On May 14, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported that new data demonstrating the ability of the Percepta Genomic Atlas to detect alterations that may inform lung cancer treatment decisions for patients from the same small biopsy that was used for diagnosis (Press release, Veracyte, MAY 14, 2021, View Source [SID1234580028]). Study findings presented today at the American Thoracic Society (ATS) 2021 International Conference show that the test accurately detects known lung-cancer gene variants using small biopsy samples, which may help guide treatment with targeted therapies.

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The in-development Percepta Genomic Atlas will provide comprehensive genomic profiling information on cancerous lung nodules, masses or lymph nodes, utilizing small samples from the same biopsy procedure used for diagnosis (i.e., transbronchial needle aspirate biopsies or TBNA). The test uses targeted DNA and whole-transcriptome RNA sequencing to detect alterations in more than 50 genes known to be present in lung cancer. Veracyte plans to launch the test in the second half of 2021 as part of the company’s comprehensive portfolio of genomic tests in lung cancer.

For the analyses presented at the ATS Conference today, researchers evaluated the feasibility of detecting National Comprehensive Cancer Network (NCCN)-recommended gene alterations with the Percepta Genomic Atlas, using samples from 25 patients obtained during initial bronchoscopy procedures. Results show that the in-development test accurately detected the gene variants in these samples with over 95 percent concordance to a reference next-generation sequencing assay.

"Physicians today are often challenged to obtain genomic profiling information on their lung cancer patients’ tumors, due to inadequate tissue, logistical delays and other factors. As a result, patients often do not receive optimal treatment," said Giulia C. Kennedy, Ph.D., Veracyte’s chief scientific officer and chief medical officer. "These findings give us confidence that the Percepta Genomic Atlas will be able to accurately detect gene alterations that may allow patients to be treated with targeted therapies that are available now and that will be available in the future. Importantly, we believe it will be able to provide this critical information near the time of diagnosis, enabling earlier and more appropriate treatment."

The Percepta Genomic Atlas ATS 2021 abstract can be viewed here. The poster presentation is available on demand to ATS conference registrants through July 2.

The Percepta Genomic Atlas will be a key part of Veracyte’s lung cancer portfolio, which aims to transform care at every step of the patient journey. Collectively, the company’s tests are leveraging cutting-edge genomic science and technology to provide answers and insights that enable physicians and patients to make better, faster and more confident care decisions.

On May 14, 2021 PMV Pharmaceuticals, Inc. (Nasdaq: PMVP), a precision oncology company pioneering the discovery and development of small molecule, tumor-agnostic therapies targeting p53 mutants, reported financial results for the quarter ended March 31, 2021 and provided corporate highlights (Press release, PMV Pharma, MAY 14, 2021, View Source [SID1234579983]).

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"PMV had a successful first quarter. We continued to advance our clinical and discovery programs, and our presentation at AACR (Free AACR Whitepaper) provided compelling evidence that PC14586 selectively reactivates the p53 Y220C mutant protein, both in vitro and in vivo," said David Mack, Ph.D., President and Chief Executive Officer. "Enrollment in our Phase 1/2 trial of PC14586, our first in class p53 Y220C reactivator, is progressing as planned. We are also thrilled to have welcomed Chuck Baum to our Board and Gigi Lozano to our SAB and look forward to leveraging their deep expertise in precision therapeutics and genetic drivers of cancer."

Corporate Highlights:

In March 2021, PMV Pharma presented late breaking preclinical data on PC14586, the Company’s first-in-class, tumor-agnostic, investigational small molecule p53 Y220C reactivator at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021. The Oral presentation titled, "PC14586: The First Orally Bioavailable Small Molecule Reactivator of Y220C Mutant p53 in Clinical Development" was given by Melissa L. Dumble, Ph.D., Vice President Preclinical Development and Translational Science of PMV.
PMV Pharma continued to enroll patients in the Phase 1 portion of a Phase 1/2 clinical trial of PC14586 (NCT04585750). PC14586 is being developed for the treatment of patients with locally advanced or metastatic solid tumors that have a p53 Y220C mutation.
Appointment of Charles Baum, M.D., Ph.D., to the Board of Directors. Dr. Baum is the President and Chief Executive Officer and a Board Member of Mirati Therapeutics Inc.
Expanded Scientific Advisory Board with the appointment of p53 pioneer Dr. Guillermina (Gigi) Lozano, Professor and Chair, Department of Genetics, MD Anderson Cancer Center.
PMV Pharma is the process of relocating to a state-of-the-art laboratory and office building located in Princeton, NJ.
First Quarter 2021 Financial Results

PMV Pharma ended the fourth quarter with $348.4 million in cash, cash equivalents, and marketable securities, compared to $361.4 million as of December 31, 2020. Net cash used in operations was $12.9 million for the three months ended March 31, 2021 compared to $8.1 million for the three months ended March 31, 2020.
Net loss for the quarter ended March 31, 2021 was $11.6 million compared to $7.3 million for the quarter ended March 31, 2020.
Research and development (R&D) expenses were $7.5 million for the three months ended March 31, 2021 compared to $6.0 million for the three months ended March 31, 2020. The increase in R&D expenses was primarily related to increased headcount and clinical expenses for advancing development of PC14586, the Company’s lead drug candidate.
General and administrative (G&A) expenses were $4.2 million for the three months ended March 31, 2021, compared to $1.7 million for the three months ended March 31, 2020. The increase in G&A expenses was primarily due to expanding the infrastructure necessary for operating as a public company.
About p53

p53 plays a pivotal role in preventing abnormal cells from becoming a tumor by inducing programmed cell death. Mutant p53 takes on oncogenic properties that endow cancer cells with a growth advantage and resistance to anti-cancer therapy. The p53 Y220C mutation is associated with many cancers, including but not limited to breast, non-small cell lung cancer, colorectal, pancreatic, and ovarian cancers.

About PC14586

PC14586 is a first-in-class, small molecule, p53 reactivator designed to selectively bind to the crevice present in the p53 Y220C mutant protein, hence, restoring the wild-type, or normal, p53 protein structure and tumor suppressing function. PC14586 is being developed for the treatment of patients with locally advanced or metastatic solid tumors that have the p53 Y220C mutation and has been granted Fast Track designation by the U.S. FDA.

On May 14, 2021 Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, reported financial results for the first quarter ended March 31, 2021 (Press release, Forma Therapeutics, MAY 14, 2021, View Source [SID1234580013]). The company also highlighted recent progress and upcoming milestones for its pipeline programs.

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"During the first quarter, we successfully completed the multiple ascending dose portion of our Phase 1 trial in sickle cell disease, and despite challenges from the COVID-19 pandemic also began enrolling patients in the Phase 2/3 trial of FT-4202, called The Hibiscus Study, as well as in the Phase 1 trial of FT-7051 for metastatic castration resistant prostate cancer," said Frank Lee, President and Chief Executive Officer of Forma. "We look forward to sharing additional pipeline results over the course of 2021 in our mission to transform the lives of people living with rare hematologic diseases and cancers."

Key Business and Clinical Highlights

PKR Program in Sickle Cell Disease (SCD):

MAD cohorts completed with approximately 71% of participants achieving hemoglobin increase ≥ 1 g/dL from baseline, and improvement across markers of RBC health. Doubling the dose of FT-4202 to 600 mg daily for 14 days compared to the previous 300 mg cohort was well-tolerated with no dose-limiting toxicities or treatment-related adverse events observed. Improvements in hematologic (hemoglobin and reticulocytes) and hemolytic (bilirubin and lactate dehydrogenase) parameters were comparable to that observed with the 300 mg dose, with best response typically observed at the end of the 14-day treatment period. In the combined cohorts, 10 of 14 (71%) patients on FT-4202 achieved a hemoglobin increase ≥ 1 g/dL from baseline to Day 14. Improvement in RBC health was evidenced by increased sickle RBC survival and reduced intravascular hemolysis in patients with SCD based on a reduction in reticulocytes, bilirubin and LDH levels.
Patient enrollment began in Phase 2/3 registrational trial, the Hibiscus Study. The Phase 2/3 Hibiscus Study is currently enrolling people living with SCD. This adaptive, randomized, placebo-controlled, double-blind, multi-center trial is expected to enroll approximately 344 adults and adolescents with SCD. FT-4202 doses of 200mg and 400mg administered once-daily are being evaluated in the Phase 2 portion of the trial. Primary endpoints in the Phase 3 portion of the trial are hemoglobin response rate at week 24 (increase of > 1 g/dL from baseline), intended to support accelerated approval, and annualized vaso-occlusive crisis rate during the 52-week blinded treatment period, which if positive is expected to support full approval.
CPB/p300 Program in Prostate Cancer:

FT-7051 Phase 1 clinical trial initiated for the treatment of mCRPC. In January 2021, Forma announced that the first patient was dosed in the ongoing Phase 1 clinical trial evaluating FT-7051 for the treatment of mCRPC. The trial is a multicenter, open-label evaluation of the safety and tolerability, preliminary anti-tumor activity (prostate specific antigen (PSA) and radiographic responses), and pharmacokinetics/pharmacodynamics (PK/PD) of FT-7051 in men with mCRPC who have progressed despite prior therapy with at least one anti-androgen therapy. The trial will include genetic mutation analysis to identify the basis of resistance to standard-of-care and will also evaluate expression of the AR-v7 splice variant, for which there are no approved therapies. The trial utilizes an adaptive trial design, intended to accelerate the escalation to potentially therapeutic doses and yield important safety information, as well as to identify biomarkers of clinical benefit such as PSA response.
IDH1 Program in AML and Glioma:

Olutasidenib NDA preparation for R/R AML. With the conclusion of the Phase 2 R/R AML trial, Forma has begun preparing an NDA for submission to the U.S. Food and Drug Administration (FDA).
Upcoming Milestones

Presentation of updated Phase 1 FT-4202 results in SCD. A poster presentation on FT-4202 in SCD is scheduled for the EHA (Free EHA Whitepaper) Virtual Congress taking place June 9-17, 2021. The presentation will include combined unblinded data from the two-week MAD cohorts as well as initial OLE results to date. In addition, full results from the MAD dose cohorts and the OLE are expected to be presented at a scientific congress in late 2021.
Initial Phase 1 clinical results from FT-7051 in mCRPC anticipated later this year. This adaptive trial is assessing multiple doses of FT-7051 with dose escalation dependent upon safety and tolerability. Initial results anticipated in the fourth quarter of 2021 may include safety/tolerability, PK/PD results and preliminary biomarker data.
Olutasidenib results presentation in R/R AML. Phase 2 registrational results of olutasidenib in R/R AML will be presented at the 2021 ASCO (Free ASCO Whitepaper) Annual Meeting taking place from June 4-8, 2021, and the EHA (Free EHA Whitepaper) Virtual Congress taking place June 9-17, 2021.
Possibility of COVID-19 impact remains. The COVID-19 pandemic remains a factor in the successful completion of these milestones. Many clinical trials across the biopharma industry, including ours, have been impacted by the COVID-19 pandemic, with clinical trial sites implementing new policies in response to COVID-19, resulting in potential delays to enrollment of clinical trials or changes in the ability to access sites participating in clinical trials.
Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $603.7 million as of March 31, 2021, as compared to $645.6 million as of December 31, 2020. Current cash runway is projected through the third quarter of 2024.
Research and Development (R&D) Expenses: R&D expenses were $26.3 million for the quarter ended March 31, 2021, compared to $23.2 million for the quarter ended March 31, 2020. The increase was primarily attributable to increases in FT-4202 development expenses, partially offset by reduced spending on olutasidenib development.
General and Administrative (G&A) Expenses: G&A expenses were $9.9 million for the quarter ended March 31, 2021, compared to $8.9 million for the quarter ended March 31, 2020. The increase in general and administrative expense was primarily attributable to stock compensation expense and insurance, partially offset by a reduction in professional fees.
Net Income/Loss: Net loss was $36.0 million for the quarter ended March 31, 2021, compared to net income of $11.2 million for the quarter ended March 31, 2020.
Forma will conduct a conference call and webcast May 14th at 8 a.m. Eastern Daylight Time (EDT) to discuss first quarter 2021 results and business update. The call can be accessed by dialing (833) 301-1146 in the U.S., and (914) 987-7386 internationally, with conference ID 8597396.

On May 14, 2021 Prescient Therapeutics (PTX) reported a new research program with the Peter MacCallum Cancer Centre to advance its next-generation CAR-T programs (Press release, Prescient Therapeutics, MAY 14, 2021, View Source;utm_medium=rss&utm_campaign=prescient-therapeutics-asxptx-signs-new-research-agreement-with-peter-maccallum-cancer-centre [SID1234579984]).

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CAR-T is a type of cellular therapy that reprograms the immune cells of cancer patients to recognise and destroy cancerous cells.

As previously reported, PTX has an existing research agreement with Peter Mac focusing on cell therapy enhancement programs, which looks to improve the current CAR-T approaches.

This new agreement extends the relationship between the parties to include the development of the OmniCAR platform.

OmniCAR is a next-generation CAR T therapy platform that offers multiple advantages over its predecessor such as control, safety, flexibility and efficacy.

PTX is developing three OmniCAR programs, including next-generation CAR-T
therapies for acute myeloid leukaemia. Her2+ solid tumours and glioblastoma multiforme.

Under the terms of the research agreement, PTX will have access to the expertise and facilities of Peter Mac.

"We are delighted to deepen our ties with a world-class institute in Peter Mac. Prescient is committed to developing all three OmniCAR programs expediently, and to the highest standard," CEO and Managing Director Steven Yatomi-Clarke said.

"This latest research program with Peter Mac is an important part of Prescient’s development plans, which include institutional and commercial laboratories," he added.

On May 14, 2021 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported financial results for the first quarter ended March 31, 2021 (Press release, Leap Therapeutics, MAY 14, 2021, View Source [SID1234580014]).

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Leap First Quarter Highlights:

Completed enrollment for first-line patient cohort in the DisTinGuish study, a clinical trial evaluating Leap’s anti-Dickkopf-1 (DKK1) antibody, DKN-01, in combination with tislelizumab, BeiGene Ltd.’s anti-PD-1 antibody, with or without chemotherapy, in patients with gastric or gastroesophageal junction cancer (G/GEJ)
Presented updated clinical data from the Phase 2 study of DKN-01 as a monotherapy and in combination with paclitaxel in patients with advanced gynecological malignancies at the Society of Gynecologic Oncology (SGO) 2021 Virtual Annual Meeting on Women’s Cancer
Announced partnership to use a clinically validated tumor expression assay utilizing RNAscope and image analysis with Flagship Biosciences for patient enrollment
"We’re off to a strong start this year as we’ve continued to advance our understanding of DKN-01 and the potential role it can play as both a monotherapy or in combination with existing treatments in multiple DKK1 biomarker defined cancer indications," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "The completion of enrollment of the first-line patient cohort in the DisTinGuish study brings us one step closer to an important milestone for us with our partner BeiGene, anticipated later this year."

DKN-01 Development Update

DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the DKK1 protein. DKK1 modulates the Wnt/Beta-catenin and PI3kinase/AKT signaling pathways, which have an important role in tumor cell signaling and in mediating an immuno-suppressive tumor microenvironment through enhancing the activity of myeloid-derived suppressor cells and downregulating NK cell ligands on tumor cells.

Leap Announced Completion of Enrollment in First-Line Cohort in the DisTinGuish Study of DKN-01 plus Tislelizumab and Chemotherapy in Gastric Cancer – In April 2021, Leap announced the completion of enrollment for the first-line patient cohort in the DisTinGuish study (NCT04363801), a clinical trial evaluating DKN-01 in combination with tislelizumab, BeiGene Ltd.’s anti-PD-1 antibody, with or without chemotherapy, in patients with G/GEJ. The study, which is being conducted in two parts in the United States and the Republic of Korea, enrolled 25 patients with first-line G/GEJ cancer and will enroll up to 48 patients with second-line G/GEJ cancer whose tumors express high levels of DKK1. Initial data is expected in the second half of 2021. Leap is conducting this combination study as part of an exclusive option and license agreement with BeiGene for the development of DKN-01 in Asia (excluding Japan), Australia, and New Zealand.

Leap Presented Final Data for DKN-01 in Gynecologic Cancers – At the SGO 2021 Virtual Annual Meeting on Women’s Cancer, Leap presented the final data from the study of DKN-01 as a monotherapy or in combination with paclitaxel in groups composed of epithelial endometrial cancer (EEC), epithelial ovarian cancer (EOC), or carcinosarcoma (MMMT) patients. The key findings from the study were:

EEC patients and patients with Wnt activating mutations express higher levels of DKK1: EEC patients expressed higher levels of DKK1 and had a higher frequency of Wnt activating mutations than patients with EOC. Within EEC, patients with endometrioid histology had higher DKK1 expression than those with non-endometrioid histology. Patients whose tumors had Wnt activating mutations expressed 14.4 times higher levels of DKK1.

DKN-01 has enhanced activity in patients whose tumors express high levels of DKK1: In the group of 22 EEC patients treated with DKN-01 monotherapy for whom DKK1 expression data was available, patients with DKK1-high tumors (n=7) had greater ORR (14% vs. 0%), DCR (57% vs. 7%), and median PFS (3.0 months vs. 1.8 months [HR 0.39; 95% CI: 0.14, 1.1]) compared to patients with DKK1-low tumors (n=15). Additionally, seven patients did not have DKK1 expression results available, of whom one had a complete response (14%) and five (72%) had a best response of stable disease, including three patients with Wnt activating mutations. In the group of 24 EEC patients treated with DKN-01 plus paclitaxel, 72% of whom had received three or more prior systemic therapies, DKK1-high patients (n=11) had improved median PFS (5.4 months vs. 1.8 months [HR 0.34; 95% CI: 0.12, 0.97]) compared to DKK1-low patients (n=9). Four patients did not have DKK1 expression data available.

Presented DKK1 Biomarker Assay Validation Data – At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021, Leap and its clinical laboratory partner, Flagship Biosciences, presented data on the validation of a DKK1 RNAscope chromogenic in situ hybridization (CISH) assay and digital image analysis solution. Leap and Flagship have demonstrated that the DKK1 RNAscope assay and accompanying digital image analysis solution is specific, sensitive, accurate and reproducible according to Clinical Laboratory Improvements Amendments (CLIA) guidelines. The assay is currently being used to prospectively identify G/GEJ patients with elevated tumoral expression of DKK1 in the ongoing DisTinGuish clinical trial.
Selected First Quarter 2021 Financial Results

Net Loss was $9.1 million for the first quarter 2021, compared to $7.2 million for the same period in 2020. This increase was primarily due to increased development activity for the DKN-01 program and an increase in headcount and compensation expense as the Company has grown throughout the year.

License revenues for each of the first quarter 2021 and 2020 were $0.4 million, and relate to the BeiGene Agreement for the development and commercialization of DKN-01 in Asia (excluding Japan), Australia, and New Zealand. The BeiGene Agreement became effective on January 3, 2020.

Research and development expenses were $6.8 million for the first quarter 2021, compared to $4.6 million for the same period in 2020. The increase of $2.2 million in research and development expenses was primarily due to an increase of $0.8 million in payroll and other related expenses due to an increase in headcount of our research and development full time employees, an increase of $0.6 million in manufacturing costs related to clinical trial material and an increase of $0.8 million in clinical trial costs due to timing of patient enrollment.

General and administrative expenses were $2.7 million for the first quarter 2021, compared to $2.2 million for the same period in 2020. The increase of $0.5 million in general and administrative expenses was due to a $0.3 million increase in payroll and other related expenses during the three months ended March 31, 2021 as compared to the same period in 2020 and a $0.2 million increase in professional fees primarily due to increased recruiting and information technology costs.

Cash and cash equivalents totaled $43.5 million at March 31, 2021. Research and development incentive receivables totaled $0.02 million at March 31, 2021.