On May 12, 2022 Checkmate Pharmaceuticals, Inc. (Nasdaq: CMPI) ("Checkmate"), a clinical stage biopharmaceutical company focused on developing its proprietary technology to harness the power of the immune system to combat cancer, reported first quarter 2022 financial results and provided a business update (Press release, Checkmate Pharmaceuticals, MAY 12, 2022, View Source [SID1234614368]).

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"We remain focused on the execution of the clinical program for vidutolimod and are delighted with the opportunity to become part of Regeneron, who will help to accelerate the development of vidutolimod as a potential novel treatment for multiple tumor types," said Alan Bash, President and Chief Executive Officer of Checkmate. "This is an exciting opportunity for all stakeholders as we bring this innovative medicine forward and work to make a meaningful difference for patients with difficult to treat cancers."

Recent Business Updates

On April 19, 2022, Regeneron Pharmaceuticals, Inc. and Checkmate Pharmaceuticals, Inc. announced a definitive agreement for the acquisition of Checkmate by Regeneron at an all-cash price of $10.50 per share of Checkmate common stock. The proposed acquisition values Checkmate at a total equity value of approximately $250 million. On May 2, 2022, Scandinavian Acquisition Sub, Inc., a wholly owned subsidiary of Regeneron, commenced a tender offer for all of the outstanding shares of Checkmate common stock. The transaction is expected to close in mid-2022.
First Quarter 2022 Financial Results

Research and development expenses (R&D): R&D expenses for the first quarter of 2022 were $11.6 million, compared to $10.4 million for the same period in the prior year. This increase was primarily driven by higher third-party contract research organization, internal personnel and consulting costs related to our ongoing clinical trials. First quarter 2021 expenses included the impact of a $2.0 million milestone payment to Kuros in March 2021, which became payable upon initiating dosing of the first patient in a Phase 2 clinical trial. There was no corresponding expense in Q1 2022.
General and administration expenses (G&A): G&A expenses for the first quarter of 2022 were $4.2 million, compared to $3.8 million for the same period in the prior year. This increase was primarily attributable to professional fees incurred in Q1 2022 in connection with the proposed acquisition by Regeneron and recruiting costs associated with hiring a chief executive officer in March 2022.
Cash, cash equivalents and investments: Cash, cash equivalents and available-for-sale investments were $60.1 million as of March 31, 2022.

On May 12, 2022 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the first quarter ended March 31, 2022 and recent corporate highlights (Press release, Checkpoint Therapeutics, MAY 12, 2022, View Source [SID1234614367]).

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "The positive topline results generated in the first quarter from our ongoing registrational trial of cosibelimab in metastatic cutaneous squamous cell carcinoma ("cSCC") laid the foundation for a potentially transformational year for our lead immunotherapy product candidate and for our company as a whole. We remain on track to submit our U.S. Biologics License Application ("BLA") for cosibelimab later this year and continue to evaluate partnership opportunities for the potential commercialization of cosibelimab in Europe and other key territories worldwide." Mr. Oliviero continued, "Our focus remains on advancing our pipeline of potentially life-saving novel oncology therapies with the goal of expanding patient access globally through a disruptive pricing strategy, beginning with the $30 billion and growing PD-(L)1 market."

Recent Corporate Highlights:

In January 2022, Checkpoint announced positive topline results from the ongoing registration-enabling clinical trial evaluating the safety and efficacy of its anti-PD-L1 antibody, cosibelimab, administered as a fixed dose of 800 mg every two weeks in patients with metastatic cSCC. The study met its primary endpoint, with cosibelimab demonstrating a confirmed objective response rate of 47.4% (95% CI: 36.0, 59.1) based on independent central review of 78 patients enrolled in the metastatic cSCC cohort using Response Evaluation Criteria in Solid Tumors version 1.1 criteria. Checkpoint intends to submit a BLA for cosibelimab in late 2022, followed by a Marketing Authorization Application submission in Europe and other territories worldwide. With a potentially favorable safety profile versus anti-PD-1 therapy and a plan to commercialize at a substantially lower price, Checkpoint believes cosibelimab has the potential to be a market disruptive product in the $30 billion and growing PD-(L)1 class.
In April 2022, Checkpoint announced that the results of its pivotal trial of cosibelimab in cSCC were selected for poster presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held at McCormick Place, in Chicago, June 3-7, 2022.
Financial Results:

Cash Position: As of March 31, 2022, Checkpoint’s cash and cash equivalents totaled $41.5 million, compared to $54.7 million at December 31, 2021, a decrease of $13.2 million.
R&D Expenses: Research and development expenses for the first quarter of 2022 were $14.7 million, compared to $4.2 million for the first quarter of 2021, an increase of $10.5 million. Research and development expenses for the first quarters of 2022 and 2021 each included $0.2 million of non-cash stock expenses.
G&A Expenses: General and administrative expenses for the first quarter of 2022 were $2.2 million, compared to $2.4 million for the first quarter of 2021, a decrease of $0.2 million. General and administrative expenses for the first quarter of 2022 included $0.7 million of non-cash stock expenses, compared to $1.2 million for the first quarter of 2021.
Net Loss: Net loss attributable to common stockholders for the first quarter of 2022 was $16.8 million, or $0.20 per share, compared to a net loss of $6.5 million, or $0.09 per share, in the first quarter of 2021. Net loss for the first quarter of 2022 included $0.9 million of non-cash stock expenses, compared to $1.4 million for the first quarter of 2021.

On May 12, 2022 Ensysce Biosciences, Inc. ("Ensysce" or the "Company") (NASDAQ: ENSC, OTC: ENSCW), a clinical-stage biotech company applying transformative chemistry to improve prescription drug safety and performance focused on reducing abuse and overdose, reported financial results for the first quarter 2022 (Press release, Leisure Acquisition, MAY 12, 2022, View Source [SID1234614365]).

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Dr. Lynn Kirkpatrick, Chief Executive Officer of Ensysce, commented, "The first few months of 2022 are off to an exciting start, evident in the most recent clinical results from trials PF614-102 and PF614-MPAR-101. As we progress on our studies and clinical trials, we are closer to realizing our mission – providing abuse and overdose protection where needed. I look forward to our upcoming corporate update call next week to discuss the recent results from the trials, especially regarding the longer-lasting profile of PF614 and the encouraging initial data from our overdose protection study. Additionally, we expect the Bioequivalence (BE) trial data of our TAAP opioid, PF614, to be available prior to the end of the second quarter, positioning PF614 as our first commercial candidate and demonstrating progress towards our goal to bring a unique pipeline of products to the market."

TAAPTM (Opioid Abuse Deterrent Program) Updates

●On May 5, 2022, the Company announced clinical trial results from trial PF614-102 confirming the safety and longer-lasting profile of PF614 versus Oxycontin.

●The results of the study show the longer-lasting half-life of PF614 versus OxyContin as seen in the prior Phase 1 single-ascending dose study of PF614 oral solution versus OxyContin. We believe this data confirms the findings from our Phase 1 study that demonstrate PF614 should provide true twice-daily dosing.

●The safety data for the study also showed that PF614 performed similarly to OxyContin with no serious treatment emergent adverse events recorded.

The second part of the PF614-102 study, the bioequivalence (BE) arm, continues to be analyzed and as reported previously, we anticipate that this BE data will be available by the end of the second quarter of 2022. The Company believes that the data will support the 505(b)(2) regulatory path for clinical development of PF614, an abbreviated pathway to FDA approval.

●Human abuse liability (HAL) studies to determine labeling claims for PF614 are scheduled to initiate in the second quarter of 2022.

●PF614 is designed using the abuse protective platform TAAP, Trypsin Activated Abuse Protection, a chemical modification which inactivates the active ingredient in Ensysce’s opioid products, including PF614, until swallowed and exposed to the enzyme trypsin in the digestive system. Our TAAP platform, which we believe provides abuse protection and resistance to manipulation and other forms of recreational drug abuse, should also control the rate of release of the active opioid.

MPARTM (Opioid Abuse Deterrent and Overdose Protection Program) Updates

●On May 5, 2022, the Company announced initial results from trial PF614-MPAR-101, providing first human data showing the potential for overdose protection with MPAR, Multi-Pill Abuse Resistance.

●This data demonstrated how the combination product PF614-MPAR could reduce the trypsin activation and reduce the release of oxycodone in a simulated overdose situation. It also demonstrated the PF614 in the systemic circulation (simulated injection) did not convert to oxycodone. We believe this is the first step to identifying the first MPAR drug product that will be marketed in the coming years.

●The study is continuing with additional subjects enrolling to receive PF614 and trypsin inhibitor nafamstat in various combinations through the third quarter of 2022. Safety and pharmacokinetic results are expected by the end of the third quarter 2022.

Financial Results

●Cash – Cash and cash equivalents were $8.4 million as of March 31, 2022, as compared to $12.3 million at December 31, 2021. Cash used in operating activities for the first quarter of 2022 totaled $3.4 million, an increase from $0.5 million in the first quarter of 2021 that primarily resulted from the clinical advancement of our product candidates and increased costs related to operating as a public company.

●Federal Grants – Funding from federal grants was $0.6 million for the first quarter of 2022 compared to $0.3 in the comparable year ago quarter. Funding increased by $0.4 million under the MPAR Grant, offset by a decrease of $0.1 million under the OUD Grant, due to the timing of research activities eligible for funding.

●Research & Development Expenses – R&D expenses were $3.1 million for the first quarter of 2022 compared to $0.3 million in the comparable year ago quarter. The increase was primarily the result of increased external research and development costs related to preclinical and clinical programs for PF614 and PF614-MPAR.

●General & Administrative Expenses – G&A expenses were $2.3 million for the first quarter of 2022 compared to $0.5 million for the same period in 2021. The increase was primarily a result of increased expenses related to operating as a public company, including legal and accounting fees and director and officer insurance expenses.

●Other Income (Expense) – Total other income (expense), was income of $3.9 million in the first quarter of 2022 and expense of $0.4 million in the first quarter of 2021. The income in the 2022 period is due to non-cash valuation adjustments of current obligations of the Company. The 2021 expense primarily reflects non-cash interest expense on notes that were converted on June 30, 2021.

●Net Income (Loss) – Net loss for the first quarter of 2022 was $1.0 million compared to net loss of $0.9 million for the comparable year ago period. As we are a clinical stage biotech company, our research and development of, and regulatory approvals for, our product candidates are expected to continue, resulting in expected losses for the foreseeable future.

Additional Company Highlights

●On February 8, 2022, the Company added further depth to its Board of Directors with the appointment of Lee Rauch.

●On April 18, 2022, the Company announced the appointment of Dr. Nily Osman as Chief Medical Officer.

Corporate Update Conference Call

Management will host a corporate update conference call on Tuesday, May 17, 2022, at 11:00am ET to provide a corporate update and review the recently discussed results from Clinical Trials PF614-102 and PF614-MPAR-101. The call will conclude with Q&A from participants. An accompanying presentation will be posted prior to the call to the Company’s investor relations website.

Please dial in at least 10 minutes before the start of the call to ensure timely participation. A playback of the call will be available through Tuesday, June 14, 2022. To listen, call 1-844-512-2921 within the United States and Canada or 1-412-317-6671 when calling internationally. Please use the replay pin number 13729812.

On May 12, 2022 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, reported financial results for the first quarter ended March 31, 2022 and provided a corporate update (Press release, Vincerx Pharma, MAY 12, 2022, View Source [SID1234614364]).

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"In April, we presented positive preclinical and preliminary clinical data on PTEFb/CDK9 inhibitor VIP152 at AACR (Free AACR Whitepaper). These data, coupled with our findings presented at ASH (Free ASH Whitepaper) last year, suggest that VIP152 has the potential to provide new treatment options for patients across various MYC and MCL-1-driven tumor types," said Ahmed Hamdy M.D., Chief Executive Officer of Vincerx.

"We continue dosing patients in our VIP152 clinical program and are seeing improvements in trial enrollment following a challenging pandemic year," added Dr. Hamdy. "This June, we are excited for our poster presentation at the annual European Hematology Association (EHA) (Free EHA Whitepaper) meeting, which will show data from patients with lymphoma treated with VIP152. We remain on track to initiate Phase 2 studies of VIP152 in the second half of this year."

"Looking to the second half of this year and into 2023, we look forward to continuing to advance our preclinical bioconjugation platform – a diverse, modular platform of linkers and payloads that can be conjugated with antibodies and small molecules to create novel targeted therapeutics for a broad range of solid tumors and hematologic malignancies. We remain on track to file an IND in the second half of this year for VIP236, a small molecule drug conjugate (SMDC) engineered to bind an αvß3 integrin adhesion molecule that is abundantly expressed in advanced metastatic solid tumors. αvß3 is a hallmark of aggressive cancers and poor prognosis. We also remain on-target to file INDs in the second half of 2023 for our two initial antibody drug conjugates (ADCs), VIP943, an anti-IL3RA-KSPi, and VIP924, an anti-CXCR5-KSPi, both with a CellTrapper moiety. From a financial perspective, our current cash resources position us to continue to pursue our upcoming regulatory and clinical milestones," concluded Dr. Hamdy.

RECENT CORPORATE HIGHLIGHTS

VIP152

Abstract accepted for poster presentation at the upcoming European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting, titled "VIP152 is a novel CDK9 inhibitor with improved selectivity, target modulation, and cardiac safety in patients with lymphoma."

Presenting author: Melanie Frigault, PhD

Abstract number: P1269

Session date and time: Friday, June 10, 2022; 16:30-17:45 CEST

Presented poster, "VIP152, a selective CDK9 inhibitor, demonstrates sensitivity in gynecologic cell lines that are cisplatin sensitive or resistant and delivers in vivo antitumor efficacy," at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2022.

Bioconjugation Platform

Invited talk on the Company’s modular assets that can build innovative and high-potency ADCs titled, "ADCs with KSP-Inhibitor Payloads and a Tailored Design of Linker and Metabolite Profile," at the Festival of Biologics meeting March 2022.

Continue to advance our next generation modular bioconjugation platform, comprised of a first-in-class SMDC for solid tumors (VIP236) and two best-in-class preclinical-stage assets for hematologic malignancies (VIP943 and VIP924).

VIP236 consists of an αvß3 integrin binder and a linker that is cleavable by neutrophil elastase. The payload is an optimized camptothecin derivative designed for high cellular permeability and low efflux. IND filing in solid tumors expected in 2H 2022.

VIP943 (anti-IL3RA) and VIP924 (anti-CXCR5) are two antibodies linked to a KSP inhibitor – a novel payload class in ADCs. These ADCs also have a stable linker specifically cleaved by legumain, a tumor associated protease. The payload (i.e., KSP inhibitor) is modified to be trapped in the cell by the CellTrapper moiety. Manufacturing is underway and IND filings expected in 2H 2023.

FIRST QUARTER FINANCIAL RESULTS

Vincerx Pharma had $96.5 million in cash as of March 31, 2022, as compared to $111.5 million as of December 31, 2021. Based on its current business plans and assumptions, Vincerx believes its available cash will be sufficient to meet its operating requirements through 2023.

Research and development (R&D) expenses for the quarter ended March 31, 2022 were $16.0 million, as compared to $4.8 million for the same period in 2021. The increase was primarily related to increases in manufacturing services of approximately $4.0 million, including the initiation of manufacturing associated with our ADC program, new employee salaries of approximately $2.5 million, third party research and preclinical work of approximately $2.0 million, clinical services of approximately $1.4 million and stock-based compensation of approximately $0.8 million.

General and administrative (G&A) expenses for the quarter ended March 31, 2022 were $5.6 million, as compared to $4.8 million for the same period in 2021. The increase was primarily related to new employee salaries and increases in legal (patent protection and filings), accounting and other professional services in support of our operations as a public company.

For the quarter ended March 31, 2022, Vincerx reported a net loss of $16.4 million, or $0.79 per share. For the quarter ended March 31, 2021, Vincerx reported a net loss of $6.3 million, or $0.46 per share.

On May 12, 2022 Day One Biopharmaceuticals (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported financial results for the first quarter of 2022 and highlighted recent corporate achievements (Press release, Day One, MAY 12, 2022, View Source [SID1234614363]).

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"We continue to make excellent progress across our clinical programs and look forward to reporting initial data from our pivotal Phase 2 FIREFLY-1 trial in relapsed pLGG next month," said Jeremy Bender, Ph.D., chief executive officer of Day One. "These initial data will provide preliminary insights into the potential of tovorafenib to transform patient care for the most common childhood brain cancer, which currently has no approved therapies. Beyond FIREFLY-1, we are preparing to initiate a pivotal Phase 3 study, FIREFLY-2, for the first-line treatment of pLGG patients and recently initiated the combination portion of our Phase 2 FIRELIGHT-1 study with tovorafenib and our investigational oral MEK inhibitor, pimasertib. As our clinical programs advance, we continue to accelerate planning for our first potential regulatory submission for tovorafenib in 2023 and execute on our business strategy to make an impact for patients of all ages with life threatening diseases."

Program Highlights

Initial data from FIREFLY-1, a pivotal Phase 2 clinical trial of tovorafenib in relapsed pLGG, is expected in June 2022.

Day One anticipates releasing topline results from the fully-enrolled pivotal study in the first quarter of 2023. Pending positive results from FIREFLY-1, Day One anticipates filing a new drug application (NDA) with the U.S. Food and Drug Administration (FDA) in 2023.

Day One has expanded the FIREFLY-1 study to include two additional study arms:

An expanded access arm that enables treatment for eligible patients once the primary cohort has completed enrollment; and
An advanced solid tumor arm to evaluate the preliminary efficacy of tovorafenib in patients aged 6 months to 25 years with a relapsed or progressive extracranial solid tumors with activating RAF fusion.

Day One plans to initiate a pivotal Phase 3 clinical trial (FIREFLY-2) evaluating tovorafenib as a front-line therapy in pLGG in the second quarter of 2022.

Day One is enrolling patients in the Phase 2 FIRELIGHT-1 trial evaluating tovorafenib monotherapy in adults with recurrent, progressive, or refractory solid tumors harboring MAPK pathway aberrations. Day One recently expanded FIRELIGHT-1 to include a Phase 1b/2 portion to evaluate tovorafenib in combination with pimasertib, Day One’s investigational MEK inhibitor.
First Quarter 2022 Financial Highlights

Cash Position: Cash and cash equivalents totaled $262.7 million on March 31, 2022. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into 2024.

R&D Expenses: Research and development expenses were $15.0 million for the first quarter of 2022 compared to $12.6 million for the first quarter of 2021. The increase was primarily due to additional employee compensation costs, clinical trial and product development expenses which were offset by a decrease in milestone payments for licensing agreements.

G&A Expenses: General and administrative expenses were $12.7 million for the first quarter of 2022 compared to $3.5 million for the first quarter of 2021. The increase was primarily due to additional employee compensation costs, initial commercial buildout, and professional expenses to support company growth.

Net Loss: Net loss totaled $27.7 million for the first quarter of 2022 compared to $16.1 million for the first quarter of 2021 with non-cash stock compensation expense of $6.2 million and $0.5 million for the first quarters of 2022 and 2021, respectively.
Upcoming Events

2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting
A trial-in-progress poster on Day One’s FIREFLY-1 pivotal study, abstract number TPS10062, will be presented at the ASCO (Free ASCO Whitepaper) Annual Meeting on Monday, June 6, 2022, from 8 to 11 a.m. CST.

The 20th International Symposium on Pediatric Neuro-Oncology (ISPNO) Annual Meeting
An educational exhibit on Day One’s pipeline will be displayed at Booth #F.05, at ISPNO’s Annual Meeting, which is being held June 12-15, 2022.
About Tovorafenib
Tovorafenib is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which is being investigated in primary brain tumors or brain metastases of solid tumors. Tovorafenib has been studied in over 250 patients to date. Currently tovorafenib is under evaluation in a pivotal Phase 2 clinical trial (FIREFLY-1) among pediatric, adolescent and young adult patients with pediatric low-grade glioma (pLGG), which is an area of considerable unmet need with no approved therapies. Tovorafenib is also being evaluated alone or as a combination therapy for adolescent and adult patient populations with recurrent or progressive solid tumors with MAPK pathway aberrations (FIRELIGHT-1). Tovorafenib has been granted Breakthrough Therapy and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma, and from the European Commission (EC) for the treatment of glioma.

About Pimasertib
Pimasertib is an investigational, oral, highly selective, small molecule inhibitor of mitogen‐activated protein kinases 1 and 2 (MEK-1/-2) within the MAPK signaling pathway. Pimasertib has been dosed in over 850 patients to date for various tumor types. Preclinical data indicates that the combination of a MEK inhibitor, such as pimasertib, and a type II RAF inhibitor, such as tovorafenib, has synergistic anti-tumor activity.

Day One is conducting a Phase 1b/2 study (FIRELIGHT-1) to evaluate the safety, tolerability, and preliminary efficacy of combining pimasertib with tovorafenib in adolescent and adult patients (≥12 years of age) with recurrent, progressive, or refractory solid tumors with MAPK pathway aberrations.