On March 24, 2020 OncoQuest Inc. ("OncoQuest" or the "Company"), a privately held, cancer immunotherapy company reported the publication of two reports relating to the recently completed Phase 2 trial conducted in the US and Italy utilizing oregovomab, the Company’s lead investigational drug in frontline ovarian cancer (Press release, OncoQuest, MAR 24, 2020, View Source [SID1234555804]).

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The first report which appears in the journal Gynecologic Oncology is titled Front-line chemo-immunotherapy with carboplatin-paclitaxel using oregovomab indirect immunization in advanced ovarian cancer: A randomized phase II study, reported the clinical results in the completed 97-patient randomized controlled multi-site study in which 47 patients were randomized to receive chemoimmunotherapy with standard carboplatin and paclitaxel chemotherapy combined with oregovomab or standard chemotherapy (standard carboplatin and paclitaxel).

The study was conducted with a median of 42 months follow up and shows highly statistically significant outcomes for both progression-free and overall survival favoring the chemoimmunotherapy arm. The risk of progression and of death was reduced by more than 50% in the chemoimmunotherapy arm. Safety data shows that the addition of orgeovomab did not add incremental toxicity to chemotherapy.

The second report published in Cancer Immunology, Immunotherapy is titled Translational immune correlates of indirect antibody immunization in a randomized phase II study using scheduled combination therapy with carboplatin/paclitaxel plus oregovomab in ovarian cancer. The report examined translational laboratory outcomes from a subset of the Italian cohort of patients in the above described study from the laboratory of Professor Scambia at Catholic University Hospital in Rome. The report confirms that chemoimmunotherapy increased the presence of CA125-specific CD8+T lymphocytes measured in the peripheral blood compared to chemotherapy, which correlated with favorable clinical outcomes. Myeloid derived immune suppression was measured by MDSC4 (flow cytometry) and NMLR (neutrophil-monocyte to lymphocyte ratio) and it was found that lower levels of these parameters at baseline predicted more favorable outcomes in the patients receiving chemoimmunotherapy compared to chemotherapy. These findings offer promise of development of a readily accessible prognostic indicator for guiding the therapeutic strategy for newly diagnosed patients who would be candidates for chemoimmunotherapy with orgeovomab.

"The publication of our Phase II clinical results in peer reviewed journals validates the quality of the clinical data generated from our clinical study", said Dr. Madiyalakan, CEO of OncoQuest "We have discussed our Phase 2 results in an End of Phase 2 meeting with the FDA and based on those discussions are proceeding with our planned Phase 3 registration trial."

The Company is currently planning to launch a Phase 3 trial in Q2 2020. The planned Phase 3 study is expected to enroll over 600 patients with newly diagnosed, advanced ovarian cancer globally. The double-blind, placebo-controlled trial design is expected to incorporate analyses of the effect of the addition of oregovomab in both the adjuvant and neo-adjuvant settings. In both the adjuvant and neo-adjuvant arms, the primary endpoint will be to evaluate progression-free survival of patients treated with oregovomab plus a standard-of-care chemotherapy combination, carboplatin and paclitaxel, compared to the chemotherapy alone.

On April 17, 2020 Rafael Pharmaceuticals, Inc. (‘Rafael’ or the ‘Company’), a leader in the growing field of cancer metabolism-based therapeutics, reported that it has enrolled more than 75% of the 500 patients needed for its pivotal Phase 3 clinical trial for metastatic pancreatic cancer (AVENGER 500), which is evaluating the efficacy and safety of Rafael’s lead compound CPI-613️ (devimistat) in combination with modified FOLFIRINOX (mFFX) as first-line therapy (Press release, Rafael Pharmaceuticals, MAR 24, 2020, View Source [SID1234556415]).

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‘We are continuing to enroll patients ahead of schedule, as the patients in our trials suffer from life-threatening diseases and are in dire need of treatment; patient safety is our top priority,’ said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. ‘In order to support our clinical trial sites and enrolled patients during this unsettling and unprecedented time, we have established a COVID-19 task force to closely monitor our clinical trials and make changes as needed. We want to express our deepest gratitude to all of the principal investigators, nurses, hospital staff, patients and their families for their continued support and cooperation.’

‘To Save a Life is to Save a Universe’ – Rafael’s motto – represents its dedication to patients who suffer from rare cancers globally, as well as to its employees and the communities it serves. As such, the company has implemented measures to help ensure the continuity of its clinical trials and programs, while maintaining the safety of patients, clinicians and employees. The COVID-19 task force will closely monitor all clinical trials and collect data relevant to the patients’ study treatments and schedules.

Rafael is working closely with the clinical trial sites to preempt and promptly address any potential challenges and ensure that clinical supplies are available in sufficient quantities in order to support the continuity of care of all enrolled patients. Each clinical trial site has implemented their own safety measures based on guidelines from local authorities. If patients have questions about their clinical trial, they should reach out to the clinical trial coordinator at their medical center.

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 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’s side effects. Combination with devimistat represent a diverse range of opportunities to substantially improve patient’s 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 and Burkitt’s lymphoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.

On March 24, 2020 Sun BioPharma, Inc. (OTCQB: SNBP), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of pancreatic cancer, reported financial results for the year ended December 31, 2019 (Press release, Sun BioPharma, MAR 24, 2020, View Source [SID1234555789]). Sun BioPharma is developing SBP-101, a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI), a metabolic pathway of critical importance to cell function in multiple tumor types, including pancreatic cancer.

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"I am delighted with the progress we have made in advancing the development of SBP-101 for patients with metastatic pancreatic cancer. In the past 12 months, we have completed enrollment in the four dose escalation cohorts of our current trial, presented interim data ASCO (Free ASCO Whitepaper) GI Cancer Symposium, initiated two new clinical sites in the United States, and opened an expansion cohort to patient enrollment at what we believe to be the optimal dose and schedule," said Michael T. Cullen, MD, MBA, Chairman and CEO of Sun BioPharma. "We look forward to completing the design of a randomized phase 2 study."

Significant Progress Made in Advancing SBP-101 Clinical Development
In early 2020, enrollment was completed in the fourth cohort of the ongoing Phase 1a/1b study of SBP-101 in patients with metastatic pancreatic ductal adenocarcinoma (PDA). Based upon the study data generated to date, an expansion cohort in patients with PDA was initiated in February of 2020. This expansion phase will enroll up to 36 additional patients at the recommended dose and schedule. A total of six clinical sites are now participating in the SBP-101 clinical program.

Interim Phase 1 Results Presented in Poster Session at ASCO (Free ASCO Whitepaper) GI Cancers Symposium Interim Phase 1 clinical data for SBP-101 was presented in a poster session at the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Gastrointestinal Cancers Symposium on January 24, 2020. The adverse event profile of SBP-101 below the maximal tolerated dose was manageable, and SBP-101 was well tolerated in combination with gemcitabine and nab-paclitaxel. The addition of SBP-101 to the combination of gemcitabine plus nab-paclitaxel did not increase the frequency of moderate or severe hematologic adverse events, peripheral neuropathy, nausea or diarrhea when compared to historical control data for patients who were treated with gemcitabine plus nab-paclitaxel. The most common adverse events attributed to treatment with SBP-101 were fatigue, elevated hepatic enzymes, and injection site pain.

Available study results in 13 evaluable subjects enrolled in the two highest dose cohorts (n=16) demonstrated an overall response rate (ORR) of 62% by RECIST criteria. The disease control rate (DCR) was 85% by RECIST criteria. Eleven subjects in those cohorts (69%, n=16) saw a maximum decrease in CA 19-9 of more than 60%.

Financial Results for the Three Months and Full Year Ended December 31, 2019

General and administrative (G&A) expenses were flat at $468,000 in the fourth quarter of 2019, compared with $467,000 in the fourth quarter of 2018. G&A expenses for the full year decreased 6.4% to $2.0 million in 2019, down from $2.1 million in 2018. The decrease for the full year of 2019 is primarily due to fewer staff members versus the 2018 staff level.

Research and development (R&D) expenses increased 74.9% to $787,000 in the fourth quarter of 2019 up from $450,000 in the fourth quarter of 2018. R&D expenses for the full year of 2019 increased 31.7% to $2.3 million as compared with $1.8 million for 2018. The increase in the fourth quarter resulted from the cost to begin production of drug substance for future phase 2 clinical trials. The full-year increase in R&D expenses resulted from an increase in spending on the Company’s clinical trial during 2019 and the manufacturing of drug substance initiated during the fourth quarter of 2019.

Other income, net, was $196,000 in the current quarter compared to other expense, net, of $64,000 in the fourth quarter of 2018. Other expense, net, was flat at $2.3 million for both 2019 and 2018. The amounts reflected in the fourth quarter of each year are primarily a foreign currency translation adjustment on the intercompany balance with our Australian subsidiary. For both 2018 and 2019 the full year expense is primarily the amortization of debt discount, which is recorded as interest expense.

Net loss for the quarters ended December 31, 2019 and 2018 was $1.0 million and $0.9 million, or $0.15 and $0.18 per diluted share, respectively. The net loss for the full year 2019 was $6.2 million, or $1.09 per diluted share, compared to a net loss of $5.9 million, or $1.27 per diluted share, for 2018.

Balance Sheet and Cash Flow

Total cash resources were $2.4 million as of December 31, 2019, compared to $1.4 million as of December 31, 2018. Total current assets were $3.1 million and $1.8 million as of December 31, 2019, and December 31, 2018, respectively. These increases resulted primarily from the proceeds raised from the sale of convertible promissory notes in January 2019 of $0.8 million and the sale of equity securities in the second half of 2019 totaling $3.2 million, partially offset by the Company’s use of cash to fund operations during 2019.

Current liabilities increased to $1.8 million as of December 31, 2019, compared to $1.6 million as of December 31, 2018. The increase in current liabilities is reflective of an increase in vendor payables associated with increased clinical and manufacturing activity in 2019.

Net cash used in operating activities was $2.7 million for the year ended December 31, 2019, compared to $2.4 million for the year ended December 31, 2018. The net cash used in each of these periods primarily reflected the net loss for these periods and was partially offset by stockbased compensation expense and amortization of debt discount as well as by the effects of changes in operating assets and liabilities.

On March 24, 2020 IDEAYA Biosciences, Inc. (Nasdaq:IDYA), an oncology-focused precision medicine company committed to the discovery and development of targeted therapeutics for patient populations selected using reported that financial results for the fourth quarter ended December 31, 2019 (Press release, Ideaya Biosciences, MAR 24, 2020, View Source [SID1234555805]).

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"IDEAYA is building a leading synthetic lethality biopharmaceutical company, advancing a broad pipeline of synthetic lethality programs, including our MAT2A program for which we have selected a lead compound MAT2A inhibitor. We also continue to advance development of IDE196 in our Phase 1/2 tissue-agnostic basket trial in patients with solid tumors harboring GNAQ or GNA11 (GNAQ/11) mutations such as metastatic uveal melanoma (MUM), cutaneous melanoma and colorectal cancer, including evaluation of IDE196 in combination with binimetinib under a clinical trial collaboration and supply agreement with Pfizer," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

We continue to progress our MAT2A synthetic lethality program for tumors with MTAP deletion. We have selected a lead compound which we believe has favorably differentiated activity, physical properties and tolerability, and have scaled this lead compound for non-GLP toxicology studies in two species to support selection of a development candidate in the second quarter of 2020.

We also continue to progress our broad pipeline of synthetic lethality programs, including Pol theta for tumors with BRCA or other homologous recombination deficiency (HRD) mutations, Werner (WRN) for tumors with high microsatellite instability (MSI), and PARG for tumors with BRCA2 mutations, impaired base excision repair, or replication stress signature. We are applying our fully integrated research and translational capabilities to these programs. We have solved the crystal structures for each of these research programs, and we are conducting preclinical in vivo efficacy studies in three of our synthetic lethality programs.

Key highlights for IDEAYA’s research and development programs include:

Clinical Program IDE196

IDE196

Advanced IDEAYA’s Phase 1/2 tissue-type agnostic basket trial, initiated in June 2019, to evaluate IDE196 in solid tumors harboring activating GNAQ/11 mutations, entitled "A phase 1/2 study of IDE196 in patients with solid tumors harboring GNAQ/11 mutations or PRKC fusions" (ClinicalTrials.gov Identifier: NCT03947385). As of March 15, 2020:
Enrolled 53 patients in IDE196 monotherapy arm of Phase 1/2 clinical trial
Ongoing evaluation of IDE196 monotherapy arm in MUM, with aggregate enrollment of 49 patients in the Phase 1 dose escalation and tablet formulation studies
Initiated the Phase 2 expansion for IDE196 as a monotherapy in solid tumors other than MUM having GNAQ or GNA11 hotspot mutations, with aggregate Phase 1/2 enrollment of 4 cutaneous melanoma patients
Selected 400mg BID (with one week 200 mg BID run-in) as Phase 2 monotherapy dose; observed higher average steady state exposure of free IDE196 (AUCfree, increase of approximately 44%) and higher trough concentration of IDE196 (Cmin, increase of approximately 40%) at 400 mg BID relative to 300 mg BID dose
Evaluating tablet formulation of IDE196 in MUM patients in a Phase 1 sub-study, with the pharmacokinetic profile of the tablet formulation comparable to the powder-in-capsule form of IDE196
Completed in-life portion of the ongoing 13-week GLP-compliant toxicology studies in two species, initiated in November 2019
Interim data from the monotherapy arm of the Phase 1/2 basket trial targeted for second half 2020
Entered into a clinical trial collaboration and supply agreement with Pfizer; targeting to initiate combination arm of Phase 1/2 clinical trial in mid-2020 to evaluate safety and efficacy of IDE196 in combination with binimetinib, a MEK inhibitor, in patients having tumors with activating GNAQ or GNA11 hotspot mutations, including in metastatic uveal melanoma and other solid tumors
Design and initiation of potential registration-enabling study in MUM will be evaluated based on results of ongoing IDE196 monotherapy arm and planned IDE196 / binimetinib combination arm of the Phase 1/2 clinical trial
Preclinical Synthetic Lethality Programs

MAT2A

Observed single agent in vivo efficacy of our MAT2A inhibitors, including tumor growth inhibition or tumor regression in multiple MTAP -/- endogenous models
Selected a lead compound which we believe has favorably differentiated in vivo activity, physical properties and tolerability profile relative to published Agios compounds
Scaled the MAT2A lead compound for non-GLP toxicology studies in two species to support selection of a development candidate in the second quarter of 2020
Expect to file an IND for MAT2A inhibitor development candidate in fourth quarter of 2020
Pol Theta

Observed monotherapy activity, showing cell viability activity and in vivo tumor growth inhibition in a DLD1 BRCA2 -/- engineered model
Observed combination activity with a PARPi, Olaparib, as well as synergistic cell viability activity and synergistic in vivo tumor growth inhibition in the DLD1 BRCA2 -/- engineered model, with a weak drug-drug interaction signal
Targeting designation of Pol-theta inhibitor development candidate in second half of 2020
Werner (WRN)

Observed dose-dependent cellular viability effect in multiple endogenous MSI high cell lines, with an expected lack of activity in microsatellite stable, or MSS, cell lines
Observed dose-dependent cellular pharmacodynamic (PD) response in multiple endogenous MSI high cell lines
Solved crystal structure of WRN helicase domain
Targeting to demonstrate in vivo proof of concept in relevant animal models in 2020
PARG

Observed dose-dependent cellular pharmacodynamic response and cellular viability effect in a HCC1806 XRCC1 -/- cell line
Expanded research collaboration with Cancer Research UK (CRUK) and the University of Manchester, UK, to evaluate IDEAYA’s potent selective PARG inhibitors in vitro and in vivo in multiple ovarian cancer cell lines and xenograft models, respectively, and to evaluate replication stress signature as a potential patient selection biomarker
"We continue to advance our programs, expand our capabilities and enhance our team. We believe that the IDE196 clinical program and our preclinical pipeline of synthetic lethality programs are maturing, moving forward toward our goal of improving lives through transformative precision medicines," said Yujiro S. Hata, Chief Executive Officer and President at IDEAYA Biosciences.

Corporate Updates

IDEAYA anticipates that existing cash, cash equivalents, and short-term and long-term marketable securities of $100.5 million (as of December 31, 2019) will be sufficient to fund planned operations into the end of 2021 to early 2022.

Our updated corporate presentation is available on our website, in the Presentations section of our Investor Relations page. See: View Source

Financial Results

As of December 31, 2019, IDEAYA had cash, cash equivalents, and short-term and long-term marketable securities totaling $100.5 million. This compared to cash, cash equivalents and short-term marketable securities of $90.0 million at December 31, 2018. The increase was primarily due to the receipt of $50.2 million in net proceeds from IDEAYA’s initial public offering, which was completed in May 2019, offset by cash used in operations.

Research and development expenses for the three months ended December 31, 2019 totaled $8.5 million compared to $7.6 million for the same period in 2018. The increase was primarily due to costs in connection with IDEAYA’s Phase 1/2 clinical trial to evaluate IDE196 in solid tumors, and costs for personnel and consulting in support of our research programs during the three months ended December 31, 2019.

General and administrative expenses for the three months ended December 31, 2019 totaled $2.8 million compared to $1.6 million for the same period in 2018. The increase was primarily due to an increase in costs for personnel and directors’ and officers’ liability insurance premiums in connection with becoming a publicly traded company.

Research and development expenses for the year ended December 31, 2019 totaled $34.3 million compared to $31.7 million for 2018. The increase was primarily due to an increase in costs in connection with IDEAYA’s Phase 1/2 clinical trial to evaluate IDE196 in solid tumors, and costs for personnel and consulting in support of our research programs during the year ended December 31, 2019, offset by a decrease in license fees for our IDE196 license agreement with Novartis during the year ended December 31, 2018.

General and administrative expenses for the year ended December 31, 2019 totaled $10.0 million compared to $4.7 million for 2018. The increase was primarily due to an increase in costs for personnel, directors’ and officers’ liability insurance premiums, and professional fees in connection with becoming a publicly traded company.

The net loss for the three months ended December 31, 2019 was $10.8 million compared to $8.6 million for the same period in 2018. Total stock compensation expense for the three months ended December 31, 2019 was $0.7 million compared to $0.3 million for the same period in 2018.

The net loss for the year ended December 31, 2019 was $42.0 million compared to $34.3 million for the same period in 2018. Total stock compensation expense for the year ended December 31, 2019 was $2.2 million compared to $1.0 million for the same period in 2018.

On April 17, 2020 Rafael Pharmaceuticals, Inc. (‘Rafael’ or the ‘Company’), a leader in the growing field of cancer metabolism-based therapeutics, reported that it has enrolled more than 75% of the 500 patients needed for its pivotal Phase 3 clinical trial for metastatic pancreatic cancer (AVENGER 500), which is evaluating the efficacy and safety of Rafael’s lead compound CPI-613️ (devimistat) in combination with modified FOLFIRINOX (mFFX) as first-line therapy (Press release, Rafael Pharmaceuticals, MAR 24, 2020, View Source [SID1234556415]).

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Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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‘We are continuing to enroll patients ahead of schedule, as the patients in our trials suffer from life-threatening diseases and are in dire need of treatment; patient safety is our top priority,’ said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. ‘In order to support our clinical trial sites and enrolled patients during this unsettling and unprecedented time, we have established a COVID-19 task force to closely monitor our clinical trials and make changes as needed. We want to express our deepest gratitude to all of the principal investigators, nurses, hospital staff, patients and their families for their continued support and cooperation.’

‘To Save a Life is to Save a Universe’ – Rafael’s motto – represents its dedication to patients who suffer from rare cancers globally, as well as to its employees and the communities it serves. As such, the company has implemented measures to help ensure the continuity of its clinical trials and programs, while maintaining the safety of patients, clinicians and employees. The COVID-19 task force will closely monitor all clinical trials and collect data relevant to the patients’ study treatments and schedules.

Rafael is working closely with the clinical trial sites to preempt and promptly address any potential challenges and ensure that clinical supplies are available in sufficient quantities in order to support the continuity of care of all enrolled patients. Each clinical trial site has implemented their own safety measures based on guidelines from local authorities. If patients have questions about their clinical trial, they should reach out to the clinical trial coordinator at their medical center.

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 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’s side effects. Combination with devimistat represent a diverse range of opportunities to substantially improve patient’s 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 and Burkitt’s lymphoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.