On May 6, 2020 for patients with solid tumor cancers, reported financial results for the first quarter ended March 31, 2020, and recent corporate highlights (Press release, Checkpoint Therapeutics, MAY 6, 2020, View Source [SID1234561602]).

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James F. Oliviero, President and Chief Executive Officer of Checkpoint, said, "In the first quarter of 2020, we continued to make progress with the development of cosibelimab, our lead antibody product candidate that is poised to be a potentially differentiated treatment from currently marketed PD-1 and PD-L1 antibodies. As announced in January, the U.S. Food and Drug Administration ("FDA") confirmed the registration submission pathway for cosibelimab in its initial indication of metastatic cutaneous squamous cell carcinoma ("CSCC") based on the ongoing multicenter Phase 1 clinical trial. We look forward to reporting additional interim data from this clinical trial later this year. Additionally, we recently announced that the U.S. Patent and Trademark Office issued a composition of matter patent for cosibelimab, which provides broad, foundational composition of matter protection for our antibody through at least May 2038. With these developments, we look forward to continuing to enroll CSCC patients in order to support our first marketing application submission based on this ongoing clinical trial."

Recent Corporate Highlights:

·In January 2020, Checkpoint announced confirmation of the registration path for cosibelimab in metastatic CSCC. FDA feedback supports the Company’s plan to submit a Biologics License Application ("BLA") based on data from the ongoing Phase 1 trial. Over one-third of enrollment is complete in the cohort of patients with metastatic CSCC.

·In April 2020, Checkpoint announced that the U.S. Patent and Trademark Office issued a composition of matter patent for cosibelimab. U.S. Patent No. 10,590,199 specifically covers the antibody, cosibelimab, or a fragment thereof, providing protection through at least May 2038, exclusive of any additional patent-term extensions that might become available.

Financial Results:

·Cash Position: As of March 31, 2020, Checkpoint’s cash and cash equivalents totaled $21.5 million, compared to $26.1 million as of December 31, 2019, a decrease of $4.6 million.

·Revenue: Revenue for the first quarter of 2020 was $1.0 million, compared to $0.4 million for the first quarter of 2019, an increase of $0.6 million.

·R&D Expenses: Research and development expenses for the first quarter of 2020 were $2.6 million, compared to $4.6 million for the first quarter of 2019, a decrease of $2.0 million. Research and development expenses for the first quarter of 2020 included $0.1 million of non-cash stock expenses, compared to $0.2 million in the first quarter of 2019.

·G&A Expenses: General and administrative expenses for the first quarter of 2020 were $1.7 million, essentially flat as compared to the first quarter of 2019. General and administrative expenses for the first quarter of 2020 included $0.5 million of non-cash stock expenses, compared to $0.6 million for the first quarter of 2019.

·Net Loss: Net loss attributable to common stockholders for the first quarter of 2020 was $3.3 million, or $0.06 per share, compared to a net loss of $5.9 million, or $0.18 per share, in the first quarter of 2019.

On May 6, 2020 ERYTECH Pharma (Nasdaq & Euronext: ERYP), a clinical-stage biopharmaceutical company developing innovative therapies by encapsulating therapeutic drug substances inside red blood cells, reported a business and financial update (Press release, ERYtech Pharma, MAY 6, 2020, View Source [SID1234557381]).

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"Our focus during the past several weeks of the COVID-19 pandemic has been to continue our clinical operations and preserving study integrity while ensuring the health of our employees, the patients and the medical professionals involved in our clinical programs." said Gil Beyen, CEO of ERYTECH Pharma. "We are succeeding in ensuring patients’ continued access to treatment and appropriate follow-up, and are continuing to enroll new patients in the trials, be it at a slower pace than before the pandemic. With more than 75% of patients enrolled in the TRYbeCA-1 Phase 3 trial and based on the event rate observed thus far, we expect to report the results of the interim superiority analysis around year-end of 2020 and the final result in the second half of 2021. The FDA’s Fast Track designation for eryapase underscores the high unmet medical need eryaspase is addressing."

Business Highlights

TRYbeCA-1, the pivotal Phase 3 clinical trial evaluating ERYTECH’s lead product candidate, eryaspase, in second-line metastatic pancreatic cancer, has randomized more than 75% of the approximately 500 patients to be enrolled in the trial. In March 2020, the independent data monitoring committee (IDMC) reviewed the safety data of the first 320 patients enrolled and treated in the trial. In line with the two earlier safety reviews, no safety issues were identified and the IDMC recommended to continue the trial as planned. The Company has put measures in place to facilitate trial conduct during the COVID pandemic and is expecting to complete enrollment in the fourth quarter of 2020. The interim superiority analysis, to be conducted by the IDMC when two-thirds of the events have occurred, is currently expected to take place around year-end 2020. Since the interim analysis will not include a test for futility, there will be two possible outcomes: (1) the trial will either continue toward a final analysis, expected in the second half of 2021, or (2) will be stopped for superiority; if the primary endpoint is met by demonstrating a significant improvement in overall survival (OS).

A Phase 1 investigator-sponsored trial evaluating the safety of eryaspase in combination with FOLFIRINOX as a first-line treatment for metastatic pancreatic cancer, is being readied for launch. Georgetown Lombardi Comprehensive Cancer Center, the sponsor of the trial, received the clearance of their Investigational New Drug application (IND) from the U.S. Food and Drug Administration. Enrollment of the first patient in the trial is expected in the second half of 2020.

TRYbeCA-2, the Company’s randomized, open-label Phase 2 clinical trial in first-line triple-negative breast cancer (TNBC), is enrolling patients in three countries in Europe (Spain, Belgium, and Hungary). Target enrollment is approximately 64 patients and the primary endpoint is objective response rate. Results of the trial are expected in 2021.

A Phase 2 investigator-sponsored trial sponsored by the Nordic Organization of Pediatric Hematology and Oncology (NOPHO), is approaching the enrollment target of 50 patients to be treated in the trial. The trial is evaluating the safety and efficacy of eryaspase in patients with acute lymphoblastic leukemia (ALL) who developed hypersensitivity to pegylated asparaginase at 22 clinical sites in the Nordic and Baltic countries of Europe. The Company expects that an interim update on this trial will be available in the second quarter and final results by the end of this year.

Update on Q1 2020 Cash position

As of March 31, 2020, ERYTECH had cash and cash equivalents totaling €58.6 million (approximately $64.6 million), compared with €73.2 million on December 31, 2019. The €14.6 million decrease in cash position in the first quarter 2020 was the result of a €15.3 million net cash utilization and was mostly comprised of a €16.7 million net cash utilization in operating activities, €1.1 million used for investing activities and €2.4 million generated in financing activities, while the variation in the period of the U.S. dollar against the euro led to a €0.7 million favorable currency exchange impact.

The €14.6 million decrease in cash position in the first quarter of 2020 was in line with the Company’s operating plan. While closely monitoring the budget impact of the COVID-19 pandemic on its operations, the Company confirms its earlier guidance on sufficient cash position to fund operations into the first quarter of 2021.

Due to the COVID-19-related challenges in terms of compiling and auditing full financial information, the P&L highlights for the first quarter 2020 will be provided at a later time.

Key News Flow and Milestones Expected Over the Next 12 Months

Interim (superiority) analysis in TRYbeCA-1, the Phase 3 clinical trial in second-line metastatic pancreatic cancer (YE 2020)

Complete enrollment and interim update on Phase 2 investigator-sponsored NOPHO trial in second-line acute lymphoblastic leukemia (Q2 2020); final results (Q4 2020)

Initiation of a Phase 1 investigator-sponsored trial in first-line metastatic pancreatic cancer (2H 2020)

Conference Call Details

ERYTECH management will hold a conference call and webcast on Thursday May 7, 2020 at 02:30pm CEST / 08:30am ET on the business and financial highlights for the quarter ended March 31, 2020. Gil Beyen, CEO, Eric Soyer, CFO/COO, and Iman El-Hariry, CMO, will deliver a brief presentation, followed by a Q&A session.

The call is accessible via the below teleconferencing numbers, followed by the Conference ID#: 9688486#

The webcast can be followed live online via the link: View Source

An archived replay of the call will be available for 7 days by dialing + 1 855 859 2056, Conference ID: 9688486#. An archive of the webcast will be available on ERYTECH’s website, under the "Investors" section at investors.erytech.com

Financial Calendar

Business Update and Financial Highlights for the 2nd Quarter of 2020: September 21, 2020 (after U.S. market close), followed by a conference call and webcast on September 22, 2020 (2:30pm CET/8:30am ET)

Business Update and Financial Highlights for the 3rd Quarter of 2020: November 5, 2020 (after U.S. market close), followed by a conference call and webcast on November 6, 2020 (2:30pm CET/8:30am ET)

About TRYbeCA-1

TRYbeCA-1 is a randomized, controlled Phase 3 clinical trial evaluating eryaspase in second-line metastatic pancreatic cancer. The trial is planned to enroll approximately 500 patients at approximately 100 clinical sites in Europe and the United States. Eligible patients are randomized 1-to-1 to receive eryaspase in combination with standard chemotherapy (gemcitabine/nab-paclitaxel or an irinotecan-based regimen) or chemotherapy alone. The primary endpoint of TRYbeCA-1 is overall survival. An interim superiority analysis will be conducted when approximately two-thirds of the events will have occurred.

On May 6, 2020 Bonnie J Addario Lung Cancer Foundation reported that the Food and Drug Administration granted accelerated approval to capmatinib (TABRECTA, Novartis) for adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA-approved test (Press release, Bonnie J Addario Lung Cancer Foundation, MAY 6, 2020, View Source [SID1234557257]).

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Today, FDA also approved the FoundationOne CDx assay (Foundation Medicine, Inc.) as a companion diagnostic for capmatinib.

Efficacy was demonstrated in the GEOMETRY mono-1 trial (NCT02414139), a multicenter, non-randomized, open-label, multicohort study enrolling 97 patients with metastatic NSCLC with confirmed MET exon 14 skipping. Patients received capmatinib 400 mg orally twice daily until disease progression or unacceptable toxicity.

The main efficacy outcome measures were overall response rate (ORR) determined by a blinded independent review committee using RECIST 1.1 and response duration. Among the 28 treatment-naïve patients, the ORR was 68% (95% CI: 48, 84) with a response duration of 12.6 months (95% CI: 5.5, 25.3). Among the 69 previously treated patients, the ORR was 41% (95% CI: 29, 53) with a response duration of 9.7 months (95% CI: 5.5, 13.0).

The most common adverse reactions (≥ 20% of patients) were peripheral edema, nausea, fatigue, vomiting, dyspnea, and decreased appetite. Capmatinib can also cause interstitial lung disease, hepatotoxicity, photosensitivity, and embryo-fetal toxicity. Based on a clear positive signal for phototoxicity in early laboratory studies in cells, patients may be more sensitive to sunlight and should be advised to take precautions to cover their skin, use sunscreen, and not tan while taking capmatinib.

The recommended capmatinib dose is 400 mg orally twice daily with or without food.

View full prescribing information for TABRECTA.

This indication is approved under accelerated approval based on overall response rate and response duration. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate FDA’s assessment. The FDA approved this application 3 months ahead of the FDA goal date.

FDA granted capmatinib orphan drug and breakthrough therapy designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

On May 6, 2020 Kitov Pharma Ltd. ("Kitov") (NASDAQ/TASE: KTOV), a clinical-stage company advancing first-in-class therapies to overcome tumor immune evasion and drug resistance, reported that it has entered into definitive agreements with several institutional and accredited investors for the purchase and sale of 25,000,002 of the Company’s ordinary shares represented by American Depositary Shares (ADSs), at a purchase price of $0.40 per ADS, in a registered direct offering, for aggregate gross proceeds of approximately $10 million (Press release, Kitov Pharmaceuticals , MAY 6, 2020, View Source [SID1234557202]). Kitov has also agreed to issue to the investors unregistered warrants to purchase up to an aggregate of 25,000,002 ADSs. Each ADS represents one ordinary share, no par value, of Kitov. The offering is expected to close on or about May 8, 2020, subject to satisfaction of customary closing conditions.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The warrants will have an exercise price of $0.40 per ADS and will be exercisable at any time upon issuance and will expire five and one-half years from the date of issuance.

Kitov intends to use the net proceeds of this offering to fund the development of its oncology drug candidates, acquisition of new assets and for general working capital purposes.

The ADSs (but not the warrants or the ADSs underlying the warrants) are being offered by Kitov pursuant to a "shelf" registration statement on Form F-3 (File No. 333- 235327) previously filed with the U.S. Securities and Exchange Commission (the "SEC") on December 2, 2019 and declared effective by the SEC on December 13, 2019. The offering of the ADSs will be made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the ADSs being offered will be filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained, when available, on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (646) 975-6996 or e-mail at [email protected].

The warrants described above were offered in a private placement under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Act"), and Regulation D promulgated thereunder and, along with the ADSs underlying the warrants, have not been registered under the Act, or applicable state securities laws. Accordingly, the warrants and underlying ADSs may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Act and such applicable state securities laws.

On May 6, 2020 Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage biotechnology company focused on Alzheimer’s disease, reported recent business highlights and financial results for the first quarter ended March 31, 2020 (Press release, Pain Therapeutics, MAY 6, 2020, View Source [SID1234557201]). Net loss was $1.2 million, or $0.05 per share, compared to a net loss of $1.4 million, or $0.08 per share, for the same period in 2019. Net cash used in operations was $1.2 million during the first quarter of 2020. Net cash use in full-year 2020 is expected to be approximately $5 million. Cash and cash equivalents were $25.6 million as of March 31, 2020, with no debt.

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"Cassava Sciences had a productive quarter with its research programs in Alzheimer’s disease," said Remi Barbier, President & CEO. "As a result, we anticipate having top-line results for our Phase 2b study of PTI-125, our lead drug candidate for Alzheimer’s, earlier than mid-2020."

Phase 2b Study – Early data readout on effects of PTI-125 on tau protein anticipated
In March 2020, Cassava Sciences announced the completion of a double-blind, randomized, placebo-controlled study of PTI-125 in 64 patients with mild-to-moderate Alzheimer’s disease, 50-85 years of age, with 16 ≤ MMSE ≤ 26. Study participants received PTI-125 100 mg, 50 mg or matching placebo, twice-daily, for 28 continuous days. The primary efficacy endpoint is the effect of PTI-125 vs placebo on CSF levels of tau protein, and other biomarker assessments.

Open-label Study – Initiated in March, approximately 20% enrolled
In March 2020, Cassava Sciences announced the initiation of an open-label, multi-center study of PTI-125 at 100 mg twice-daily for 12 months. Every study participant receives drug treatment in an open-label design. This on-going study has a target enrollment of approximately 100 patients with mild-to-moderate Alzheimer’s disease. The study is approximately 20% enrolled.

Finance Update – $2.9 million of new NIH research grant awards announced in 2020
Cassava Sciences’ scientific programs continue to be supported by research grant awards from the National Institutes of Health (NIH), the nation’s foremost medical research agency. In April 2020, the Company announced it had been awarded a new $2,500,000 research grant from NIH. In March 2020, NIH awarded the Company supplemental research funding in the amount of $374,000. The NIH’s National Institute on Aging awarded the Company these research grant award following an in-depth, peer review of PTI-125. Peer review, one of the gold standards of science, is a process where independent, outside scientists evaluate the merits of new research.

Operations Update – No major disruptions to date
In these times of pandemic, Cassava Sciences’ top priorities are to protect the health, well-being, and safety of its employees and partners, while still focusing on the key drivers of its business. The company believes it remains on-track to achieve its major strategic objectives for 2020. The Company has not experienced major disruptions across its drug manufacturing operations or supply of materials. Its broad spectrum of technical consultants, scientific advisors and service providers continue to provide timely services. The Company has adapted flexible business practices, such as remote work arrangements and temporary travel restrictions, to insure it continues to operate safety and cautiously while meeting its public health responsibilities.

Cassava Sciences recognizes the on-going pandemic has created an unstable and uncertain situation in the national economy. The Company continues to closely monitor the latest information to make timely, informed business decisions and public disclosures regarding the potential impact of pandemic on its operations. However, the scope of this pandemic is unprecedented and its long-term impact on the Company’s operations and financial condition cannot be reasonably estimated at this time.

Financial Highlights for First Quarter 2020

At March 31, 2020, cash and cash equivalents were $25.6 million, compared to $23.1 million at December 31, 2019, with no debt.
Cash balance included $3.6 million in proceeds from exercise of warrants in the first quarter of 2020. Approximately 1.6 million warrants remain outstanding, each with an exercise price of $1.25 per share. All warrants expire February 2021.
Net cash used in operations during the quarter ended March 31, 2020 was $1.2 million, net of reimbursements received from NIH grant awards.

Net cash use for full year 2020 is expected to be approximately $5.0 million, consistent with previous financial guidance.
Research grant funding reimbursements of $1.3 million were received from NIH and recorded as a reduction in research and development (R&D) expenses. This compared to $0.8 million of NIH grant receipts received for the same period in 2019.
R&D expenses were $0.5 million. This compared to $0.6 million for the same period in 2019, representing a 5% decrease. While Phase 2 clinical program expenses were higher in Q1 2020, overall expense was reduced by greater NIH reimbursement.
General and administrative (G&A) expenses were $0.8 million. This compared to $0.9 million for the same period in 2019, representing a 11% decrease. The decrease was due primarily to lower stock-based compensation expense compared to 2019.
About PTI-125
Cassava Sciences’ lead therapeutic product candidate is for the treatment of Alzheimer’s disease. PTI-125 is a proprietary, small molecule (oral) drug that restores the normal shape and function of altered filamin A (FLNA), a scaffolding protein, in the brain. Altered FLNA in the brain disrupts the normal function of neurons, leading to Alzheimer’s pathology, neurodegeneration and neuroinflammation. The underlying science is published in peer-reviewed scientific journals, including Journal of Neuroscience, Neurobiology of Aging, Journal of Biological Chemistry and Journal of Prevention of Alzheimer’s Disease. The Company is also developing an investigational diagnostic, called SavaDx, to detect Alzheimer’s disease with a simple blood test.

About Alzheimer’s Disease
Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking skills. Currently, there are no drug therapies to halt Alzheimer’s disease, much less reverse its course. In the U.S. alone, approximately 5.8 million people are currently living with Alzheimer’s disease, and approximately 487,000 people age 65 or older developed Alzheimer’s in 2019.1 The number of people living with Alzheimer’s disease is expected to grow dramatically in the years ahead, resulting in a growing social and economic burden.2