On February 25, 2020 Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, reported fourth quarter and full year 2019 financial results and provided a corporate update (Press release, Kura Oncology, FEB 25, 2020, View Source [SID1234554729]).

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"We entered the new year with three wholly owned, clinical-stage drug candidates and a focus on operational execution across our pipeline," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "The conduct of AIM-HN, our registrational-directed trial of tipifarnib in HRAS mutant head and neck squamous cell carcinoma (HNSCC), remains our highest priority. We have also taken a number of steps to better position the company for our next phase of growth, including an increasing investment in pre-commercial activities, expansion of our team of medical science liaisons and the appointment of a Chief Commercial Officer.

"Meanwhile, our growing body of clinical data in T-cell lymphomas, coupled with positive regulatory feedback and the high unmet need for these patients, support our efforts to expand the development of tipifarnib beyond our initial focus in HRAS mutant solid tumors," Dr. Wilson continued. "And we continue to make progress with our emerging drug candidates, KO-947 and KO-539, each with the potential to address genetically defined populations of cancer patients. We believe we are well positioned to advance each of our programs to meaningful inflection points, and we look forward to providing updates on our progress in the year ahead."

Recent Highlights

Strengthened leadership team to support pre-commercial efforts – In January 2020, Kura appointed Kirsten Flowers to the newly created position of Chief Commercial Officer. Ms. Flowers joined from Array Biopharma, where she served as head of commercial operations until the completion of its $11.4 billion acquisition by Pfizer in July 2019. Ms. Flowers was responsible for building and leading the commercial organization that delivered the successful launch of Braftovi + Mektovi for patients with BRAF-mutant melanoma in the U.S. Previously, she held several leadership roles at Pfizer, including U.S. commercial lead for the launches of Ibrance in breast cancer and Inlyta in renal cell carcinoma.

Fast Track designation for tipifarnib in HRAS mutant HNSCC – In December 2019, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to tipifarnib for the treatment of patients with HRAS mutant HNSCC after progression on platinum therapy. Fast Track Designation highlights the potential for tipifarnib to address unmet need for patients with this devastating disease. The designation offers the opportunity for frequent interactions with the FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, as well as eligibility for rolling submission of a New Drug Application.

Robust activity from tipifarnib in advanced T-cell lymphomas – Kura reported updated clinical data from its Phase 2 trial of tipifarnib as a monotherapy in relapsed or refractory nodal T-cell lymphomas, including angioimmunoblastic T-cell lymphoma (AITL). The data, presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2019, showed an objective response rate (ORR) of approximately 50% in heavily pre-treated AITL patients. Patients who carried mutations in the killer-cell immunoglobulin-like receptor (KIR), a CXCL pathway-associated biomarker, achieved an ORR of 70% and a complete response rate of 40%.

Positive feedback from end of Phase 2 meeting with the FDA – Kura is now preparing to initiate a registration-directed trial of tipifarnib in advanced nodal lymphomas of T-follicular helper (TFH) origin, including AITL. This single-arm trial will target enrollment of 128 patients who are relapsed or refractory to at least one prior systemic cytotoxic therapy. The trial has two independent primary objectives, ORR in all patients enrolled and ORR in patients who carry KIR mutations. Each objective can statistically be met independently. Feedback from the Company’s end-of-Phase 2 meeting with the FDA indicate that the trial, as designed, could support a New Drug Application seeking accelerated approval for tipifarnib monotherapy in lymphomas of TFH origin and/or lymphomas of TFH origin with KIR mutations.

New preclinical data support proof-of-concept study in pancreatic cancer – Kura has generated new preclinical data showing tipifarnib’s ability to block CXCL12 production by activated pancreatic stellate cells. In addition, the potential for synergy between tipifarnib and chemotherapy has been observed in preclinical models of pancreatic cancer. The Company believes these preclinical data, coupled with a previously identified association between CXCL12 expression and clinical benefit, support an upcoming proof-of-concept trial of tipifarnib in combination with chemotherapy in the second-line setting for the treatment of advanced pancreatic cancer.

Recommended Phase 2 dose defined for ERK inhibitor KO-947 – Kura believes it has defined a recommended Phase 2 dose for KO-947 as a monotherapy. Pending agreement with the FDA, the Company plans to initiate an expansion cohort to evaluate KO-947 in HNSCC and esophageal squamous cell carcinoma (ESCC) patients with 11q13 amplifications, which are genetically defined populations identified as particularly sensitive to KO-947 as a monotherapy in preclinical models. Recently, a dose-limiting serious adverse drug reaction was observed in a single patient enrolled in the Phase 1 dose-escalation trial of KO-947. Although patients were permitted to remain on therapy, the Company voluntarily paused enrollment, and the FDA placed the trial on a partial clinical hold. Kura is working to lift the hold and resume dosing at the recommended Phase 2 dose with additional safety monitoring in place.

Dose-escalation continues for menin-MLL inhibitor KO-539 –KO-539 is a first-in-class, potent and selective small molecule inhibitor of the menin-MLL protein-protein interaction. Kura commenced a Phase 1/2 clinical trial of KO-539 in relapsed/refractory acute myeloid leukemia (AML) last year and the trial continues in the dose-escalation phase. The Company’s goal is to reach a recommended Phase 2 dose or maximum tolerated dose with the potential to enrich in NPM1-mutant AML and MLL-rearranged genetically defined populations later this year.
Financial Results

Research and development expenses for the fourth quarter of 2019 were $13.5 million, compared to $12.1 million for the fourth quarter of 2018. Research and development expenses for the full year 2019 were $47.8 million, compared to $46.8 million for the prior year.

General and administrative expenses for the fourth quarter of 2019 were $5.5 million, compared to $4.6 million for the fourth quarter of 2018. General and administrative expenses for the full year 2019 were $19.7 million, compared to $16.1 million for the prior year.

Net loss for the fourth quarter of 2019 was $17.9 million, compared to a net loss of $16.1 million for the fourth quarter of 2018. Net loss for the full year 2019 was $63.1 million, compared to a net loss of $60.4 million for the prior year. Net loss for the fourth quarter and full year of 2019 included non-cash, share-based compensation expense of $2.4 million and $9.4 million, respectively, compared to $1.7 million and $8.7 million for the same periods in 2018, respectively.

Cash, cash equivalents and short-term investments totaled $236.9 million as of December 31, 2019, compared with $179.0 million as of December 31, 2018. Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund its current operations into 2022.
Upcoming Milestones

Additional data from Phase 2 trials of tipifarnib in HRAS mutant solid tumors, including HRAS mutant urothelial carcinoma, in 2020

Initiation of a registration-directed trial of tipifarnib in AITL in the second half of 2020

Initiation of a proof-of-concept trial of tipifarnib in pancreatic cancer in the second half of 2020

Additional data from Phase 2 trial of tipifarnib in chronic myelomonocytic leukemia (CMML) in 2020

Potential for full enrollment in the AIM-HN registration-directed trial of tipifarnib in HRAS mutant HNSCC in the first quarter of 2021

Open expansion cohort of patients with HNSCC and ESCC with 11q13 amplifications in Phase 1 trial of KO-947 in 2020

Achievement of a recommended Phase 2 dose in the Phase 1/2 dose-escalation trial of KO-539 by the end of 2020
Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 4:30 p.m. ET / 1:30 p.m. PT today, February 25, 2020, to discuss the financial results for the fourth quarter and full year 2019 and provide a corporate update. The live call may be accessed by dialing (877) 516-3514 for domestic callers and (281) 973-6129 for international callers and entering the conference code: 8077975. A live webcast of the call will be available from the Investors and Media section of the Company’s website at www.kuraoncology.com, and will be archived there for 30 days.

On February 25, 2020 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies (tumor-infiltrating lymphocyte, TIL and peripheral-blood lymphocyte, PBL), reported fourth quarter and year-end 2019 financial results and provided a corporate update (Press release, Iovance Biotherapeutics, FEB 25, 2020, View Source [SID1234554728]).

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"During 2019 we made tremendous progress in advancing Iovance TIL and PBL products," said Maria Fardis, Ph.D., MBA, Iovance President and Chief Executive Officer. "We conducted two planned pivotal programs for lifileucel in melanoma and LN-145 in cervical, initiated patient dosing in earlier lines of therapy and received clearance from FDA on a new IND to proceed to dose patients with our PBL product, IOV-2001. In anticipation for commercialization of Iovance TIL, we are building our internal manufacturing capability and expanding our commercial team and infrastructure. With the first potential cell therapy in solid tumors, as well as a broad TIL platform, Iovance is poised to become the leader in development, manufacturing, and commercialization of TIL cell therapy for solid tumors."

2019 Highlights and 2020 Updates

Clinical

·Initiation and completion of patient dosing in the pivotal melanoma program, cohort 4 in the C-144-01 trial: Patient dosing in the pivotal cohort 4 of the C-144-01 study was initiated in March 2019 and completed in January 2020, three months ahead of schedule. Iovance intends to submit a biologics license application (BLA) to FDA subsequent to consultation with the agency in 2020.
·Presentation of data for lifileucel in metastatic melanoma and LN-145 for metastatic cervical cancer at major medical meetings: Several clinical data presentations continued to demonstrate efficacy and safety of lifileucel in metastatic melanoma (from C-144-01 study) and LN-145 in recurrent, metastatic, or persistent cervical cancer (from C-145-04 study).
oMelanoma update
·The data for the C-144-01 study in melanoma demonstrated a 36.4% ORR by investigator as presented at ASCO (Free ASCO Whitepaper).1
·In a recent data cut for the C-144-01 study, a median duration of response (DOR) was not reached at 15.5 months of median study follow up (Jan. 2020).2
·Expansion of TIL treatment to earlier lines of therapy for melanoma, head and neck, and non-small cell lung cancer (NSCLC) patients. Patient dosing in the IOV-COM-202 clinical study was initiated in May 2019 to evaluate TIL plus pembrolizumab in patients with immune checkpoint inhibitor naïve melanoma, squamous cell carcinoma of the head and neck (HNSCC), and NSCLC. In the fourth cohort, LN-145 alone is offered to relapsed/refractory NSCLC patients.

Regulatory

·Receipt of Breakthrough Therapy designation (BTD), Fast Track designation and holding End of Phase 2 Meeting for LN-145 in cervical cancer: LN-145 was granted Fast Track and BTD from FDA in recurrent, metastatic or persistent cervical cancer with disease progression on or after chemotherapy. Following an end of Phase 2 meeting, the ongoing C-145-04 clinical study was expanded to dose 75 patients to support registration of LN-145. Additional cohorts in earlier and later line cervical cancer patients were added to study C-145-04 in anticipation of a changing landscape in this indication, specifically, a cohort allowing patients post anti-PD-1 was added.
·Clearance of the investigational new drug (IND) application by FDA and initiation of patient dosing for PBL (IOV-2001) therapy in the first hematologic indication, CLL: An IND application for IOV-2001 was accepted by FDA in the blood cancer indication, relapsed or refractory CLL or small lymphocytic lymphoma (SLL). The first patient was dosed in a Phase 1/2 study (IOV-CLL-01 study).

Manufacturing

·Continuation of high manufacturing success rate: The Gen 2 TIL therapy manufacturing process continues to be robust with a demonstrated success rate, as measured from the receipt of the starting material to the shipment of TIL product, of well over 90 percent in approximately 300 patients.
·Clinical introduction of Gen 3 TIL therapy: A proprietary 16-day, third generation TIL therapy manufacturing process (Gen 3) entered the clinic for initial evaluation in the expanded C-145-03 clinical study in head and neck cancer patients with significant unmet need, for which shorter time to TIL infusion may offer a potential benefit.
·Clinical introduction of PD-1 selected TIL therapy (LN-145-S1): A proprietary, selected TIL product LN-145-S1, aimed at taking further advantage of TIL’s ability to recognize the tumor, is also entering the clinic for initial evaluation in the expanded C-145-03 study in head and neck cancer, and subsequently in a new cohort in the IOV-COM-202 study.

Corporate

·Iovance manufacturing facility on track for commercial production in 2022: Iovance began construction of a state-of-the-art, 136,000 square foot commercial-scale production facility in Philadelphia for its TIL therapies in June 2019. The new facility is expected to be completed by year-end 2021 to support commercial supply in 2022, with capacity to meet demand for thousands of patients in multiple cancers. Iovance expects to invest approximately $85 million over three years for equipment and construction of the facility.
·The National Cancer Institute (NCI), with support from Iovance, received an award for TIL therapy technology transfer: NCI won the Federal Laboratory Consortium’s 2020 Excellence In Technology Transfer Award for "New, First-in-class Immunotherapy, for Treatment of Recurrent, Metastatic Cervical Cancer" for its collaboration with Iovance. Iovance supported NCI’s application for the award and remains a CRADA partner for ongoing research in TIL therapy.3
·New Iovance patents issued: Iovance has been granted or allowed a total of 10 U.S. patents for compositions and methods of treatment in a broad range of cancers related to its 22-day Gen 2 manufacturing process.

Research and Development

·Licensing of novel IL-2 analog (IOV-3001): Iovance obtained a license from Novartis to develop and commercialize an expectedly better IL-2 analog, an antibody cytokine engrafted protein, referred to as IOV-3001, as a targeted and selective IL-2 analog.
·Licensing of TALEN technology to develop gene edited TIL: Under a research collaboration and exclusive worldwide license agreement with Cellectis, Iovance licensed certain Cellectis TALEN technology to develop genetically edited TIL to potentially create more potent therapeutics in several cancer indications. The worldwide exclusive license enables Iovance’s use of TALEN technology addressing multiple gene targets to modify TIL for therapeutic use.

Anticipated 2020 Milestones

·Last patient dosed in the pivotal program of LN-145 for cervical cancer
·Pre-BLA Meeting with U.S. FDA
·Melanoma top-line pivotal data
·BLA submission

Fourth Quarter and Full-Year 2019 Financial Results

Net loss for the fourth quarter ended December 31, 2019, was $63.6 million, or $0.50 per share, compared to a net loss of $32.6 million, or $0.27 per share, for the fourth quarter ended December 31, 2018. Net loss for the full-year ended December 31, 2019, was $197.6 million, or $1.59 per share, compared to a net loss of $123.6 million, or $1.27 per share, for the full-year ended December 31, 2018.

Research and development expenses were $54.2 million for the fourth quarter ended December 31, 2019, an increase of $26.8 million compared to $27.4 million for the fourth quarter ended December 31, 2018. Research and development expenses were $166.0 million for the 12 months ended December 31, 2019, an increase of $66.2 million compared to $99.8 million for the prior year period. The increases in fourth quarter and full year 2019 over the prior year periods were primarily attributable to an increase in costs associated with manufacturing activities and capacity, clinical trials due to higher enrollment, and growth of the internal research and development team.

General and administrative expenses were $10.9 million for the fourth quarter 2019, an increase of $3.4 million compared to $7.5 million for the fourth quarter 2018. General and administrative expenses were $40.8 million for the 12 months ended December 31, 2019, an increase of $12.4 million compared to $28.4 million for the full year ended December 31, 2018. The increases in fourth quarter and full year 2019 over the prior year periods were primarily attributable to growth of the internal general and administrative team, as well as higher intellectual property legal costs and market research activities in preparation for commercialization.

Cash, cash equivalents, short term investments and restricted cash

At December 31, 2019, the company held $312.5 million in cash, cash equivalents, short-term investments and restricted cash compared to $468.5 million at December 31, 2018.

Webcast and Conference Call

Iovance will host a conference call today at 4:30 p.m. ET to discuss fourth quarter and full-year 2019 results and provide a corporate update. The conference call dial-in numbers are 1-844-646-4465 (domestic) or 1-615-247-0257 (international). The conference ID access number for the call is 4693108. The live webcast can be accessed in the Investors section of the company’s website at View Source The archived webcast will be available for a year in the Investors section at www.iovance.com.

On February 25, 2020 Heron Therapeutics, Inc. (Nasdaq: HRTX), a commercial-stage biotechnology company focused on improving the lives of patients by developing best-in-class treatments to address some of the most important unmet patient needs, reported that Barry Quart, Pharm.D., President and Chief Executive Officer of Heron Therapeutics, will present at the 40th Annual Cowen Healthcare Conference on Monday, March 2nd, 2020 at 12:00 p.m. ET at the Boston Marriott Copley Place hotel (Press release, Heron Therapeutics, FEB 25, 2020, View Source [SID1234554727]).

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A live webcast of the presentation will be available on the Company’s website at www.herontx.com in the Investor Resources section. A replay of the presentation will be archived on the site for 60 days.

On February 25, 2020 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that Chief Executive Officer Rachel King will provide a company overview at the Cowen and Company 40th Annual Health Care Conference in Boston, on Tuesday, March 03, 2020, at 12:00 p.m. ET (Press release, GlycoMimetics, FEB 25, 2020, https://ir.glycomimetics.com/news-releases/news-release-details/glycomimetics-present-cowen-and-company-40th-annual-health-care [SID1234554726]).

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To access the live webcast and subsequent archived recordings for the presentation, please visit the GlycoMimetics website at www.glycomimetics.com.

On February 25, 2020 KAHR Medical Ltd., a biopharmaceutical company developing a novel drug platform based on bi-functional, immunotherapeutic fusion proteins known as Dual Signaling Proteins ("DSP") reported that it has raised US$18 million from a global syndicate of leading investors (Press release, KAHR Medical, FEB 25, 2020, View Source [SID1234554725]). Completion of the financing round is subject to customary closing conditions and is expected to occur early next month.

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The round was led by Flerie Invest AB, Oriella Limited, Hadasit Bio-Holdings (HBL), Pavilion Capital and Mirae Asset Venture Investment. Proceeds will be used for advancing the Company’s next generation immuno-oncology drug candidates including the clinical development of the Company’s lead product, DSP107, an anti-CD47 therapy for the treatment of solid tumors through a Phase I/II study and the preclinical advancement of additional pipeline projects.

In September 2019, KAHR Medical announced a clinical collaboration with Roche to evaluate KAHR Medical’s lead program, DSP107, a SIRPα-41BBL DSP, in combination with Roche’s PD-L1-blocking checkpoint inhibitor atezolizumab (Tecentriq) in patients with advanced NSCLC who are refractory to existing immune checkpoint inhibitors. KAHR Medical expects to begin a Phase I/II trial in H2 2020 at leading sites in the US to evaluate DSP107 as a monotherapy and in combination with atezolizumab, following the filing of an Investigational New Drug (IND) application with the U.S. Food Drug Administration (FDA).

"We are grateful to our existing investors for their continued support and are pleased to welcome Oriella, a prominent private equity and technology investor in Israel, with an expanding focus on life sciences," said Yaron Pereg, Ph.D., CEO of KAHR Medical. "We are also proud to have Pavilion Capital join our investor base. Pavilion Capital brings a track record of success and expertise in the biopharmaceutical sector. We look forward to using this funding for advancing our next-generation immuno-oncology pipeline for the benefit of patients, who are non-responsive or refractory to existing immunotherapies."

"We are excited to lead this financing round for KAHR Medical together with other distinguished investors," said Thomas Eldered, Chairman of Flerie Invest AB. "Despite advances in the treatment of cancer, there is still a clear need for additional therapies to broaden the patient population that will respond to cancer immunotherapies. We look forward to supporting KAHR Medical in developing effective treatments for cancer patients."

Mr Vincent Tchenguiz, a British entrepreneur and beneficiary of the trust that owns Oriella commented, "We recognize the strength of KAHR Medical’s proprietary platform and believe that the company’s products have the potential to offer unique value to patients suffering from cancer. We are keen to work with the Company as it matures, and we look forward to helping it fulfil its mission of bringing new therapies to cancer patients."

Timothy Low, Head of Healthcare Investments, Pavilion Capital, said, "We are delighted to join KAHR Medical’s syndicate of investors, and believe the company has a tremendous opportunity to contribute new targeted therapies to the field of immunooncology."

Michel Habib, CEO of HBL, stated, "HBL has been supporting KAHR Medical since its inception and we are very pleased with the company’s impressive development.

We are proud to continue to support the company together with other existing and new global investors and look forward with anticipation to the beginning of clinical trials."