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TRACON Announces Submission Of Request For Type B Meeting With FDA To Discuss Trial Design For ENVASARC: A Potential Pivotal Study Of Envafolimab In Sarcoma

On March 16, 2020 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and utilizing a product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that it has submitted a request for a Type B meeting with the U.S. Food and Drug Administration (FDA) to discuss the trial design for a potential pivotal study of envafolimab in sarcoma (ENVASARC) (Press release, Tracon Pharmaceuticals, MAR 16, 2020, View Source [SID1234555614]).

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TRACON plans to propose a pivotal trial with two cohorts of approximately 80 patients each to assess the objective response rate in sarcoma subtypes known to be responsive to checkpoint inhibition, with one cohort receiving single-agent envafolimab and the second cohort receiving envafolimab and Yervoy (ipilimumab).

"We have taken the first step towards beginning the ENVASARC study of envafolimab in sarcoma subtypes known to respond to checkpoint inhibitors. This initiates a series of expected events that we anticipate will culminate in dosing the first patient in a potential pivotal trial in the second half of the year," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "We believe the proposed study design that utilizes two cohorts provides for greater treatment options for patients and lowers envafolimab’s clinical risk by providing two potential pathways for approval."

Expected Upcoming Envafolimab Milestones Over the Next 6 Months

Type B meeting with the FDA to discuss the potential pivotal trial design of ENVASARC for envafolimab
File IND for envafolimab to conduct the planned ENVASARC study
Apply for orphan drug designation for envafolimab in soft tissue sarcoma
Submission of regulatory approval for envafolimab in China by our partners, 3D Medicine and Alphamab Oncology
Presentation of envafolimab clinical data at ASCO (Free ASCO Whitepaper) by our partners 3D Medicine and Alphamab Oncology
Enroll the first patient in ENVASARC, a potential pivotal trial of envafolimab
About Envafolimab

Envafolimab is a novel, single-domain antibody against PD-L1 that is administered by subcutaneous injection without the need for an adjuvant. Envafolimab is currently being dosed in Phase 1 trials in the U.S., China and Japan, a Phase 2 registration trial as a single agent in MSI-H tumor patients in China, and in a Phase 3 registration trial in biliary tract cancer in combination with gemcitabine and oxaliplatin in China. Subject to positive data from the MSI-H registrational trial, 3D Medicines has stated that it plans to file a BLA in China for envafolimab in 2020 based on overall response rate in MSI-H patients. The filing is predicated on the principle that the response rate required for approval in China is similar to the response rate for Keytruda and Opdivo in MSI-H patients from separate clinical trials per the product package inserts.

Varian to Equip New Ethos Personalized Adaptive Therapy Center in Phoenix

On March 16, 2020 Varian (NYSE: VAR) reported it was selected to equip the new Ethos Personalized Adaptive Therapy Center in Phoenix, Arizona, and help launch a new era in cancer treatment for patients in the region (Press release, Varian Medical Systems, MAR 16, 2020, View Source [SID1234555613]). Ethos therapy provides the ability to personalize the patient’s radiation treatments based on their anatomy and the tumor’s shape and position on a daily basis at the time of treatment. The goal is to better target the tumor, reduce dose to healthy tissue, and potentially improve overall patient outcomes.

This new center will be equipped with Varian’s Ethos therapy. This artificial intelligence (AI)-driven holistic adaptive therapy solution is designed to deliver an entire adapted treatment for daily personalized radiation therapy in a typical 15-minute timeslot.

"We are excited to be opening this new center to bring a new level of personalized care to all cancer patients in Arizona and proud to be among the first clinics to offer Ethos therapy in the United States," said John Kresl MD PhD, senior partner and medical director at Palo Verde Cancer Specialists and Phoenix CyberKnife Center. "Ethos therapy will continue to enable our physicians to be the leaders in providing the latest state-of-the-art tumor focused radiation therapy by better visualizing the daily changes in a patient’s anatomy and quickly adapt their therapy right on the spot, thereby potentially providing better treatments to our patients. We initially plan to use Ethos therapy for pancreas, prostate, breast, head & neck, and lung cancer treatments."

"Every patient deserves personalized cancer care," said Chris Toth, president Varian Oncology Systems. "The launch of Ethos therapy was a transformational moment for cancer care, and we are honored to be working with this new center to bring these adaptive treatments to patients in Arizona and help us move closer to creating a world without fear of cancer."

Varian Halcyon 2.0 Treatment System Receives China NMPA Approval

On March 16, 2020 Varian (NYSE: VAR) reported its Halcyon 2.0 treatment system has been approved by the China National Medical Products Administration (NMPA), which allows the company to market this cancer treatment system in China (Press release, Varian Medical Systems, MAR 16, 2020, View Source [SID1234555612]). This system incorporates new imaging technologies, such as kV Cone-beam CT (CBCT) and Iterative CBCT, to expand its capabilities and ability to deliver high quality cancer care globally. This approval further expands the global availability of Halcyon and access to high-quality, cost-effective cancer treatments.

As an advanced cancer treatment system with a human-centered and user-friendly design, Halcyon is engineered to revolutionize clinical workflow, accelerate installation timeframes, expedite commissioning, simplify training, and automate treatment. The system is well suited to treat a majority of cancer patients, offering advanced treatments for lung, prostate, breast, head & neck, and many other forms of cancer.

Halcyon 2.0 features kV CBCT and Iterative CBCT imaging for better soft tissue definition. The system is capable of kV CBCT images in approximately 15 seconds. This enhanced imaging capability helps ensure proper patient positioning while also accelerating the treatment workflow.

Halcyon is supported by Varian’s Eclipse treatment planning system. This software creates an optimized radiotherapy treatment plan based on a physician’s dose instructions and information about the size, shape, and location of the tumor to be treated with radiation. The Eclipse software incorporates unique features such as RapidPlan knowledge-based planning, multi-criteria optimization (MCO), and Graphics Processing Unit (GPUs) support, which are all designed to enable clinics to efficiently create the highest quality plans.

"We are very excited to receive this approval and make Halcyon 2.0 available to clinicians in China," said Chris Toth, president Varian Oncology Systems. "The original Halcyon system has already been well received in the country and has allowed clinicians to treat thousands of patients. These new imaging capabilities will play an important role in further accelerating the patient workflow and proving the opportunity to treat more patients."

Pulse Biosciences Report Fourth Quarter and Full Year 2019 Financial Results

On March 16, 2020 Pulse Biosciences, Inc. (Nasdaq: PLSE), a novel bioelectric medicine company, reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, Pulse Biosciences, MAR 16, 2020, View Source [SID1234555610]).

Recent Highlights

Nano-Pulse Stimulation (NPS) technology was highlighted in three clinical study presentations demonstrating its high clearance rates of benign skin lesions at the Annual Meeting of the American Society for Dermatologic Surgery.

Presented data from a study evaluating the clinical and histologic response of NPS technology in treating challenging cases of nodular Basal Cell Carcinoma at the International Master Course on Aging Skin World Congress.

Completed enrollment in the Company’s CellFX Warts Pivotal Study.

Obtained ISO-13485:2016 Quality System Management Certification to begin preparations for international commercialization of the CellFX System.

Appointed industry veteran Sandra Gardiner as Executive Vice President and Chief Financial Officer.

Announced Board approval to pursue a rights offering seeking to raise $30 million in net proceeds.

Following receipt of a Not Substantially Equivalent Letter from the U.S. Food and Drug Administration (FDA) on its previous 510(k) submission the Company remains engaged with the FDA to determine the regulatory path forward for the CellFX System. The Company recently requested a formal Q-submission meeting with FDA to discuss requirements for a new 510(k) submission and based on recent communication, expects the meeting to take place in May.

"I am proud of our continued work to develop NPS technology across multiple clinical applications. We’ve developed a novel and proprietary platform, the CellFX System, that implements a novel utilization-based business model to align incentives between physicians, patients and Pulse Biosciences. Our top priority remains FDA clearance for use of the CellFX System in aesthetic dermatology," said Darrin Uecker, President and CEO of Pulse Biosciences. "After recent developments and collaboration with FDA, we are refocused on generating and providing additional data that will support a new 510(k) submission for what we believe will be a general dermatologic indication. Our confidence in the technology continues to grow and we remain excited about the potential of our system."

Financial Update

Cash, cash equivalents and investments totaled $25.4 million as of December 31, 2019, compared to $34.5 million as of September 30, 2019. Cash used in the fourth quarter totaled $9.1 million. Cash use for the full year ended December 31, 2019 totaled $34.2 million.

Operating expenses for the three months ended December 31, 2019 were $13.9 million, compared to $9.1 million for the prior year period. Operating expenses for the full year ended December 31, 2019 were $48.0 million compared to $38.0 million for the full year ended December 31, 2018. The increase in operating expenses was driven by the expansion of operational infrastructure including marketing and sales functions as well as the expansion of the research and development teams and clinical trial costs.

Net loss for the fourth quarter ended December 31, 2019 was $13.8 million compared to $9.0 million for the fourth quarter ended December 31, 2018. Net loss for the full year ended December 31, 2019 was $47.0 million compared to $37.5 million for the full year ended December 31, 2018.

Oncternal Provides Business Update and Announces Fourth Quarter and Full Year 2019 Financial Results

On March 16, 2020 Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported fourth quarter and full year 2019 financial results (Press release, Oncternal Therapeutics, MAR 16, 2020, View Source [SID1234555609]).

"We are very pleased with recent clinical results from our pipeline of novel cancer treatments for patients with critical unmet medical need," said James Breitmeyer, M.D., Ph.D., President and CEO, Oncternal. "We have seen encouraging results in all four of our clinical indications, including mantle cell lymphoma ("MCL"), Ewing sarcoma, breast cancer and chronic lymphocytic leukemia ("CLL"). We look forward to steadily advancing these programs in 2020 and expect to have multiple data updates throughout the year. Preclinical work is also identifying additional clinical targets for our existing product candidates and for our ROR1 CAR-T program."

Recent Highlights

In March 2020, we announced an interim clinical data update for cirmtuzumab, a ROR1-targeted monoclonal antibody, in combination with ibrutinib in patients with relapsed/refractory MCL enrolled in our ongoing Phase 1/2 clinical trial, including a 50% complete response ("CR") rate and an 83% best objective response (CR or partial response) rate ("ORR"). This CR rate improved meaningfully from our previously reported CR rate of 33%.

In February 2020, we presented ROR1 CAR-T preclinical data at the ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium. ROR1 CAR-T cell therapy demonstrated expansion, persistence and anti-tumor activity in an animal model of human leukemia. This research is being conducted by our collaborators at the University of California San Diego (UC San Diego) under a grant from the California Institute of Regenerative Medicine ("CIRM").

In December 2019, we presented at the San Antonio Breast Cancer Symposium clinical data from an ongoing, investigator-sponsored Phase 1 clinical study of cirmtuzumab in combination with paclitaxel in patients with HER2-negative, metastatic or locally advanced unresectable breast cancer, including an ORR of 57%.

In December 2019, we opened for enrollment a Phase 1 expansion cohort of our ongoing 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/refractory Ewing sarcoma.

In December 2019, we presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting a clinical data update from our ongoing Phase 1/2 clinical study of cirmtuzumab in combination with ibrutinib in patients with MCL or CLL, including a best ORR of 85% and progression-free survival of 100% in patients with CLL.

In November 2019, we presented at the Connective Tissue Oncology Society Annual Meeting interim clinical data from our ongoing Phase 1 clinical trial evaluating TK216 in patients with relapsed/refractory Ewing sarcoma, including a deep and sustained clinical response reported for a patient who received a TK216 dose regimen that was subsequently selected as a recommended Phase 2 dose.

In October 2019, we announced the opening of a Phase 1b expansion cohort of our clinical trial of cirmtuzumab in combination with ibrutinib in patients with MCL.

Expected Upcoming Milestones

Cirmtuzumab program

Clinical data update for patients with MCL, including for over 15 patients in the ongoing Phase 1/2 study – in mid-2020

Clinical data update for patients with CLL, including 12-month follow-up for 34 patients in the ongoing Phase 1/2 study – in mid-2020

Clinical data update for patients with HER2-negative breast cancer in the ongoing Phase 1b study – in the second half of 2020

IND-enabling data in additional indications – in mid-2020

TK216 program

Clinical data for 7-12 patients with Ewing sarcoma enrolled in the Phase 1 expansion cohort – in the second half of 2020

IND-enabling data in additional ETS-driven tumors – in the second half of 2020

ROR1 CAR-T program

First-in-human dosing in China – in the fourth quarter of 2020

Fourth Quarter and Full Year 2019 Financial Results

Our grant revenue was $0.7 million for the fourth quarter ended December 31, 2019. Our grant revenue is derived from a subaward under a grant from CIRM to UC San Diego, which was awarded to advance our Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib for the treatment of patients with MCL or CLL. For the full year 2019, grant revenue was $2.4 million.

Our total operating expenses for the fourth quarter ended December 31, 2019 were $4.9 million. Research and development expenses for the quarter totaled $2.6 million, and general and administrative expenses for the quarter totaled $2.3 million. Net loss for the fourth quarter was $4.2 million, or a loss of $0.27 per share, basic and diluted. For the full year 2019, total operating expenses were $35.5 million, which included a one-time non-cash charge for acquired in-process research and development expenses of $18.1 million that was recorded in connection with the closing of our merger in June 2019. Net loss for the full year 2019 was $34.2 million, or a loss of $3.31 per share, basic and diluted.

As of December 31, 2019, we had $20.1 million in cash and cash equivalents. We believe these funds will be sufficient to fund our operations into the third quarter of 2020. As of December 31, 2019, we had approximately 15.4 million shares of common stock outstanding.

Management Webcast

As previously announced, Oncternal will host a webcast today, March 16, 2020, at 4:30 p.m. ET (1:30 p.m. PT). The live webcast will be available online and may be accessed from the "Investors" page of the company website at View Source A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.