Aprea Therapeutics Announces FDA Clearance of IND for APR-1051, its Next Generation WEE1 Kinase Inhibitor for Cyclin E Overexpressing Cancers

On March 11, 2024 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea", or the "Company"), a clinical-stage biopharmaceutical company focused on precision oncology through synthetic lethality, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application (IND 169359) for APR-1051 (Press release, Aprea, MAR 11, 2024, View Source [SID1234641006]).

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"APR-1051 is a next-generation inhibitor of WEE1 kinase and, based on its unique characteristics, we believe it will be best in class," said Dr. Oren Gilad, President and CEO of Aprea. "The FDA’s clearance of our IND is a critical step in the APR-1051 development program. We look forward to evaluating therapeutic activity in patients, focusing on Cyclin E overexpressing cancers, including ovarian and breast cancers."

Based on preclinical studies, APR-1051 is differentiated from other WEE1 inhibitors in its:

Molecular structure;
Selectivity for WEE1 versus off-target inhibition of the polo-like kinase, or PLK, family of kinases;
Potentially superior pharmacokinetic (PK) properties;
Potential absence of QT prolongation at doses that significantly inhibit WEE1*
APR-1051 was discovered and preclinically evaluated by Aprea’s team of chemists and scientists. Aprea has conducted extensive pre-clinical studies with APR-1051, which have demonstrated that the molecule has potent anti-tumor activity, along with a favorable pharmacokinetic (PK) profile. Additionally, pre-clinical data show that APR-1051 may demonstrate less toxicity than other WEE1 inhibitors.*

Clearance of the IND application will allow Aprea to initiate the Phase 1 ACESOT-1051 dose escalation trial to evaluate the safety, tolerability, and preliminary efficacy of APR-1051. Enrollment of the first patient in this study is expected in the first half of 2024 with an update expected in the fourth quarter of the year.

* No head-to-head studies have been conducted with APR-1051

Sonnet BioTherapeutics Announces Early Safety Data from the Company’s Phase 1b/2a Clinical Trial of SON-080 in Chemotherapy-Induced Peripheral Neuropathy (CIPN) Met the Study’s Initial Pre-Specified Objective

On March 11, 2024 Sonnet BioTherapeutics Holdings, Inc. ("Sonnet" or the "Company") (NASDAQ:SONN), a clinical-stage company developing targeted immunotherapeutic drugs, reported that the first Phase 1b/2a clinical trial of SON-080 was cleared to proceed to Phase 2 after review by the independent DSMB (Press release, Sonnet BioTherapeutics, MAR 11, 2024, View Source [SID1234641023]). This study (SB211, NCT05435742) is being conducted at two sites in Australia in patients with persistent CIPN using a new proprietary version of recombinant human Interleukin-6 (rhIL-6), which required confirmation of safety before continued development in Phase 2. Many drugs cause peripheral nerve damage; patients with CIPN experience discomfort that can result in persistent, unbearable pain that may limit chemotherapeutic treatment.

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SB211 is studying a low dose of rhIL-6 that has an amino acid sequence identical to the native molecule. The trial targets serum levels similar to those induced with moderate exercise, which triggers the natural healing of nerves, muscle, and bone. As a pleiotropic cytokine, native IL-6 participates in several physiological processes, including tissue repair, glucose homeostasis, and the innate immune response at lower levels, but it can result in acute pathological inflammation at higher serum levels. Preclinical models of CIPN and DPN show that low dose rhIL-6 has the potential to stimulate nerve regrowth to re-establish normal sensations, thereby reducing pain and normalizing some of the physiological conditions that had deteriorated due to nerve degeneration. Early versions of rhIL-6, including Serono’s atexakin alfa and others, have been tested in hundreds of patients with cancer, diabetes, idiopathic aplastic anemia, or in healthy controls, showing a maximum tolerated dose of 10 µg/kg three times a week (TIW). However, fever, nausea, and vomiting were prominent at doses over 2 µg/kg TIW. Study SB211 was designed in Phase 1b to show safety using lower doses in CIPN with up to about 1 µg/kg of Sonnet’s new version of IL-6 (SON-080) given subcutaneously TIW for twelve weeks.

The protocol required DSMB to review the unblinded safety and tolerability of SON-080 in the first nine patients in SB211. While the data is still blinded to the rest of the team and we do not have access to the responses by group, the initial safety profile mimics that seen in prior studies with lower doses of exogenous rhIL-6. The most prominent symptoms in SB211 included injection site reactions (redness, bruising, pain, or itching) that resolved within a few days, as well as fatigue, body aches, or nausea that were mostly mild with some symptoms that were moderate. One patient developed severe fatigue and withdrew from the study after one month. All adverse events were transient and reversible. The DSMB concluded that the symptoms were tolerable in the initial patients and the study could proceed to Phase 2. The unblinded safety data from two dose levels of SON-080 compared to placebo are expected during the second half of 2024.

"Completion of the Phase 1b portion of SB211 is an important milestone for Sonnet in the Company’s quest to bring a potentially groundbreaking treatment forward for peripheral neuropathies, where there’s a large unmet need," said Pankaj Mohan, Ph.D., Sonnet Founder and Chief Executive Officer. "This trial was designed to initially bridge the large atexakin alfa historical safety database in cancer patients and then to study the neuroprotective and neuro-regenerative effects of SON-080 in Phase 2 in a neurotherapy indication. Owing to the larger market opportunity of the DPN indication, we have received greater potential partnering interest in this indication." Dr. Mohan further added, "The inhibition of IL-6 release in diabetic patients, even after moderate exercise, suggests there is tremendous disease modifying potential for the application of rhIL-6 in DPN. Given the high prevalence of neuropathy in diabetes and the commensurate industry interest in this market, we have prioritized DPN as the best potential indication for Phase 2 development. We have initiated a partnering process to move the asset forward towards commercialization."

"Interleukin-6 has been extensively studied in cancer patients in the past, so the use of SON-080 in CIPN was expected to provide a similar adverse event profile at low doses," said Richard Kenney, M.D., Sonnet’s Chief Medical Officer. "The preclinical models showing improvements in nerve function and histology suggest possible benefits in humans with various types of peripheral neuropathy due to cancer and diabetes. This approach is a unique way to actually treat the underlying causes of peripheral neuropathy with rhIL-6, rather than trying to mask the symptoms. Further, given the business priorities, SB211 CIPN development will be placed on hold and the data will be leveraged to initiate a Phase 2 study in the much larger DPN indication."

About the SB211 Phase 1b/2a Trial

The SB211 study is primarily designed to evaluate the safety, PK, PD, and initial efficacy of two dose levels of SON-080 compared to placebo. The drug is self-administered three times a week, subcutaneously, in patients with CIPN lasting at least 3 months after chemotherapy. The study is being conducted at multiple sites in Australia, in a blinded fashion, comparing SON-080 to placebo. The primary endpoint explores the safety and tolerability of SON-080, with key secondary endpoints intended to measure PK, PD, and immunogenicity. Preliminary efficacy is being explored using standardized pain questionnaires over the course of the trial.

Calidi Biotherapeutics and City of Hope Announce Funding from the California Institute for Regenerative Medicine (CIRM) to Advance CLD-101 (NeuroNova) in Ovarian Cancer

On March 11, 2024 Calidi Biotherapeutics Inc. (NYSE American: CLDI) ("Calidi"), a clinical-stage biotechnology company developing a new generation of targeted oncolytic virotherapies, and City of Hope, one of the largest cancer research and treatment organizations in the United States, today jointly reported that the California Institute for Regenerative Medicine (CIRM) has awarded Karen Aboody, M.D., City of Hope professor in the Department of Stem Cell Biology and Regenerative Medicine and Division of Neurosurgery, a $5.3 million grant to fund preclinical translational studies, product manufacturing, and clinical trial design using Calidi’s licensed oncolytic virotherapy product, CLD-101, in patients with chemo-resistant, metastatic ovarian cancer (Press release, Calidi Biotherapeutics, MAR 11, 2024, View Source [SID1234641041]). CLD-101 is the lead therapeutic candidate in Calidi’s NeuroNova program, comprising tumor-tropic neural stem cells (NSCs) that deliver an oncolytic adenovirus (NSC-CRAd-S-pk7) selectively to tumor sites.

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Aboody and her team have been key collaborators with Calidi over the past four years, and Calidi’s knowledge and expertise has enhanced key attributes of the CLD-101 program, in particular Calidi’s next generation manufacturing process and potency optimization, leading to higher yields and improved oncolytic activity of the CLD-101 product for use in high-grade glioma, and now ovarian cancer. This proposed CLD-101 treatment utilizes a neural stem cell line to deliver an oncolytic adenovirus directly to abdominal ovarian tumor sites. The virus infects and kills the tumor cells, even if they are chemo-resistant, which then stimulates the patient’s immune system to infiltrate, recognize, and fight the tumor. In collaboration with Calidi, Aboody will lead the groundbreaking work.

"We are delighted to receive this financial support from CIRM, which enables us to complete the preclinical translational studies, product manufacturing, and clinical trial design needed for FDA approval to move this novel treatment to ovarian cancer patients within two to three years. This CIRM grant will allow us to further utilize Calidi’s manufacturing processes, which we believe will optimize the potency of the CLD-101 oncolytic virotherapy treatment product, and improve clinical outcomes for women with stage III ovarian cancer," said Aboody.

"This is the third grant from CIRM supporting the development of Calidi’s assets, further validating our novel cell therapy platforms and their versatility for multiple cancer indications," said Allan Camaisa, CEO and Chairman of the Board of Calidi Biotherapeutics. "We have been focused on enhanced cell manufacturing processes and providing novel options to patients with a variety of serious cancers, and we are grateful for the continued support from CIRM, as we advance our clinical programs."

In July 2023, Calidi announced the dosing of the first patient in a City of Hope Phase 1 clinical trial evaluating CLD-101 in recurrent high-grade glioma patients. The company expects to provide a clinical update in the first half of 2024. Additionally, a previously completed Phase 1 dose escalation trial assessed the safety of a single dose of CLD-101 administered into the walls of the surgical resection cavity, in newly diagnosed high-grade glioma patients. In this trial, CLD-101 demonstrated safety and the stimulation of an anti-tumor response, with results published in Lancet Oncology.

For more information about this trial, click here or contact [email protected] or (626) 218-4062.

Aboody has financial interest in and is a paid advisory board member for Calidi Biotherapeutics.

About CLD-101

The CLD-101 platform, which includes NSC-CRAd-S-pk7, is an allogeneic, "off-the-shelf" therapy comprised of an immortalized NSC line loaded with an engineered oncolytic adenovirus. In High Grade Glioma brain cancer patients, upon surgical resection of tumor, NSC-CRAd-S-pk7 is injected into the walls of the resection cavity. The anti-cancer virus it releases is expected to infect and kill any remaining tumor cells, which is also postulated to stimulate a tumor-specific immune response from the patient. Calidi holds an exclusive worldwide licensing agreement covering the NSC-CRAd-S-pk7 technology.

Aprea Therapeutics Announces Private Placement Financing of up to $34.0 Million

On March 11, 2024 Aprea Therapeutics, Inc. (Nasdaq: APRE) ("Aprea", or the "Company"), a clinical-stage biopharmaceutical company focused on precision oncology through synthetic lethality, reported that it has entered into a securities purchase agreement with new and existing healthcare focused institutional investors and certain Company insiders to raise up to $34.0 million in gross proceeds, including initial upfront funding of $16.0 million and up to an additional $18.0 million upon cash exercise of accompanying warrants at the election of the investors (Press release, Aprea, MAR 11, 2024, View Source [SID1234641007]).

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The financing is being led by Sphera Healthcare and includes participation from new and existing healthcare-focused investors, including Nantahala Capital, DAFNA Capital Management, Exome Asset Management and Stonepine Capital Management, among others, as well as certain Company insiders.

"This meaningful financing led by high quality healthcare institutions will support Aprea in our goal to be a leader in the field of Synthetic Lethality (SL) and DNA Damage and Response (DDR)," said Dr. Oren Gilad, President and CEO of Aprea. "It will provide the capital to fund our Phase 1 ACESOT-1051 clinical trial evaluating a highly potent, oral WEE1 inhibitor for Cyclin E over-expressing cancers including breast and ovarian cancers as well as continuation of patient enrollment in the dose expansion portion of the Phase 1/2a clinical trial (AR-276-01) evaluating ATR inhibitor, ATRN-119, in patients with advanced solid tumors having mutations in defined DDR-related genes."

Maxim Group LLC is acting as the sole placement agent for the private placement.

Pursuant to terms of the securities purchase agreement, Aprea will issue an aggregate of 2,194,788 shares of its common stock (or pre-funded warrants in lieu thereof) and accompanying warrants to purchase up to an aggregate of 2,194,788 shares of its common stock at a combined purchase price of $7.29 per share and accompanying warrants, in accordance with the "Minimum Price" requirement as defined in the Nasdaq rules. The accompanying warrants will consist of two tranches:

Tranche A warrants to purchase up to 1,097,394 shares of common stock at an exercise price of $7.29 per share for an aggregate of up to $8.0 million and will expire at the earlier of (i) 30 days following the announcement of the recommended Phase 2 dose for the Company’s ATR inhibitor program for ATRN-119 and the daily VWAP of the Company’s common stock equaling or exceeding $14.58 per share for 30 consecutive trading days following the announcement and (ii) three years from the date of issuance.
Tranche B warrants to purchase up to 1,097,394 shares of common stock at an exercise price of $9.1125 per share for an aggregate of up to $10.0 million and will expire at the earlier of (i) 30 days following the announcement of the recommended Phase 2 dose for the Company’s WEE1 inhibitor program for APR-1051 and the daily VWAP of the Company’s common stock equaling or exceeding $18.225 per share for 30 consecutive trading days following the announcement and (ii) five years from the date of issuance.
In lieu of shares of common stock, certain investors are purchasing pre-funded warrants at a combined purchase price of $7.289 per pre-funded warrant and accompanying warrants, which equals the purchase price per share of common stock and accompanying warrant, less the $0.001 per share exercise price of each pre-funded warrant. The private placement is expected to close on or about March 13, 2024 subject to satisfaction of customary closing conditions.

Aprea intends to use the upfront net proceeds from the private placement for general corporate purposes and to fund clinical development of APR-1051, the Company’s WEE1 inhibitor product candidate which recently received IND clearance. The aggregate net proceeds (assuming the cash exercise of all accompanying warrants) are expected to be sufficient to fund the Company into 2026.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering, and the securities have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws. Accordingly, the securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. The Company has agreed to file a registration statement with the Securities and Exchange Commission registering the resale of the shares of common stock purchased in the private placement and shares of common stock underlying the warrants.

This press release does not constitute an offer to sell or the solicitation of an offer to buy the securities, nor shall there be any sale of the securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state. Any offering of the securities under the resale registration statement will only be made by means of a prospectus.

Theriva™ Biologics to Participate in the BIO-Europe Spring Conference

On March 11, 2024 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported that Company’s Management will provide a corporate update and participate in one-on-one meetings at the BIO-Europe Spring Conference, to be held in Barcelona, Spain from March 18-20, 2024 (Press release, Theriva Biologics, MAR 11, 2024, View Source [SID1234641024]).

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BIO-Europe Spring Conference
Format: Corporate presentation and one-on-one meetings
Presentation Date: Monday, March 18, 2024
Presentation Time: 3:00pm CET
Presentation Location: Room 133/134, Barcelona International Convention Centre (CCIB)