On August 12, 2022 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported financial results for the second quarter ended June 30, 2022, and recent corporate highlights (Press release, Checkpoint Therapeutics, AUG 12, 2022, View Source [SID1234618250]).

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "Over the past few months, we have made substantial progress towards the regulatory submission for, and potential approval of, cosibelimab for the treatment of cutaneous squamous cell carcinoma ("cSCC"). Simultaneously, we continue to generate compelling clinical data with cosibelimab, most recently announcing positive interim results from our registration-enabling trial in locally advanced cSCC, with over half of the patients achieving a confirmed objective response. These impressive data follow the positive results reported in metastatic cSCC earlier this year. Importantly, we successfully completed our pre-BLA meetings with the FDA in July, reaching agreement on all key aspects discussed with regard to the upcoming BLA submission, including the inclusion of both the locally advanced and metastatic indications. We remain on target to submit the cosibelimab BLA in late 2022."

Mr. Oliviero, continued, "Also during the quarter, we continued to advance our commercial planning to enable a successful launch upon possible approval next year, and believe our price disruptive strategy could generate substantial market share for cosibelimab in the U.S. In parallel, we continue to engage in active discussions with multiple pharmaceutical companies with the goal of expanding access to cosibelimab to patients outside of the U.S."

Recent Corporate Highlights:

In May 2022, Checkpoint announced that it received Pediatric Investigation Plan product-specific waivers from the European Medicines Agency and the U.K. Medicines & Healthcare products Regulatory Agency for cosibelimab in cSCC. The waivers remove the requirement to conduct pediatric clinical studies to support cosibelimab marketing authorization applications in Europe.
In June 2022, the top-line results of the pivotal trial of cosibelimab in metastatic cSCC were presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Data highlights presented include a confirmed objective response rate ("ORR") by independent central review in the modified intent to treat population of 48.7% (95% CI, 37.0-60.4) and 13.2% of patients achieved a complete response in target lesions. Cosibelimab was generally well tolerated with no unexpected safety signals.
Also in June 2022, Checkpoint announced positive interim results from its pivotal trial of cosibelimab in locally advanced cSCC. As of the March 2022 data cutoff, the confirmed ORR by independent central review in 31 patients was 54.8% (95% CI: 36.0, 72.7), substantially exceeding a clinically meaningful lower bound of the 95% two-sided confidence interval.
In July 2022, Checkpoint successfully completed two pre-BLA meetings with the FDA (chemistry, manufacturing and controls [CMC] and clinical/non-clinical). Based upon favorable interactions with the agency, the planned BLA submission will include both the metastatic and locally advanced indications. Checkpoint also reached agreement with the FDA on all key aspects discussed with regard to the content of the upcoming BLA submission.
Checkpoint is discontinuing its CONTERNO study, a global, randomized Phase 3 trial of cosibelimab in combination with pemetrexed and platinum chemotherapy for the first-line treatment of patients with non-squamous non-small cell lung cancer, due to the substantially longer enrollment period expected as a result of the ongoing conflict in Ukraine. The Company expects that the study will be wound down and closed over the coming months and all costs associated with the study to cease by the end of the fourth quarter.
Financial Results:

Cash Position: As of June 30, 2022, Checkpoint’s cash and cash equivalents totaled $30.9 million, compared to $41.5 million at March 31, 2022 and $54.7 million at December 31, 2021, a decrease of $10.6 million for the quarter and a decrease of $23.8 million for the first half of 2022.
R&D Expenses: Research and development expenses for the second quarter of 2022 were $12.1 million, compared to $7.2 million for the second quarter of 2021, an increase of $4.9 million. Research and development expenses for the second quarters of 2022 and 2021 each included $0.2 million of non-cash stock expenses.
G&A Expenses: General and administrative expenses for the second quarters of 2022 and 2021 each were $2.1 million. General and administrative expenses for the second quarter of 2022 included $0.5 million of non-cash stock expenses, compared to $0.9 million for the second quarter of 2021.
Net Loss: Net loss attributable to common stockholders for the second quarter of 2022 was $14.1 million, or $0.16 per share, compared to a net loss of $9.1 million, or $0.12 per share, in the second quarter of 2021. Net loss for the second quarter of 2022 included $0.7 million of non-cash stock expenses, compared to $1.0 million for the second quarter of 2021.

On August 12, 2022 Privo Technologies, Inc. ("Privo") reported that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to Privo’s PRV platform delivery of cisplatin for the treatment of carcinoma in situ (CIS) of the anterior 2/3 of the oral cavity (Press release, Privo Technologies, AUG 12, 2022, View Source;utm_medium=rss&utm_campaign=privo-technologies-inc-receives-us-fda-orphan-drug-designation [SID1234618249]). This represents Privo’s third ODD for a rare disease following their approval in oral cavity cancers and anal cancers. Privo Technologies is a clinical stage biopharmaceutical company that has designed and developed a nanoengineered drug delivery platform to safely deliver highly potent and toxic APIs locoregionally, led by founder and CEO Manijeh Goldberg, PhD.

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"Achieving this important regulatory milestone means we are one step closer to bringing new treatment options to patients diagnosed with oral CIS. The standard of care currently is surgery, resulting in severe disfigurement and poor quality of life. Our novel transmucosal delivery system with embedded cisplatin loaded nanoparticles, PRV111, allows for patients to be treated with topical chemotherapy providing an alternative to surgery that effectively treats cancer while maintaining the form and function of the oral cavity," said Dr. Goldberg.

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers that includes assistance in the drug development process, tax credits for clinical costs, exemption from FDA PDUFA fees, and seven years of post-approval exclusivity.

On August 12, 2022 Seagen Inc. (Nasdaq: SGEN) reported a statement regarding the outcome of the arbitration with Daiichi Sankyo Co. Ltd. ("Daiichi Sankyo") relating to the parties’ 2008 collaboration agreement for the use of Seagen’s antibody-drug conjugate (ADC) technology (Press release, Seagen, AUG 12, 2022, https://investor.seagen.com/press-releases/news-details/2022/Seagen-Statement-on-Outcome-of-Daiichi-Sankyo-Arbitration/default.aspx [SID1234618248]). The arbitrator ruled in favor of Daiichi Sankyo, citing statute of limitations and disagreement with Seagen on the interpretation of the contract.

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"While we are disappointed with the arbitration decision, it was important for us to pursue this legal action," said Roger Dansey, M.D., interim Chief Executive Officer and Chief Medical Officer, Seagen. "This does not impact our existing business. Looking forward, we are well-positioned to drive continued innovation and growth with four commercial products and a deep and diverse pipeline of promising programs. Seagen remains focused on developing innovative medicines that make a meaningful difference in the lives of cancer patients."

On August 12, 2022 Ipsen (Euronext: IPN; ADR: IPSEY) reported the closing of the definitive merger agreement under which Ipsen has acquired Epizyme, Inc. (Epizyme) (Press release, Ipsen, AUG 12, 2022, View Source [SID1234618247]). Pursuant to the transaction, Ipsen acquires all outstanding shares of Epizyme for $1.45 per share plus a contingent value right (CVR) of $1.00 per share. Epizyme now operates as ‘an Ipsen company’ at deal close.

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As part of the transaction, Ipsen acquires Epizyme’s lead medicine, Tazverik (tazemetostat), a first-in-class, chemotherapy-free EZH2a inhibitor, which was granted Accelerated Approval by the U.S. Food and Drug Administration (FDA) in 2020. It is currently indicated for adults with relapsed or refractory follicular lymphoma (FL) whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least two prior systemic therapies, and for adult patients with relapsed or refractory follicular lymphoma who have no satisfactory alternative treatment options, as well as for adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.1

Ipsen also acquires Epizyme’s first-in-class, oral SETD2 inhibitor development candidate, EZM0414, which was granted FDA Fast Track status in 2021 and is currently under evaluation in a recently initiated Phase I/Ib trial in adult patients with relapsed or refractory multiple myeloma and diffuse large B-cell lymphoma, as well as a portfolio of preclinical programs focusing on epigenetic targets.

"Throughout the pre-close phase of planning, we have continued to be impressed by the potential of Tazverik, as well as the rest of the pipeline. Now that the deal is closed, we are excited to be working closely with our Epizyme colleagues to leverage Ipsen’s established infrastructure so that these medicines may reach more patients. Additionally, through this transaction Ipsen gains scientific expertise and we look forward to integrating the two teams which share the goal of delivering innovative treatment options to underserved patients," said David Loew, Chief Executive Officer of Ipsen.

About Tazverik (tazemetostat)

Tazverik is a methyltransferase inhibitor indicated for the treatment of:

Adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection.
Adult patients with relapsed or refractory follicular lymphoma whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least two prior systemic therapies.
Adult patients with relapsed or refractory follicular lymphoma who have no satisfactory alternative treatment options.
These indications are approved under accelerated approval based on overall response rate and duration of response. Post marketing studies are required to confirm the anticipated clinical benefit and retain the labeled Accelerated Approval indications.

The most common (≥20%) adverse reactions in patients with epithelioid sarcoma are pain, fatigue, nausea, decreased appetite, vomiting and constipation. The most common (≥20%) adverse reactions in patients with follicular lymphoma are fatigue, upper respiratory tract infection, musculoskeletal pain, nausea and abdominal pain.

View the U.S. Full Prescribing Information here: View Source

About EZM0414

EZM0414 is a potent selective, oral, small molecule, investigational drug agent that inhibits the histone methyltransferase, SETD2, which plays a role in oncogenesis. SETD2 methylates histone as well as non-histone proteins, and this activity is involved in several key biological processes including transcriptional regulation, RNA splicing, and DNA damage repair. Based on the preclinical data on SETD2 inhibition by EZM0414 in multiple settings, including high risk t(4;14) multiple myeloma (MM) and in other B-cell malignancies such as diffuse large B-cell lymphoma (DLBCL), the Company is conducting SET-101, a Phase 1/1b study of EZM0414, for the treatment of adult patients with relapsed or refractory MM and DLBCL.

About follicular lymphoma2, 3

Follicular lymphoma is a type of non-Hodgkin lymphoma (NHL) which is a cancer of the lymphatic system. Follicular lymphoma develops when the body makes abnormal B lymphocytes. These lymphocytes are a type of white blood cell that normally helps fight infections. When a patient has a lymphoma, the abnormal lymphocytes build up in the lymph nodes or other body organs. Follicular lymphoma is generally slow growing. Each year, 15-20,000 people in the U.S. are diagnosed with follicular lymphoma. Most affected individuals are diagnosed with advanced disease.

About epithelioid sarcoma4

Epithelioid sarcoma is a rare, slow-growing type of soft tissue cancer. Most cases begin in the soft tissue under the skin of a finger, hand, forearm, lower leg or foot, though it can start in other areas of the body. Typically, epithelioid sarcoma starts as a small firm growth or lump that is painless. It usually starts out as a single growth, but multiple growths may occur by the time a person seeks medical help. Sometimes this sarcoma appears as ulcers that don’t heal, looking like open wounds over the growths. It is estimated that 13,040 individuals received a diagnosis of soft tissue sarcomas in the U.S. in 2018 with a corresponding 5,150 deaths.5

About diffuse large B-cell lymphoma6

Diffuse large B cell lymphoma (DLBCL) is a type of NHL. NHL is a cancer of the lymphatic system. It develops when the body makes abnormal B lymphocytes. These lymphocytes are a type of white blood cell that normally help to fight infections. When a patient has a lymphoma, the abnormal lymphocytes build up in lymph nodes or other body organs. DLBCL grows quickly and treatment starts soon after diagnosis. DLBCL is the most common type of NHL in the U.S. and worldwide, accounting for about 22 percent of newly diagnosed cases of B-cell NHL in the U.S. More than 18,000 people in the U.S. are diagnosed with DLBCL each year.7

About multiple myeloma8

Multiple myeloma is a rare form of cancer characterized by excessive production (proliferation) and improper function of certain cells (plasma cells) found in the bone marrow. Excessive plasma cells may eventually mass together to form a tumor or tumors in various sites of the body, especially the bone marrow. When multiple tumors are present or the bone marrow has greater than 10% plasma cells, the term multiple myeloma is used. In 2019, over 32,000 individuals in the U.S. were diagnosed with this disease. It is believed that approximately 100,000 Americans currently have the disease.

On August 12, 2022 Processa Pharmaceuticals, Inc. (Nasdaq: PCSA) ("Processa" or the "Company"), a clinical stage company developing drugs for patients who have unmet medical conditions and/or require better treatment options to improve a patient’s survival and/or quality of life, reported financial results for the quarter ended June 30, 2022, and provided an update on its clinical programs (Press release, Processa Pharmaceuticals, AUG 12, 2022, View Source [SID1234618246]).

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Dr. David Young, President and CEO of Processa, commented, "Our efforts to enhance enrollment through adding new sites, extensive marketing campaigns and working with our CRO partners are showing results:

We expect to close out enrollment for PCS12852 for Gastroparesis within the next month and present top-line data from the trial before the end of the year.
We now have a sufficient number of patients in the screening queue for PCS6422 to complete our interim cohorts which we believe will provide valuable insights on de novo formation of DPD. We anticipate we will determine the maximum tolerated dose by early 2023.
Patients are beginning to show a willingness to travel, and we are seeing increased patient activity in our PCS499 ulcerative necrobiosis lipoidica (uNL) trial. We are optimistic that we will enroll our interim analysis cohort before the end of the year.
As part of our efforts to increase enrollment on our PCS499 trial, we recently launched a website to increase awareness of uNL and to inform patients of the ongoing Phase 2B Study of PCS499.
Advancing these 3 drugs in their respective clinical trial allows us to obtain the clinical data to better define each pivotal trial as well as provide us with more insight into how FDA will review each of these products when we submit the New Drug Applications to FDA."

Financial Results for the Six Months Ended June 30, 2022

Our cash balance on June 30, 2022, was $12.1 million, which should be sufficient to complete our three on-going clinical trials and fund our operations into the third quarter of 2023. During the six months ended June 30, 2022, we spent $4.1 million in cash for these three clinical trials and our operations. This is significantly less than our GAAP net loss of $8.4 million due to the effect of non-cash items like amortization and stock-based compensation, and the application of amounts we had prepaid to our CROs last year.

Our net loss for the six months ending on June 30, 2022, was $8.4 million or $0.53 per share compared to a net loss of $5.3 million, or $0.35 per share for the same period of 2021. The increase in our net loss relates primarily to increased clinical trial costs we incurred in our three ongoing trials. For the six months ended June 30, 2022, we incurred $5.2 million in research and development costs, an increase of $2.1 million when compared to the same period of 2021. We anticipate clinical trial costs will continue to increase for the rest of the year as our trials continue to progress and we fund development activities for the other drugs in our pipeline.

During the six months ending June 30, 2022, our general and administrative expenses totaled $3.2 million compared to $2.1 million for the same period in 2021. The increase related primarily to increases in non-cash or stock-based compensation costs, along with other operating and consulting costs. We allocated $2.8 million of non-cash compensation costs between our R&D and G&A costs, with the majority recorded as G&A.

Our net cash used in operating activities during the six months ended June 30, 2022, decreased by $300,000 to $4.1 million, compared to $4.4 million for the same period in 2021. While we experienced increased GAAP costs related to our clinical trials and operations, we continued to make use of equity incentives to compensate our executive and development team, thereby reducing our cash outflow, and we were able to apply previously made advanced payments to our CROs against current trial costs.

As of June 30, 2022, we had 15.8 million common shares outstanding.

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