On June 16, 2022 Plus Therapeutics, Inc. (Nasdaq: PSTV) (the "Company"), a clinical-stage pharmaceutical company developing innovative, targeted radiotherapeutics for rare and difficult-to-treat cancers, reported the completion of enrollment in the first cohort of the ReSPECT-LM Phase 1/2a dose escalation clinical trial of Rhenium-186 NanoLiposome (186RNL) for the treatment of leptomeningeal metastases (LM) (Press release, Cytori Therapeutics, JUN 16, 2022, View Source [SID1234616040]).

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"We are pleased with initial response to the ReSPECT-LM trial by patients and physicians and the very early promising drug performance," said Norman LaFrance, M.D., Chief Medical Officer and SVP of Plus Therapeutics. "Patients seemed to tolerate 186RNL infusions and the drug circulated completely and rapidly throughout the cebrebrospinal fluid space. We intend to provide enrollment and complete clinical updates in the third quarter of 2022."

The investigational drug, 186RNL, is a proprietary nanoscale compound with a unique chelated radioisotope that is administered locally as a single dose via a conventional Ommaya reservoir. Rhenium-186 is a dual energy emitter (beta and gamma) with a short average path length for high precision, low dose rate that is safer for normal tissues, and high radiation density that overwhelms innate DNA repair mechanisms. The dual energy emission allows real time evaluation and monitoring of the 186RNL administration.

The ReSPECT-LM trial (NCT05034497) is a multicenter, sequential cohort, open-label, single dose, dose escalation Phase 1/2a study using a modified Fibonacci 3+3 study design. It will evaluate the maximum tolerated dose (MTD), maximum feasible dose (MFD), safety and efficacy of a single administration of 186RNL via intraventricular catheter for LM following standard surgical, radiation and/or chemotherapy treatment. The primary endpoints of the study are the incidence and severity of adverse events/serious adverse events and dose limiting toxicities. Secondary endpoints include overall response rate, duration of response, progression free survival and overall survival.

The ReSPECT-LM Phase 1/2a clinical trial follows preclinical studies in which tolerance to doses of 186RNL as high as 1,075 Gy was shown in animal models with LM with no observed significant toxicity. Treatment led to marked reduction in tumor burden in two animal models of LM.

In November 2021, the Company announced that the FDA granted Fast Track designation to 186RNL for the treatment of LM. Fast Track designation confers several benefits to the drug development program including 1) more frequent meetings with and written communication from FDA, 2) eligibility for Accelerated Approval and Priority Review, if relevant criteria are met, and 3) Rolling Review, which allows a drug company to submit completed sections of its New Drug Application (NDA) for review by FDA, rather than wait until every section of the NDA is completed before the entire application can be reviewed.

About Leptomeningeal Metastases (LM)

LM is a rare complication of cancer in which the disease spreads to the membranes (meninges) surrounding the brain and spinal cord. LM occurs in approximately 5% of people with cancer and is usually terminal with one-year and two-year survival of just 7% and 3% respectively. LM can originate from solid tumors, primary brain tumors, or hematological malignancies.

June 16, 2022 Akero Therapeutics, Inc. (Nasdaq: AKRO), a clinical-stage company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, reported two financing transactions (Press release, Akero Therapeutics, JUN 16, 2022, View Source [SID1234616039]). The first is a $25 million equity investment by Pfizer Inc. (NYSE: PFE) at $9.90 per share. The second is a term loan facility providing Akero with access to up to $100 million from Hercules Capital, Inc. (NYSE: HTGC), of which $10 million will be drawn at transaction close. Together with existing cash, proceeds will support Akero’s continued development of efruxifermin (EFX), a long-acting analog of fibroblast growth factor 21 (FGF21), including two ongoing Phase 2b clinical trials in patients with pre-cirrhotic and cirrhotic nonalcoholic steatohepatitis (NASH); manufacture of a drug product-device combination for use in Phase 3 clinical trials; and starting a Phase 3 clinical trial program. If the term loan is fully drawn, proceeds from these two transactions together with budget optimization efforts are expected to fund Akero’s current operating plan until the third quarter of 2024. This extends Akero’s previously announced cash guidance by a full year (from the third quarter of 2023 to the third quarter of 2024), two years beyond the anticipated readout of the HARMONY study in the third quarter of this year.

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"Pfizer has deep expertise and history in addressing health challenges that affect millions of patients around the world, including cardiometabolic diseases. We are honored and excited to have their confidence, collaboration and support," said Andrew Cheng, M.D. Ph.D., president and chief executive officer of Akero. "In addition, Hercules Capital has a rich history of investing in innovative biotechnology companies. We are very grateful for their significant partnership and support. With our existing cash on hand, we expect these two financings will extend our cash runway a full two years beyond our upcoming HARMONY readout, providing us the flexibility to optimize our capital structure to support the continued development of EFX."

Pfizer is investing in Akero through the Pfizer Breakthrough Growth Initiative (PBGI), which seeks to support biotechnology companies that share its commitment to delivering transformative therapies for patients in therapeutic areas consistent with Pfizer’s core areas of focus. Under the terms of the agreement, Akero has agreed to sell 2,525,252 shares to Pfizer at a price of $9.90 per share, for gross proceeds of $25 million. The shares of common stock were offered and sold to Pfizer in a registered direct offering conducted without an underwriter or placement agent. The offering is expected to close on or about June 17, 2022. Following the transaction, Pfizer will own approximately 6.7% of Akero’s outstanding common stock. As part of the transaction, Akero will establish a Scientific Advisory Board, with Pfizer appointing one member. Akero will maintain ownership and control of EFX, the rest of its pipeline, and Akero’s operations.

"EFX has quickly emerged as a promising potential NASH therapy, with a strong record of clinical trial data," said Jeff Pfefferkorn, Ph.D., Vice President of Discovery & Development, Internal Medicine Research Unit, Pfizer, who is expected to join Akero’s newly formed Scientific Advisory Board. "NASH is a priority therapeutic area for Pfizer due to the substantial global unmet medical need it represents, and we are excited to support Akero as it advances EFX towards a potential Phase 3 study."

The $100 million term loan facility is being provided by Hercules Capital, a leader in customized specialty financing for life science companies. Under the terms of the loan agreement, $10 million will be drawn at closing. An additional $10 million is immediately available to Akero at its sole discretion. Akero may draw an additional $35 million in two separate tranches upon achievement of near-term clinical and financial milestones. An additional $45 million may be drawn in a third tranche, subject to the approval of Hercules Capital. The loan bears an initial interest rate of 7.65% and adjusts with future changes in the prime rate. Akero will pay interest only for the first 24 months, extendable to 36 months on achievement of certain milestones. The loan matures 54 months from closing in December 2026. "We believe there is tremendous patient and clinical value in financing the development of potentially innovative treatments for NASH," said Cristy Barnes, Managing Director at Hercules Capital. "We’re excited to support Akero – both now and in the future – in its continued clinical development of EFX for the treatment of advanced NASH."

On June 16, 2022 Vaxart, Inc. (Nasdaq: VXRT) reported that it will host a question-and-answer webcast with investors and analysts on Wednesday, June 22, 2022, at 1:00 p.m. Eastern Time (Press release, Aviragen Therapeutics, JUN 16, 2022, View Source [SID1234616038]). During the webcast, Andrei Floroiu, President and Chief Executive Officer, Dr. Sean Tucker, SVP and Chief Scientific Officer, and Dr. James Cummings, Chief Medical Officer, will provide an overview of the Company’s oral vaccine programs.

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The Company will take written questions from investors and analysts. Please visit the webcast link to RSVP and submit written questions. Questions may also be submitted in advance to [email protected].

A replay of the webcast will be available on the Investors page of the Company’s website at www.vaxart.com approximately two hours following the conclusion of the event.

As previously announced, Vaxart’s 2022 annual meeting of stockholders has been adjourned to Wednesday, July 6, 2022, at 12:30 p.m. Eastern Time. Vaxart encourages all stockholders of record on April 11, 2022, to vote their shares or change their votes in favor of all the proposals being submitted at the annual meeting by 11:59 p.m. Eastern Time on July 5, 2022.

On June 16, 2022, Sutro Biopharma, Inc. (the "Company") presented the corporate presentation (Presentation, Sutro Biopharma, JUN 16, 2022, View Source [SID1234616035]).

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On June 16, 2022 Checkpoint Therapeutics, Inc. (Checkpoint) (NASDAQ: CKPT), a clinical-stage immunotherapy and targeted oncology company, reported positive interim efficacy results from its registration-enabling clinical trial evaluating its anti-PD-L1 antibody, cosibelimab, in patients with locally advanced cutaneous squamous cell carcinoma (cSCC) who are not candidates for curative surgery or radiation (Press release, Checkpoint Therapeutics, JUN 16, 2022, View Source [SID1234616034]). The design of the interim analysis incorporated recent feedback from the U.S. Food and Drug Administration (FDA) and is intended to potentially support the approval of cosibelimab in this indication. As of the March 2022 data cutoff, the objective response rate (ORR) determined by independent central review (ICR) 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 of 25%.

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Based on these positive results, Checkpoint intends to continue discussions with the FDA on the potential addition of locally advanced cSCC as a second indication in the planned Biologics License Application (BLA) targeted for submission later this year. Checkpoint previously reported positive top-line data from a cohort of 78 patients with metastatic cSCC in its pivotal trial of cosibelimab.

"These exciting positive interim results in this cohort of patients suggest a potential second indication for cosibelimab in locally advanced cSCC, which, when added to the potential labeling in the metastatic cSCC setting, could double the market opportunity at launch for cosibelimab globally. Locally advanced cSCC occurs when tumors become large or have grown deep into underlying tissues, muscles, or nerves, destroying nearby healthy tissue, but have not yet spread metastatically to the lymph nodes or other distant locations of the body," said James Oliviero, President and Chief Executive Officer of Checkpoint.

"We believe the data generated to date continue to show cosibelimab as a differentiated and potentially best-in-class anti-PD-L1 antibody, leveraging a two-fold mechanism of action to deliver robust efficacy with a potentially more favorable safety profile due to its binding to PD-L1 rather than PD-1, an attribute reported in scientific literature as associated with lower rates of severe or worse adverse events as compared to anti-PD-1 therapy," continued Oliviero. "Based on this compelling clinical profile, our planned market-disruptive pricing, and our patent protection through at least 2038, we believe cosibelimab has the opportunity to gain meaningful market share in the $32 billion and growing anti-PD-(L)1 class, while significantly lowering the barrier of high out-of-pocket costs patients endure worldwide to access existing premium-priced cancer therapies."

Cosibelimab was licensed by Checkpoint in 2015 from the Dana-Farber Cancer Institute.

About Cutaneous Squamous Cell Carcinoma
Cutaneous squamous cell carcinoma (cSCC) is the second most common type of skin cancer in the United States, with an estimated annual incidence of approximately 1 million cases according to the Skin Cancer Foundation. While most cases are localized tumors amenable to curative resection, approximately 40,000 cases will become advanced, and an estimated 15,000 people will die from their disease. In addition to being a life-threatening disease, cSCC causes significant functional morbidities and cosmetic deformities based on tumors commonly arising in the head and neck region and invading blood vessels, nerves and vital organs such as the eye or ear.

About Cosibelimab
Cosibelimab (formerly referred to as CK-301) is a potential best-in-class, high affinity, fully-human monoclonal antibody of IgG1 subtype that directly binds to programmed death ligand-1 (PD-L1) and blocks the PD-L1 interaction with the programmed death receptor-1 (PD-1) and B7.1 receptors. Cosibelimab’s primary mechanism of action is based on the inhibition of the interaction between PD-L1 and its receptors PD-1 and B7.1, which removes the suppressive effects of PD-L1 on anti-tumor CD8+ T-cells to restore the cytotoxic T cell response. Cosibelimab is potentially differentiated from the currently marketed PD-1 and PD-L1 antibodies through sustained >99% target tumor occupancy to reactivate an antitumor immune response and the additional benefit of a functional Fc domain capable of inducing antibody-dependent cell-mediated cytotoxicity (ADCC) for potential enhanced efficacy in certain tumor types.