weeks median overall survival (OS), with a 50% overall response rate (ORR) and 80% disease control rate (DCR). Fifteen evaluable DKK1-low patients experienced 5.9 weeks PFS and 17.4 weeks OS, with a 20% DCR. PD-L1 CPS did not predict efficacy to the combination of DKN-01 plus KEYTRUDA.
DKN-01 in GYNECOLOGICAL CANCERS: The Company presented data from the ongoing clinical study of DKN-01 as a monotherapy and in combination with paclitaxel in patients with advanced gynecological cancers at the International Gynecologic Cancer Society Annual Global Meeting held in September. In the cohort of sixteen evaluable monotherapy patients with epithelial endometrial cancer (EEC) with identified Wnt signaling mutations, patients had higher response rates and demonstrated longer PFS as compared to patients without Wnt signaling mutations. Specifically, one patient had a complete response and one patient had a partial response, representing a 12.5% single agent ORR, seven patients had a best response of stable disease, and seven patients had progressive disease. In the six evaluable monotherapy EEC patients who did not have any identified Wnt signaling mutations, none had clinical benefit. Patient follow-up is continuing in this study, which has been expanded to include focused cohorts of patients with carcinosarcoma.
DKN-01 plus OPDIVO in BILIARY TRACT CANCER: The first patients have been dosed in an investigator-initiated clinical study to evaluate DKN-01 in combination with Bristol-Myers Squibb’s OPDIVO (nivolumab) in previously treated patients with advanced biliary tract cancer. The study is being conducted by Massachusetts General Hospital and will enroll up to 36 biliary tract cancer patients who have progressed after one or more lines of systemic therapy for advanced biliary tract cancer. The primary endpoint of the study will be ORR, to be assessed in the overall population as well as in subgroups stratified by tumor DKK1 and PD-L1 expression. Bristol-Myers Squibb is providing OPDIVO drug supply and partial funding for the study, with Leap providing DKN-01 drug supply as well as additional partial funding.
TRX518 Development Program Update

FURTHER DEVELOPMENT OF TRX518 HAS BEEN DEPRIORITIZED: Leap has completed enrollment in dose escalation phase of the clinical trial evaluating TRX518 in combination with cyclophosphamide chemotherapy and BAVENCIO (avelumab). However, instead of pursuing additional enrollment through the expansion cohorts in this study as initially planned, the Company has decided to reprioritize resources on the further development of the DKN-01 program. There were no safety or efficacy concerns leading to this decision, and patients who are benefitting from the combination therapy will continue to be treated in the study.
Selected Third Quarter 2019 Financial Results

Net loss was $7.9 million for the third quarter 2019, compared to $6.6 million for the same period in 2018. This increase was primarily due to the recording of a $1.8 million gain in the third quarter 2018 as a result of a change in the fair value of the warrant liability, partially offset by a decrease in research and development expense.

Research and development expenses were $5.8 million for the third quarter 2019, compared to $6.5 million for the same period in 2018. This decrease was primarily due to a decrease of $0.4 million in clinical trial costs as a result of the timing of patient enrollment and a decrease of $0.3 million in manufacturing costs related to clinical trial material manufacturing campaigns.

General and administrative expenses were $2.2 million for the third quarter 2019, compared to $2.1 million for the same period in 2018. The increase was primarily due to a $0.1 million increase in stock based compensation as a result of new stock options granted to employees and directors in 2019.

Cash, cash equivalents and marketable securities totaled $10.1 million at September 30, 2019. Research and development incentive receivables, short term, totaled approximately $752,000 at September 30, 2019.

On November 14, 2019 Incyte Corporation (Nasdaq:INCY) reported that it will present at the Piper Jaffray 31st Annual Healthcare Conference on Wednesday, December 4, 2019 at 8:30 am (EST) in New York (Press release, Incyte, NOV 14, 2019, View Source [SID1234551241]).

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The presentation will be webcast live and can be accessed at www.incyte.com under For Investors, Events and Presentations and will be available for replay for 30 days. Investors interested in listening to the live webcast should log on before the start time in order to download any software required.