On May 14, 2020 Applied DNA Sciences Inc. (Nasdaq: APDN) ("Applied DNA" or the "Company") a leader in Polymerase Chain Reaction (PCR)-based DNA manufacturing that enables in vitro diagnostics, pre-clinical nucleic acid-based therapeutic drug candidates, supply chain security, anti-counterfeiting and anti-theft technology, reported consolidated financial results for the fiscal second quarter and the six months ended March 31, 2020 (Press release, Applied DNA Sciences, MAY 14, 2020, View Source [SID1234558023]).

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"Continued execution on our strategic priorities in our second fiscal quarter resulted in an expansion of our linear DNA story to include COVID-19 vaccine candidate and diagnostic kit programs and the addition of new development customers. These new development customers, several of which are leaders in the field of gene and redirected-cell therapies, demonstrate broadening interest and adoption of our proprietary linear DNA approach to preclinical biotherapeutics and diagnostics development and further validate linear DNA as a viable alternative for plasmid DNA," stated Dr. James A. Hayward, president and CEO of Applied DNA.

"Most of our activities during the quarter were devoted to our COVID-19 development programs that serve to also elevate the profile of our PCR-based LinearDNA platform to the biopharma industry as a powerful, large-scale tool for the rapid manufacture of DNA-based therapeutics," continued Dr. Hayward. "The global focus on the pandemic and the speed with which vaccine candidates are being developed are laying bare the limitations of modern vaccine development that are almost-exclusively reliant on plasmid-based technologies that are often slow and require much more downstream processing. Because our linear DNA approach does not require bacterial fermentation and has the added advantage of essentially no risk of contamination by genes for antibiotic resistance and other genetic contaminants, we believe our platform is well suited for such DNA-based therapeutics as engineered T cells, gene therapies, RNAi, and vaccines, such as for COVID-19 or future emerging infectious diseases. One new development customer, a global Top-20 pharmaceutical manufacturer, is evaluating the full scope of our platform to potentially serve as a foundational tool for their future genetic therapy pipeline. As the industry comes to terms with the limitations of plasmid-based technologies in terms of production speed and risk, we believe this will set the stage for a paradigm shift towards the rapid manufacturing of linear DNA vaccines with our LinearDNA platform as the accelerator."

Concluded Dr. Hayward, "Looking ahead, we remain focused on driving interest and building demand for our linear DNA approach across certain highly-regulated markets, such as drug development, that we believe offer a path to higher and more recurring revenue. Takis Biotech ("Takis") has initiated preclinical animal trials of our LinearDNA version of their vaccine candidates that we believe will demonstrate similar, robust immune responses to their plasmid-based constructs, but with the added advantage of speed and scalability to manufacture for global use. Concurrently, we are preparing for the possibility for Takis to progress to human trials this fall by readying our facility for compliance with the FDA’s cGMP regulations that govern the quality of biologics for human use. We have also developed our Linea COVID-19 high-throughput and high-sensitivity SARS-CoV-2 detection kit to enable mass-testing that can be a crucial tool in the toolbox of health systems and governments in the fight to further ‘flatten the curve’. Having received Emergency Use Authorization from the FDA today, we are now focused on the commercialization of our kit to assist frontline workers leading the charge against the pandemic. Pharmaceutical molecular tagging is another target market and one where the FDA has granted us entry into its Emerging Technology Program that gives us a path to drive the industry’s adoption of our authentication technologies and mitigate participation of counterfeit and adulterated drugs in the legitimate pharmaceutical supply chain. We will continue to build our business development pipeline and execute on our strategy to advance and prove the capabilities of linear DNA to drug developers and the broader pharmaceutical industry."

Fiscal Second Quarter 2020 Financial Results:

·Revenues decreased 29% for the second quarter of fiscal 2020 to $552 thousand, compared with $778 thousand reported in the same period of the prior fiscal year and decreased 13% from $634 for the first quarter of fiscal 2020. This decrease in revenues year over year is due to a decrease of $305 thousand in service revenues, offset by an increase of $79 thousand in product revenues. The decrease in service revenues is primarily attributable to a decrease from a government contract award that ended during the second half of fiscal 2019 and a decline in precommercial feasibility projects in both textiles and cannabis. The increase in product revenues was primarily related to an increase in biopharmaceutical revenues during the quarter ended March 31, 2020.

·Total operating expenses decreased to $3.1 million for the second fiscal quarter of 2020, compared with $3.3 million in the prior fiscal year’s second quarter. This decrease is attributable to a decrease in professional fees of approximately $183 thousand due to reduced legal fees. To a lesser extent, the decrease relates to a reduction of approximately $64 thousand in travel fees as result of travel restrictions associated with COVID-19. This decrease was offset by an increase in research and development expenses of $52 thousand.

·Net loss for the quarter ended March 31, 2020 was $3.0 million, or $0.79 per share, compared with a net loss of $2.7 million, or $3.22 per share, for the quarter ended March 31, 2019, an increase of 10%, and a net loss of $2.7 million, or $1.12 per share, for the quarter ended December 31, 2019.

·Excluding non-cash expenses, Adjusted EBITDA was negative $2.6 million and a negative $2.3 million for the quarters ended March 31, 2020 and 2019, respectively. See below for information regarding non-GAAP measures.

·Cash was $8.7 million at March 31, 2020 that includes the exercise of warrants associated with the November 15, 2019 underwritten public offering ("the offering") totaling approximately $2.8 million in net proceeds. Subsequent to March 31, 2020 the Company received a further $2.9 million of net proceeds through the exercise of warrants associated with the offering. There are a total of approximately 1.5 million warrants outstanding from the offering at May 14, 2020.

Six Months Ended March 31, 2020 Financial Results:

·Revenues decreased 29% for the first half of fiscal 2020 to $1.2 million, compared with $1.7 million reported in the same period of the prior fiscal year. This decrease in revenue year over year is due to a decrease of $472 thousand in service revenues and a decrease of $5 thousand in product revenues. The decrease in service revenues is primarily attributable to a decrease from a government contract award that ended during the second half of fiscal 2019.

·Total operating expenses decreased to $6.1 million for the six months ended March 31, 2020, compared with $7.2 million in the prior fiscal year’s first six months. This decrease is primarily attributable to decreases in payroll of approximately $272 thousand, attributable to headcount reductions, a decrease in stock-based compensation expense of approximately $262 thousand as well as decreases in legal and professional fees of $139 thousand, consulting fees of $121 thousand, reduced travel expenses of $110 thousand and research and development of $93 thousand.

·Net loss for the six-month period ended March 31, 2020 was $5.6 million, or $1.76 per share, compared with a net loss of $5.9 million, or $6.51 per share, for the six months ended March 31, 2019, an improvement of 5%.

·Excluding non-cash expenses, Adjusted EBITDA was negative $5.0 million and a negative $4.9 million for the six months ended March 31, 2020 and 2019, respectively. See below for information regarding non-GAAP measures.

Select Quarterly Operational Highlights:

Applied DNA progressed its COVID-19 development program that spans both vaccine development and clinical diagnostic kit development:

·On May 14, 2020, Applied DNA announced that it had received Emergency Use Authorization for its COVID-19 diagnostic Assay kit from the U.S. Food and Drug Administration ("FDA"). Clinical laboratories in the United States certified under Clinical Laboratory Improvement Amendments can immediately begin ordering and using the LineaTM COVID-19 assay kit to detect SARS-CoV-2, the virus that causes COVID-19;

·On May 5, 2020, the Company announced that it had applied to the U.S. FDA for Emergency Use Authorization for its COVID-19 diagnostic kit, Linea COVID-19, a RT-PCR-based, high-sensitivity and high-throughput COVID-19 diagnostic test;

·On May 4, 2020, Applied DNA and its COVID-19 development partner, Takis, announced that the first injections of their DNA vaccine candidates against the Spike protein (‘S’ gene) of the SARS-CoV-2 virus that causes the COVID-19 disease, have produced neutralizing antibodies in test animals. The studies were completed at the renowned Lazzaro Spallanzani National Institute for Infectious Diseases in Rome. These initial results were obtained using plasmids (the templates for LinearDNA) to baseline results; Applied DNA’s linear DNA dose-response trials begin in the week of May 4, 2020;

·On April 21, 2020, the Company signed an agreement with Stony Brook University Hospital to validate and implement the Company’s COVID-19 diagnostic test: Linea COVID-19. The diagnostic test targets the SARS-CoV-2 Spike (S) gene, which is also the subject of the Company’s COVID-19 vaccine development program;

·On April 15, 2020, Applied DNA announced that it had shipped five linear DNA versions of COVID-19 vaccine candidates to its drug development partner, Takis, to support the immediate start of preclinical animal testing. Shipment of the vaccine candidates follows Takis’ receipt of approval from Italy’s Ministry of Health to begin preclinical testing. Concurrent with Takis’ animal trials, Applied DNA is preparing for cGMP production of selected vaccine candidate(s) to support human trials scheduled to begin this fall; and

·On March 24, the Company announced that it had filed a provisional patent application with the U.S. Patent and Trademark Office for a diagnostic assay for COVID-19.

The Company’s LinearDNA platform for preclinical biotherapeutic (gene and redirected-cell therapies) and diagnostic applications development continued to shift towards broader adoption:

·On March 26, the Company announced that it had shipped CAR T amplicons proprietary to a new development customer, the biologics subsidiary of a U.S.-based, global biopharmaceutical company, for use in evaluating the use of linear DNA; and

·On March 20, Applied DNA announced the signing of a research agreement with a new development customer, a global Top-20 pharmaceutical manufacturer, to evaluate the full scope of the Company’s linear DNA platform to potentially improve the efficacy and safety of the customer’s CAR T therapy pipeline. The research agreement includes the Company’s patented technologies to maximize protein expression and unique linear DNA anti-CD19 CAR T construct for the treatment of acute lymphocytic leukemia.

The Company’s DNA tagging business for supply chain security continued to build awareness and adoption:

·On February 24, the Company announced the receipt of international patents on its Beacon secure optical molecular market technologies in China, Canada, and the U.S., each a key market for brand protection and assurance for Applied DNA;

·On February 20, Applied DNA received Pareve kosher certification for products within its SigNature and CertainT brands from Orthodox Union, the world’s largest and most widely recognized international kosher certification agency. Certification supports the Company’s business development efforts in the food, pharmaceutical, and dietary supplements markets to maximize adoption of its molecular-based tagging platform; and

·On February 19, the Company entered the dietary supplements market with a multi-year contract with Nutrition21, a trusted developer and supplier of novel and clinically substantiated branded ingredients for the nutritional supplement industry. Under the terms of the contract, the application of Applied DNA’s CertainT platform to Nutrition21’s leading sports supplement, Nitrosigine, offers Nutrition21 the certainty of its ingredients all the way through to their customers’ finished products and onto retail shelves and online stores. Separately, the two companies signed an additional agreement to enable Nutrition21 to represent the CertainT platform throughout Nutrition21’s extensive network in the dietary supplement market.

Fiscal Second Quarter 2020 Conference Call Information

The Company will hold a conference call and webcast to discuss its fiscal second quarter-end 2020 results on Thursday, May 14, 2020 at 4:30 PM EDT. To participate on the conference call, please follow the instructions below. While every attempt will be made to answer investors’ questions on the Q&A portion of the call, due to the large number of expected participants, not all questions may be answered.

To Participate:

·Participant Toll Free: 1-844-887-9402
·Participant Toll: 1-412-317-6798
·Please ask to be joined to the Applied DNA call

Live webcast: View Source

Replay (available 1 hour following the conclusion of the live call through May 21, 2020):

·Participant Toll Free: 1-877-344-7529
·Participant Toll: 1-412-317-0088
·Participant Passcode: 10141899
·Webcast replay: View Source

For those unable to attend the live call, a copy of management’s PowerPoint presentation will be available for review under the ‘Events and Presentations’ section of the company’s Investor Relations web site: View Source

On May 14, 2020 Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, reported financial results for the first quarter ended March 31, 2020 (Press release, Alpine Immune Sciences, MAY 14, 2020, View Source [SID1234558022]).

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First Quarter 2020 Highlights and Subsequent Updates:

The U.S. FDA Granted Orphan Drug Designations for ALPN-101 in the Prevention and Treatment of Acute Graft Versus Host Disease. In March, the United States Food and Drug Administration (FDA) granted two orphan drug designations for ALPN-101, our first-in-class inhibitor of the CD28 and ICOS inflammation pathways, for the prevention of, and for the treatment of, acute GVHD.
ALPN-202 Study Design Presented at AACR (Free AACR Whitepaper). In April, Alpine presented the design of NEON-1, the first-in-human study of ALPN-202, our first-in-class clinical stage conditional CD28 costimulator and dual checkpoint inhibitor, at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting I, in the Phase I Trials in Progress Virtual Poster Session.
ALPN-101 Preclinical Data in Inflammatory Bowel Disease (IBD) Presented at 2020 Crohn’s & Colitis Congress and Digestive Disease Week 2020. ALPN-101 modulated inflammatory cytokines in vitro from human IBD peripheral blood mononuclear cells (PBMC) more potently than CD28 or ICOS single-pathway inhibitors and significantly reduced disease activity in vivo in the CD4+CD45RBhi T cell transfer mouse colitis model.
ALPN-101 and ALPN-202 Clinical Trials Ongoing. A phase 1b/2 trial of ALPN-101 in acute GVHD (BALANCE, NCT04227938) has been initiated. The NEON-1 trial of ALPN-202 (NCT04186637) is open for enrollment.
"The first quarter was a highly productive one, with now two open clinical studies for both our lead product candidates," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "We were gratified to receive orphan drug designations for ALPN-101 in acute GVHD, where current standard therapies remain inadequate to prevent or control the disease. We also reiterate our excitement over ALPN-202, which is now poised clinically to address a potential key mechanism accounting for checkpoint inhibitor resistance."

First Quarter 2020 Financial Results

As of March 31, 2020, Alpine had cash, cash equivalents, restricted cash, and short-term investments totaling $36.1 million. Net cash used in operating activities for the first quarter ended March 31, 2020 was $9.7 million compared to $11.2 million for the first quarter ended March 31, 2019. Alpine recorded a net loss of $5.5 million and $12.4 million for the first quarters ended March 31, 2020 and 2019, respectively.
Research and development expenses for the first quarter ended March 31, 2020 were $4.9 million compared to $10.4 million for the first quarter ended March 31, 2019.
General and administrative expenses for the first quarter ended March 31, 2020 were $1.8 million compared to $2.3 million for the first quarter ended March 31, 2019.
Cash Guidance

Alpine expects to have sufficient cash to fund its planned operations for at least the next 12 months, including the clinical advancement of Alpine’s lead autoimmune/inflammatory program, ALPN-101, and its lead oncology program, ALPN-202.

For additional information regarding Alpine’s planned operations, please refer to "Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operation – Liquidity and Capital Resources" in Alpine’s Quarterly Report on Form 10-Q for the three months ended March 31, 2020, which Alpine anticipates filing with the Securities and Exchange Commission on or about May 14, 2020.

About ALPN-101

ALPN-101 is a novel Fc fusion protein of a human inducible T cell costimulator ligand (ICOSL) variant immunoglobulin domain (vIgD), a first-in-class therapeutic designed to inhibit simultaneously the CD28 and ICOS inflammation pathways. CD28 and ICOS are closely related costimulatory molecules with partially overlapping roles in T cell activation likely playing a role in multiple autoimmune and inflammatory diseases. In preclinical models of graft versus host disease, inflammatory arthritis, connective tissue disease, and multiple sclerosis, ALPN-101 demonstrates efficacy superior to agents blocking the CD28 – CD80/86 and/or ICOS – ICOSL pathways alone. A phase 1b/2 trial of ALPN-101 in acute GVHD (BALANCE, NCT04227938) has been initiated.

About ALPN-202

ALPN-202 is a first-in-class, conditional CD28 costimulator and dual checkpoint inhibitor with the potential to improve upon the efficacy of combined checkpoint inhibition while limiting significant toxicities. Preclinical studies of ALPN-202 have successfully demonstrated superior efficacy in tumor models compared to checkpoint inhibition alone. A phase 1 trial of ALPN-202 in advanced malignancies (NEON-1, NCT04186637) is open for enrollment.

On May 14, 2020 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, 2020 (Press release, Leap Therapeutics, MAY 14, 2020, View Source [SID1234558008]).

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Leap First Quarter Highlights:

·Entered into an exclusive option and licence agreement with BeiGene, Ltd. for the clinical development and commercialization of DKN-01 in Asia (excluding Japan), Australia, and New Zealand
·Completed a $27 million equity financing with BeiGene, Perceptive Advisors, and a lead institutional investor
·Presented updated data for DKN-01 monotherapy that showed a complete response and partial response in endometrial cancer patients with additional reponses observed for the DKN-01 plus paclitaxel combination in carcinosarcoma patients
·Presented final data from its Phase 1/2 clinical trial of DKN-01 plus Keytruda (pembrolizumab) in patients with advanced or recurrent esophagogastric cancer at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Gastrointestinal Cancers Symposium
·Announced Executive Leadership Team and Board of Directors changes

"We’ve made great strides in the first quarter, as we continue to generate compelling data demonstrating DKN-01’s potential as a single agent or in combination with PD-1 antibody therapy or chemotherapy in treating multiple biomarker-defined cancer indications," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "Our partnership with BeiGene for the clinical development and commercialization of DKN-01 is off to a strong start. Site initiation activities are now underway for our combination study of DKN-01 plus tislelizumab, BeiGene’s anti-PD-1 antibody, in patients with gastric or gastroesophageal junction cancer, and we look forward to dosing the first patients in the second half of this year."

Business Update

·Leap and BeiGene Sign Exclusive Option and License Agreement for DKN-01 – Leap and BeiGene announced an exclusive option and license agreement for the clinical development and commercialization of DKN-01 in Asia (excluding Japan), Australia, and New Zealand. Under the terms of the agreement, Leap received an upfront cash payment of $3 million from BeiGene. Leap will be eligible to receive an additional payment from BeiGene upon BeiGene’s exercise of the option and payments based upon the achievement of certain development, regulatory, and sales milestones, for a total deal value of up to $132 million. In addition, Leap is entitled to receive tiered royalties on any product sales of DKN-01 in the licensed territory. Leap will retain exclusive rights for the development, manufacturing, and commercialization of DKN-01 for the United States, Europe, Japan, and the rest of the world.

·Leap Completes $27 Million Equity Financing with BeiGene, Perceptive Advisors and Another Institutional Investor – Simultaneous with the BeiGene Agreement, Leap also entered into a securities purchase agreement and issued and sold 1,421,801 shares of Series A mandatorily convertible preferred stock to a lead investor and an aggregate of 1,137,442 shares of Series B mandatorily convertible preferred stock to BeiGene and Perceptive Advisors. On March 5, 2020, the Leap stockholders approved the conversion of the Series A preferred stock into a pre-funded warrant to purchase 14,413,902 shares of common stock and the conversion of the Series B preferred stock into 11,531,133 shares of common stock. Each investor also received a warrant to purchase an equal number of shares at an exercise price of $2.11 per share. In addition, the lead investor has the right to appoint a member to Leap’s Board of Directors.

·Leap Makes Executive Leadership Changes – Leap announced the following changes to the Company’s Executive Leadership Team and Board of Directors:
oChristopher K. Mirabelli, Ph.D., stepped down from his role as President and Chief Executive Officer and will continue to serve as the Chairman of Leap’s Board of Directors and an employee, providing ongoing leadership around the Company’s research and development efforts.
oDouglas E. Onsi was named President and Chief Executive Officer, while maintaining his previous role as Chief Financial Officer. Mr. Onsi will also serve as a member of the Board of Directors.
oJohn Littlechild stepped down from Leap’s Board of Directors.
oCynthia Sirard, M.D., was promoted to Chief Medical Officer from Vice President, Clinical Research and Development.
oMark O’Mahony was promoted to Chief Manufacturing Officer from Vice President of Chemistry, Manufacturing, and Controls.

DKN-01 Clinical Update

DKN-01 is a humanized monoclonal antibody that binds to and blocks the activity of the Dickkopf-1 (DKK1) protein, a modulator of Wnt/Beta-catenin signaling. DKK1 has an important role in tumor cell signaling and in mediating an immuno-suppressive tumor microenvironment.

·Leap Presented Updated Data for DKN-01 Monotherapy and Paclitaxel Combination in Gynecologic Cancers – Leap announced updated clinical data from its ongoing Phase 2 clinical trial of DKN-01, as both a monotherapy and in combination with paclitaxel chemotherapy, in patients with advanced gynecological malignancies. Leap hosted a conference call with Rebecca Arend, M.D., Assistant Professor and Associate Scientist, Gynecologic Oncology Clinic, The University of Alabama at Birmingham School of Medicine Comprehensive Cancer Center Experimental Therapeutics Program, on April 23, 2020, to discuss the data. Key findings from the P204 Study include the following:

DKN-01 Single Agent Activity

○Endometrial Cancer: Twenty-nine endometrial cancer patients were enrolled in the DKN-01 monotherapy arm, over 75% of whom had experienced three or more prior lines of therapy. Of those patients, 26 were evaluable for response. In the 20 patients with a Wnt signaling alteration, one patient (5%) has an ongoing complete response, one patient (5%) had a partial response, eight patients (40%) had a best response of stable disease, and 10 patients (50%) had progressive disease, representing an overall response rate (ORR) of 10% and a disease control rate (DCR) of 50%. In the group of six patients without any Wnt signaling alterations, one patient (16.6%) had a best response of stable disease and five patients (83.3%) had progressive disease.

○Carcinosarcoma: Ten patients with carcinosarcoma have been enrolled in the DKN-01 monotherapy arm, five of whom were evaluable for response as of the data-cut off date. Two patients (40%) had a best response of stable disease, one of whom has continued on monotherapy for nearly two years, and three patients (60%) had progressive disease. Five patients had not reached their first tumor assessment.

DKN-01 plus Paclitaxel Combination Activity

○Carcinosarcoma: Fifteen patients with carcinosarcoma were enrolled in the DKN-01 plus paclitaxel arm, six of whom were evaluable for response as of the data-cut off date. Two patients (33%) have had a partial response, one patient (17%) has had a best response of stable disease, and three patients (50%) had progressive disease, representing an ORR of 33% and a DCR of 50%. Nine patients had not reached their first tumor assessment.

○Endometrial Cancer: Twenty-five patients with heavily pretreated endometrial cancer were enrolled in the DKN-01 plus paclitaxel arm. All of these patients had previously received paclitaxel, 44% had received hormonal therapies, 32% had received bevacizumab, 20% had received immunotherapy, and 12% had received a PARP inhibitor. Of those patients, 22 were evaluable for response. A total of 12 patients (55%) have had a best response of stable disease, and ten patients (45%) had progressive disease.

○Monotherapy Patients with Wnt activating mutations have longer Progression-Free Survival (PFS) and Overall Survival (OS): In a pooled analysis of all DKN-01 monotherapy patients, patients with Wnt activating mutations have demonstrated a longer median PFS of 168 days as compared to patients without Wnt activating mutations with median PFS of 56 days. Median OS has not been reached in the Wnt activating mutation group in the pooled analysis as compared to median OS of 328 days in the non-Wnt activating mutation group.

○Monotherapy Patients with DKK1-high tumors have longer PFS and OS: DKK1 expression as measured by in situ hybridization RNAscope assay is currently available for 68 of the patients on the study, 32 of whom were treated with DKN-01 monotherapy. Seven patients (22%) were identified as having DKK1-high tumoral expression (DKK1-high). Consistent with the results from Leap’s study in patients with esophagogastric cancer, patients whose tumors are DKK1-high have prolonged median PFS of 168 days as compared to patients with tumors that are DKK1-low with median PFS of 56 days. Median OS was 450 days in the DKK1-high group in the pooled analysis as compared to 276 days in the DKK1-low group.

·Leap Presented Final Data for DKN-01 plus KEYTRUDA (pembrolizumab) in Esophagogastric Cancer: Leap completed its multi-part Phase 1/2 clinical study of DKN-01, as a monotherapy and in combination with paclitaxel or KEYTRUDA (pembrolizumab), in patients with advanced esophagogastric cancer. At the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2020 Gastrointestinal Cancers Symposium, Leap presented final data for the combination of DKN-01 plus pembrolizumab. The combination of DKN-01 and pembrolizumab in gastroesophageal junction cancer (GEJ) and gastric cancer (GC) patients demonstrated improved outcomes in DKK1-high patients who had not previously been treated with PD-1/PD-L1 therapy. DKK1-high patients experienced over 22 weeks median PFS and nearly 32 weeks median OS, with a 50% ORR and 80% DCR in ten evaluable patients. DKK1-low patients experienced nearly 6 weeks median PFS and over 17 weeks OS, with a 20% DCR in 15 evaluable patients.

·Site Initiation Activities Underway for DKN-01 plus Tislelizumab Combination Study: As part of the collaboration with BeiGene, Leap intends to study the combination of DKN-01 and BeiGene’s anti-PD-1 antibody, tislelizumab. The Company plans to evaluate approximately 40 patients with second-line GC or GEJ whose tumors are DKK1-high. In addition, Leap will evaluate the combination of DKN-01 with tislelizumab and chemotherapy in approximately 20 patients with first-line GC or GEJ. Site initiation activities for this clinical trial are underway, and the Company expects to dose the first patients in the second half of 2020.

Selected First Quarter 2020 Financial Results

Net loss was $7.2 million for the first quarter 2020, compared to $8.6 million for the same period in 2019. This decrease was primarily due to revenue recognized from the BeiGene agreement and a decrease in clinical development expenses, offset by a non-cash charge for foreign currency losses associated with changes in the Australian dollar exchange rate related to activities of Leap’s Australian subsidiary.

Research and development expenses was $4.6 million for the first quarter 2020, compared to $6.8 million for the same period in 2019. The decrease was primarily due to decreases in clinical trial costs due to timing of patient enrollment and decreases in consulting fees associated with research and development activities.

General and administrative expenses was $2.2 million for the first quarter 2020, compared to $2.0 million for the same period in 2019. The increase was primarily due to increases in legal, audit and consulting fees associated with corporate and business development activities.

Cash, cash equivalents and marketable securities totaled $25.5 million at March 31, 2020. Research and development incentive receivables, current and long term, totaled approximately $0.2 million at March 31, 2020.

On May 14, 2020 TCR2 Therapeutics Inc. (Nasdaq: TCRR), a clinical-stage immunotherapy company developing the next generation of novel T cell therapies for patients suffering from cancer, reported financial results for the first quarter ended March 31, 2020 and provided a corporate update (Press release, TCR2 Therapeutics, MAY 14, 2020, View Source [SID1234558007]).

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"Based on our clinical progress to date and a growing confidence in our TRuC platform, we are advancing a third mono TRuC-T cell candidate targeting CD70, a clinically validated target which will allow us to make further inroads into both solid tumors and hematological malignancies," said Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics. "In addition, the expression of CD70 in tumors where CD19 and mesothelin are overexpressed represents a good opportunity for our dual TRuCs. With this new program and the strengthening of our Board with the appointments of Axel Hoos and Stephen Webster, we are well-positioned to deliver on the near- and long-term value opportunities of our platform as we approach data for our lead assets in 2020."

"Despite steps taken to protect our lead programs from the impact of COVID-19, we are updating our TC-210 guidance to expect an interim update from the Phase 1 portion in mid-2020, allowing for additional time to work with clinical investigators and patients on activities that require visits to clinical sites, including data monitoring. We look forward to presenting on the efficacy, safety and translational data in the very near future," added Dr. Menzel.

Recent Developments

The U.S. Food and Drug Administration granted orphan drug designation to TC-110 for the treatment of acute lymphoblastic leukemia.

TCR2 nominated a CD70 targeted TRuC-T cell product candidate designed to treat patients with either hematological malignancies or solid tumors, including potential indications such as acute myeloid leukemia, non-Hodgkin lymphoma, renal cell carcinoma, nasopharyngeal cancer, glioblastoma, and malignant pleural/peritoneal mesothelioma.

TCR2 announced the appointment of Axel Hoos, M.D., Ph.D., to its Board of Directors. Dr. Axel Hoos is Senior Vice President, R&D Governance Chair, and Therapeutic Area Head for Oncology at GlaxoSmithKline Pharmaceuticals. He is responsible for technical and funding decisions and leads the Oncology business including discovery and development with the four focus areas of immuno-oncology, epigenetics, cell & gene therapy and synthetic lethality.

TCR2 announced the appointment of Stephen Webster to its Board of Directors. Mr. Stephen Webster served as Chief Financial Officer of Spark Therapeutics until its acquisition by Roche and brings nearly 30 years of experience in raising capital, business development transactions and operations.

COVID-19 Update

As the Company balances the commitment to treat our cancer patients while mitigating the risk of viral spread to patients, employees and their families, TCR2 has instituted protective policies consistent with guidelines provided by the Centers for Disease Control and Prevention at all TCR2 facilities.

COVID-19 has significantly impacted the global healthcare system, including the conduct of clinical trials as medical institutions prioritize the treatment of those afflicted with COVID-19. TCR2 continues to closely monitor the adverse impact of the COVID-19 pandemic on operations and ongoing clinical and preclinical development.

While the Company believes it has been able, to date, to mitigate some of the impact from the COVID-19 pandemic on its ongoing clinical programs, the Company is committed to providing an interim update of the Phase 1 portion of the TC-210 Phase 1/2 clinical trial in mid-2020 and the Phase 1 portion of the TC-110 Phase 1/2 clinical trial in the second half of 2020.

Anticipated Milestones

TCR2 anticipates an interim update from the Phase 1 portion of the TC-210 Phase 1/2 clinical trial for patients with mesothelin-expressing solid tumors in mid-2020.

TCR2 anticipates an interim update from the Phase 1 portion of the TC-110 Phase 1/2 clinical trial for patients with CD19+ non-Hodgkin lymphoma or adult acute lymphoblastic leukemia in 2H20.

TCR2 anticipates clinical production of TRuC-T cells at its manufacturing facility in Stevenage, UK, in 2H20.

TCR2 anticipates a target IND filing for the third mono TRuC-T cell program targeting CD70 in 1H21.

Financial Highlights

Cash Position: TCR2 ended the first quarter of 2020 with $140.7 million in cash, cash equivalents, and investments compared to $158.1 million as of December 31, 2019. Net cash used in operations was $16.4 million for the first quarter of 2020 compared to $10.8 million for first quarter of 2019. TCR2 projects net cash use of $60-70 million for 2020.

R&D Expenses: Research and development expenses were $12.0 million for the first quarter of 2020 compared to $7.9 million for the first quarter of 2019. The increase in R&D expenses is primarily related to increase in headcount, activities related to the Phase 1/2 clinical trial of TC-210 and activities related to the Phase 1/2 clinical trial of TC-110.

G&A Expenses: General and administrative expenses were $4.3 million for the first quarter of 2020 compared to $2.9 million for the first quarter of 2019. The increase in general and administrative expenses was primarily due to an increase in personnel costs and costs associated with operations as a public company.

Net Loss: Net loss was $15.5 million for the first quarter of 2020 compared to $9.9 million for the first quarter of 2019, driven predominantly by increased R&D expenses.

Upcoming Events

TCR2 Therapeutics management is scheduled to participate at the following upcoming conference.

Jefferies Global Healthcare Conference: Garry Menzel, Ph.D., President and Chief Executive Officer of TCR2 Therapeutics, and Ian Somaiya, Chief Financial Officer of TCR2 Therapeutics, will participate in a fireside chat using a virtual platform on Tuesday, June 2, 2020 at 9:00am ET

On May 14, 2020 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics targeting infectious diseases and cancers based on its proprietary arenavirus platform, reported its financial results and corporate update for the first quarter ended March 31, 2020 (Press release, Hookipa Pharma, MAY 14, 2020, View Source [SID1234558006]).

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"The current unusually challenging times are impacting our industry significantly. HOOKIPA continues to progress well in areas which are under our control," commented Joern Aldag, HOOKIPA’s Chief Executive Officer. "The HOOKIPA team is continuing to activate sites for our oncology candidate HB-201 and patients are accruing. We expect to submit the combined HB-202/201 IND to the U.S. Food and Drug Administration in the first half of 2020, as previously forecasted. For HB-101, although enrollment of kidney transplant patients is currently hampered by the impact of COVID-19, we expect to report preliminary safety and immunogenicity data in July 2020 and efficacy data by the end of 2020, roughly at the same time we expect to report preliminary safety and efficacy for HB-201. With approximately $105 million in cash and cash equivalents, we believe that we are well funded beyond multiple clinical data points across our programs. In addition, I am particularly proud of the remarkable contribution of the HOOKIPA team voluntarily accepting a significant reduction of their fixed salaries and thereby extending our cash runway."

R&D Pipeline Update and Clinical Progress

HB-101, lead product candidate in infectious diseases
HOOKIPA’s VaxWave-based prophylactic Cytomegalovirus (CMV) vaccine candidate, HB‑101, is in a randomized, double‑blinded Phase 2 clinical trial in patients awaiting kidney transplantation who are at risk for CMV-associated complications post-transplant. Due to the COVID-19 pandemic, nearly all ongoing Phase 2 trial sites have suspended patient enrollment, and it remains unclear when kidney organ transplants will resume at any of the trial sites.

By the end of July 2020, HOOKIPA expects to report safety data (on approximately one-third of the total 150 patients to be enrolled) as well as immunogenicity data (on approximately one-quarter of the total patients to be enrolled). The immunogenicity data will focus on CMV-neutralizing antibody responses. Analyses of cellular immune responses, including CD8+ T cells, have been limited to date due to the complexities of sample collection, transport, and analysis. HOOKIPA reiterates its guidance to deliver preliminary efficacy data by the end of 2020. Due to the COVID-19-impacted accrual, the timing of study completion will be delayed.

HB-201 and HB-202, programs for the treatment of Human Papillomavirus-positive cancers
HOOKIPA’s lead oncology product candidates, HB‑201 and HB‑202, are in development for the treatment of Human Papillomavirus‑positive (HPV+) cancers. In December 2019, HOOKIPA initiated the Phase 1/2 clinical trial for HB-201 and expects preliminary results in late 2020 or early 2021. The open label, dose escalating Phase 1/2 clinical trial is evaluating HB-201 in HPV16+ cancers, alone and in combination with an approved checkpoint inhibitor. HOOKIPA plans to enroll 100 patients in total with 20 patients in each dose escalation and expansion group, respectively. Enrollment of the first group of patients receiving the intravenously administered first dose level has been completed and the trial is accruing patients at the next higher dose level.

HOOKIPA remains on track to file the HB‑202/201 Initial New Drug (IND) submission with the U.S. Food and Drug Administration in the first half of 2020. The planned clinical trial combining HB-202 with HB-201, also in patients with HPV16+ cancers, is an open label, dose escalation Phase 1/2 trial with the primary endpoint to evaluate safety and tolerability. That trial is expected to commence later in 2020.

Strategic Collaborations

Gilead Sciences Collaboration for HIV and HBV Therapeutic Vaccines
During 2019, HOOKIPA received $6.0 million in milestone payments from Gilead for the delivery of research vectors and for advancing the programs towards clinical trials. Based on preclinical data generated to date, Gilead committed to preparations to advance the HBV and HIV vectors toward development, with the HBV development decision triggering a milestone payment of $4.0 million, which the Company received in early 2020. To enable the development activities and expanded research programs, Gilead agreed to reserve manufacturing capacity and increase reimbursement budgeted for the Company’s expanded resources allocated to the Gilead collaboration.

Other
At HOOKIPA’s New York City headquarters, all employees will continue to work from home and are fully operational. The Austrian government has removed some of the previously mandated COVID-19 restrictions that it put in place during March 2020, and this has allowed HOOKIPA’s Austrian site to ramp up its activities. The Company plans to increase its lab capacity to the extent safe and reasonable, and at the same time encourages all employees who can reasonably work from home to continue to do so.

First Quarter 2020 Financial Results

Cash Position:

HOOKIPA’s cash, cash equivalents and restricted cash as of March 31, 2020 was $104.9 million compared to $113.6 million as of December 31, 2019. The decrease was primarily attributable to cash used in operating activities.

Revenue was $3.7 million for the three months ended March 31, 2020 compared to $2.2 million for the three months ended March 31, 2019. Revenue was driven by the recognition of milestone payments and partial recognition of the upfront payment as well as cost reimbursements received under the Collaboration Agreement with Gilead.

Research and Development Expenses:

HOOKIPA’s research and development expenses were $11.5 million for the three months ended March 31, 2020 compared to $10.2 million for the three months ended March 31, 2019.

The primary drivers of this increase were $1.3 million higher personnel expenses along with a general increase in internal research and development expenses, partially offset by an overall decrease in direct research and development expenses by $0.6 million. The latter reduction was a consequence of reduced activity around our earlier stage programs; in addition, preparation costs of clinical trials for our HB-201 and HB-202 programs were lower than in the same period last year. This decrease was partially offset by an increase in the direct costs related to our collaboration with Gilead due to an acceleration program and a modest increase in direct expenses for the HB-101 Phase 2 trial.

General and Administrative Expenses: General and administrative expenses amounted to $4.6 million for the three months ended March 31, 2020 compared to $2.7 million for the three months ended March 31, 2019. The increase was mainly due to personnel related expenses, an increase in professional and consulting fees, as well as costs related to being a public company, such as premiums for directors and officers liability insurance.

Net Loss: HOOKIPA’s net loss was $10.9 million for the three months ended March 31, 2020 compared to a net loss of $9.3 million for the three months ended March 31, 2019. This increase was driven by higher research and development expenses, in particular for HOOKIPA’s oncology programs, and an increase in general and administrative expenses following HOOKIPA’s IPO.