Aravive Reports Third Quarter 2021 Financial Results and Provides Corporate Updates

On October 28, 2021 Aravive, Inc. (Nasdaq: ARAV), a clinical-stage oncology company developing transformative, targeted therapeutics to treat life-threatening cancers, reported recent corporate updates and financial results for the third quarter ended September 30, 2021 (Press release, Aravive, OCT 28, 2021, View Source [SID1234594066]).

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"During the third quarter, we expanded our clinical oncology pipeline with the initiation of a Phase 1b trial of batiraxcept (AVB-500) for the treatment of pancreatic adenocarcinoma," said Gail McIntyre, Ph.D., DABT, Chief Executive Officer of Aravive. "Aravive is now advancing three clinical trials for life threatening cancers with unmet medical needs. We look forward to presenting new preliminary data from our Phase 1b trial of batiraxcept in clear cell renal cell carcinoma at the upcoming Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting in November. We remain optimistic about the broad potential of batiraxcept to improve the lives of people living with cancer."

Recent Corporate Highlights

Batiraxcept (AVB-500) in Platinum Resistant Ovarian Cancer (PROC): As a result of the evolving pandemic and other factors, the pace of enrollment in the registrational Phase 3 clinical trial of batiraxcept in PROC has been slower than originally anticipated. Aravive now expects to conduct the interim analysis in mid-2022. The interim analysis is being conducted to determine whether randomization will continue with all patients, regardless of prior bevacizumab treatment, or only with patients medically ineligible to receive bevacizumab or who chose not to receive bevacizumab. The final analysis of the primary endpoint preserves the opportunity to evaluate the efficacy in patients who received bevacizumab prior to study entry, as well as those patients who never received bevacizumab. This provides an additional opportunity to be successful in both patient populations, regardless of the results of the interim analysis. The global, randomized, double-blind, placebo-controlled adaptive trial is designed to evaluate efficacy and safety of batiraxcept at a dose of 15 mg/kg in combination with paclitaxel versus paclitaxel alone.

In October, the European Commission granted orphan drug designation to batiraxcept for the treatment of PROC. Orphan drug designation is granted for medicines being developed for the diagnosis, prevention or treatment of life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union.
Batiraxcept in Clear Cell Renal Cell Carcinoma (ccRCC): Aravive plans to present new preliminary data from the Phase 1b portion of its Phase 1b/2 clinical trial of batiraxcept in ccRCC at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting on November 13, 2021. The Company previously announced positive initial safety, pharmacokinetic and pharmacodynamic results from three patients dosed with 15 mg/kg of batiraxcept in combination with cabozantinib in the Phase 1b portion of the trial. Aravive expects to complete enrollment in the Phase 1b portion of the trial and initiate the Phase 2 portion of the trial in the fourth quarter of 2021.

Batiraxcept in Pancreatic Adenocarcinoma: In August 2021, Aravive dosed the first patient in the Phase 1b portion of its Phase 1b/2 trial of batiraxcept in combination with gemcitabine and nab-paclitaxel as a first-line treatment in patients with advanced or metastatic pancreatic adenocarcinoma. The Phase 1b portion of the clinical trial will evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of batiraxcept in combination with gemcitabine and nab-paclitaxel. The Company expects to complete enrollment in the Phase 1b portion of the trial in the first half of 2022 and initiate the Phase 2 portion of the trial in the second half of 2022.

Achieved Second Development Milestone with 3D Medicines: In July 2021, Aravive achieved a $3.0 million development milestone based on the Center for Drug Evaluation of the China National Medical Products Administration approval of the Investigational New Drug application submitted by 3D Medicines Inc. to participate in Aravive’s registrational Phase 3 clinical trial of batiraxcept in PROC. The Company received the $3.0 million development milestone payment in the third quarter of 2021. Under the terms of the collaboration and license agreement with 3D Medicines, Aravive is eligible to receive up to an aggregate of $207 million in development and commercial milestone payments and royalties.
Third Quarter 2021 Financial Results
Revenue for the three and nine months ended September 30, 2021 were $2.4 million and $6.5 million, respectively, compared to $0 for both periods in 2020. Revenue for the three and nine months ended September 30, 2021 was derived solely from the Company’s collaboration and license agreement with 3D Medicines and represents a portion of initial signing and milestone payments received from 3D Medicines that is recognized at the time of the receipt and a portion of the payments that is deferred and recognized over the PROC trial period.

Total operating expenses for the three and nine months ended September 30, 2021 were $14.0 million and $33.4 million, respectively, compared to $10.7 million and $26.7 million for the same periods in 2020.

Total operating expenses for the three and nine months ended September 30, 2021 included non-cash stock-based compensation expense of $0.6 million and $1.7 million, respectively, compared to $0.4 million and $1.6 million for the same periods in 2020. In addition, during the nine months ended September 30, 2020, there were non-recurring non-cash charges for impairment of the Company’s right-of-use asset and leasehold improvements of $5.8 million.

For the three and nine months ended September 30, 2021, Aravive reported a net loss of $11.1 million and $26.2 million, or $0.53 per share and $1.33 per share, respectively, compared to a net loss of $10.7 million and $26.5 million, or $0.66 per share and $1.69 per share, for the same periods in 2020.

Cash Position
As of September 30, 2021, cash and cash equivalents were $67.5 million, compared to $60.5 million as of December 31, 2020. Based upon its current operating plan and balance sheet as of September 30, 2021, Aravive expects to have sufficient cash to be able to fund the base operating plan into the second half of 2022.

About Batiraxcept (Formerly AVB-500)
Batiraxcept is a therapeutic recombinant fusion protein that has been shown to neutralize GAS6 activity by binding to GAS6 with very high affinity in preclinical models. In doing so, batiraxcept selectively inhibits the GAS6-AXL signaling pathway, which is upregulated in multiple cancer types including ovarian, renal and pancreatic cancer. In preclinical studies, GAS6-AXL inhibition has shown anti-tumor activity in combination with a variety of anticancer therapies, including radiation therapy, immuno-oncology agents, and chemotherapeutic drugs that affect DNA replication and repair. Increased expression of AXL and GAS6 in tumors has been correlated with poor prognosis and decreased survival and has been implicated in therapeutic resistance to conventional chemotherapeutics and targeted therapies. Batiraxcept is currently being evaluated in multiple clinical trials and has been granted Fast Track designation by the U.S. Food and Drug Administration and orphan drug designation by the European Commission in platinum resistant recurrent ovarian cancer. Analysis of all safety data to date showed that batiraxcept has been generally well tolerated with no dose-limiting toxicities or unexpected safety signals.

Rafael Pharmaceuticals Provides Update on Pivotal Phase 3 Clinical Trial in Patients with Metastatic Pancreatic Cancer and Interim Analysis of Pivotal Phase 3 Clinical Trial in Patients with Relapsed or Refractory Acute Myeloid Leukemia

On October 28, 2021 Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a company focused on the growing field of cancer metabolism-based therapeutics, reported that the AVENGER 500 Phase 3 clinical trial evaluating the efficacy and safety of CPI-613 (devimistat) in combination with modified FOLFIRINOX (mFFX) as a first-line therapy in patients with metastatic adenocarcinoma of the pancreas did not meet its primary endpoint of overall survival (Press release, Rafael Pharmaceuticals, OCT 28, 2021, View Source [SID1234592091]).

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In this multi-national Phase 3 randomized clinical trial, 528 patients with metastatic adenocarcinoma of the pancreas who had no prior therapy were randomized to receive either devimistat in combination with modified FOLFIRINOX (mFFX) or FOLFIRINOX, a current standard of care chemotherapy regimen. Devimistat given with mFFX did not significantly improve overall survival (HR=0.95, p=0.66). The median overall survival in the devimistat and mFFX arm was 11.1 months, compared to 11.7 months in the FOLFIRINOX arm.

"These cancers are incredibly difficult to treat, with few to no effective treatments available, but Rafael took the risk because we will always fight for our patients," said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. "While we are disappointed by the outcomes of these well-designed and well-executed studies, we remain committed to furthering our research and development in cancer metabolism for the treatment of hard-to-treat cancers, as our other studies continue. I personally want to express my heartfelt appreciation to the patients, their loved ones, the researchers and principal investigators for their trust and support. I am also incredibly grateful to my team, who works tirelessly for the patients we treat."

"Pancreatic cancer carries a high mortality rate and is extremely difficult to treat, but promising earlier clinical data encouraged us to move into this advanced phase trial with devimistat. While these are not the results we all hoped for, we will not give up, and we are hopeful that devimistat with its novel mechanism of action will demonstrate efficacy in other studies," said Philip Philip, M.D., Ph.D., F.R.C.P, principal investigator and medical oncologist at the Karmanos Cancer Institute at Wayne State University. "We are working diligently to further analyze the data and determine the best plan of action to further assess the drug’s capabilities in the clinic."

Devimistat is also being evaluated in a Phase 3 study, ARMADA 2000, in patients with relapsed or refractory acute myeloid leukemia (AML). Following a prespecified interim analysis, the independent data monitoring committee has recommended that the trial be stopped due to lack of efficacy.

"Earlier clinical trial data demonstrated positive results, despite the challenges we face in treating AML," said Jorge Cortes, M.D., director of the Georgia Cancer Center at Augusta University. "We were all hoping for positive results, and while this trial did not meet our expectations, we will continue to test devimistat in other studies."

The company will work with investigators to complete a full evaluation of the data on both trials.

About CPI-613 (Devimistat)
CPI-613 (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases cellular stress and the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patients’ benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500) and acute myeloid leukemia (ARMADA 2000) and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma, soft tissue sarcoma, Burkitt’s lymphoma and biliary tract cancer. The European Medicines Agency (EMA) has granted orphan drug designation to devimistat for pancreatic cancer, acute myeloid leukemia and Burkitt’s lymphoma.

Intellia Therapeutics to Hold Conference Call to Discuss Third Quarter 2021 Earnings and Company Updates

On October 28, 2021 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, reported that it will present its third quarter 2021 financial results and operational highlights in a conference call on November 4, 2021 at 8 a.m. ET (Press release, Intellia Therapeutics, OCT 28, 2021, View Source [SID1234592108]).

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To join the call:

U.S. callers should dial 1-833-316-0545 and international callers should dial 1-412-317-5726, approximately five minutes before the call. All participants should ask to be connected to the Intellia Therapeutics conference call.
Please visit this link for a simultaneous live webcast of the call.
A replay of the call will be available through the Events and Presentations page of the Investors & Media section on Intellia’s website at www.intelliatx.com, beginning on November 4, 2021 at 12 p.m. ET.

Samsung Receives FDA Clearance for AI Algorithms that Detect Lung Nodules in Chest X-rays

On October 28, 2021 NeuroLogica Corp. reported it has received Food and Drug Administration (FDA) 510(k) clearance for its Auto Lung Nodule Detection (ALND) tool (Press release, Samsung BioLogics, OCT 28, 2021, View Source [SID1234592141]). The offering provides an on-device, computer-assisted detection (CADe) solution for detecting pulmonary nodules from 10 to 30mm in size through an artificial intelligence (AI) algorithm.*

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It is designed to aid the physician in reviewing PA chest radiographs of adults and is part of S-Station, an operation software installed on Samsung Digital X-ray Imaging systems.

"This FDA clearance is a huge milestone for Samsung and is the result of our tireless work to design diagnostic solutions that empower providers to deliver patients the absolute best care possible," said David Legg, Vice President of Digital Radiography and Ultrasound at Samsung NeuroLogica. "The fact that it delivers clinically reliable results means clinicians can present it to patients with the utmost confidence, and for that we’re very proud."

Benefits of ALND include:

Aiding the reader’s diagnosis by indicating the location of suspected lung nodules on chest X-ray images (posteroanterior chest radiographs). The deep-learning technology has been clinically verified in multiple university hospitals and has been approved with a sensitivity of 80% or more. Investigators at these hospitals – Freiburg University Hospital, Freiburg, Germany; Massachusetts General Hospital, Boston, Massachusetts; Samsung Medical Center, Seoul, South Korea; and Severance Hospital, Seoul, South Korea – retrospectively identified 600 chest radiographs with lung cancer and 200 normal chest radiographs.
Extensive external clinical validation that has been recognized by the radiological society for having been performed with ‘unprecedented’ diversity of images acquired under different conditions and demographics, while only producing a small number of false positives per image (0.15).
Providing an option (Autorun) to automatically perform nodule detection immediately after chest X-ray imaging, and providing PACS transmission options to suit the hospital environment, both which simplify a user’s workflow.
Clinical evaluation results have demonstrated that all readers’ nodule detection performances using ALND have increased with statistical significance.

As part of our commitment to advancing diagnostic radiology using AI, Samsung is collaborating with Vuno, a leading developer of AI solutions in healthcare. As part of our collaboration, we will expand the uses of the chest CADe solution and improve the diagnostic accuracy and workflow.

For more information on Samsung’s healthcare business and products, please visit www.SamsungHealthcare.com.

* ALND cannot be used on patients who have lung lesions other than abnormal nodules.

Heron Therapeutics to Report Third Quarter 2021 Financial Results on Wednesday, November 3, 2021

On October 28, 2021 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 the company will host a conference call and live webcast on Wednesday, November 3, 2021 at 4:15 p.m. ET to report third quarter 2021 financial results and discuss recent business highlights (Press release, Heron Therapeutics, OCT 28, 2021, View Source [SID1234592157]).

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The conference call can be accessed by dialing 877-311-5906 for domestic callers and 281-241-6150 for international callers. Please provide the operator with the passcode 7242566 to join the conference call. The conference call will also be available via webcast under the Investor Relations section of Heron’s website at www.herontx.com. An archive of the teleconference and webcast will also be made available on Heron’s website for 60 days following the call.