On March 28, 2024 Heron Therapeutics, Inc. (Nasdaq: HRTX), a commercial-stage biotechnology company, reported that Craig Collard, Chief Executive Officer of Heron, will participate in a fireside chat on Wednesday, April 10, 2024, at 11:00 AM ET at the 23rd Annual Needham Virtual Healthcare Conference (Press release, Heron Therapeutics, MAR 28, 2024, View Source [SID1234641565]). The conference is being held in a virtual format, April 8-11, 2024.

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A live webcast of the fireside chat will be available on the Company’s website at www.herontx.com in the Investor Resources section.

On March 28, 2024 Galera Therapeutics, Inc. (Nasdaq: GRTX), a biopharmaceutical company focused on developing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer, reported financial results for the fourth quarter and year ended December 31, 2023 and provided recent corporate updates (Press release, Galera Therapeutics, MAR 28, 2024, View Source [SID1234641564]).

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"As a result of last year’s FDA request for a second Phase 3 trial to support the avasopasem NDA, we have implemented key measures to extend our cash runway and assess strategic alternatives," said Mel Sorensen, M.D., Galera’s President and CEO. "Among these measures, we recognize that the decision to discontinue our GRECO trials was challenging for our team and our clinical investigators, but we believe it was the appropriate decision, following the negative futility analysis in the pancreatic trial. We are continuing to explore strategic options to maximize value to our shareholders, including a potential development path for avasopasem. This process could ultimately result in the dissolution of the Company."

Recent Corporate Updates

General Corporate Updates

In August 2023, the Company announced a reduction in force, which reduced the Company’s workforce by approximately 70%, as of August 9, 2023 (Workforce Reduction). The decision was made to reduce operational costs as part of broader cost-saving measures.
In October 2023, the Company announced it had engaged Stifel, Nicolaus & Company, Inc., as its financial advisor, to assist in reviewing strategic alternatives with the goal of maximizing value for its stockholders.
Radiotherapy-Induced Severe Oral Mucositis (SOM)

In February 2023, the Company announced that the U.S. Food and Drug Administration (FDA) accepted for filing and granted priority review to the New Drug Application (NDA) for avasopasem manganese (avasopasem) for radiotherapy-induced SOM in patients with head and neck cancer (HNC) undergoing standard-of-care treatment. The Prescription Drug User Fee Act (PDUFA) target date assigned by the FDA for the NDA was August 9, 2023.
In August 2023, the FDA issued a Complete Response Letter (CRL) for the NDA for avasopasem. In the CRL, the FDA communicated that the data from the Phase 2b GT-201 and Phase 3 ROMAN trials were not sufficient for approval and that an additional clinical trial will be required for an NDA resubmission.
In September 2023, a Type A meeting was held with the FDA to understand the FDA’s rationale for its decision and discuss next steps to support an NDA resubmission.
In October 2023, the Company received official meeting minutes from the Type A meeting in which the FDA reiterated the need for an additional Phase 3 trial to support resubmission. The Company continues to explore potential options for the development of avasopasem.
Cisplatin-Related Chronic Kidney Disease (CKD)

In November 2023, the Company presented the prospectively collected cisplatin-related CKD data from the Phase 3 ROMAN trial at the American Society of Nephrology (ASN) Kidney Week 2023 meeting, which took place November 2-5 in Philadelphia, PA. The oral presentation, titled "Effects of Avasopasem on Rates of Cisplatin-Induced Acute Kidney Injury and Chronic Kidney Disease," reported significant preservation of kidney function across cisplatin dosing schedules, including a 50% reduction in the avasopasem arm in the incidence of CKD one year following treatment with cisplatin compared to placebo. During the treatment phase, lower incidences of acute renal adverse events were also observed in the avasopasem arm compared to placebo.
Locally Advanced Pancreatic Cancer (LAPC)

In May 2023, the FDA granted Orphan Drug Designation for rucosopasem for the treatment of pancreatic cancer.
In October 2023, the Company decided to halt the Phase 2b GRECO-2 trial of rucosopasem manganese (rucosopasem) in patients with LAPC, following an early futility analysis of the trial which indicated that the trial was unlikely to succeed as designed.
Non-Small Cell Lung Cancer (NSCLC)

In October 2023, following the futility analysis of the GRECO-2 trial, the Company decided to halt the randomized, placebo-controlled Phase 1/2 GRECO-1 trial of rucosopasem in patients with NCSLC.
Fourth Quarter 2023 Financial Highlights

Research and development expenses were $3.2 million in the fourth quarter of 2023, compared to $8.1 million for the same period in 2022. The decrease was primarily attributable to a decrease in avasopasem development costs and lower personnel-related expenses due to the Workforce Reduction.
General and administrative expenses were $2.0 million in the fourth quarter of 2023, compared to $5.0 million for the same period in 2022. The decrease was primarily attributable to lower personnel-related expenses due to the Workforce Reduction and the halting of avasopasem commercial preparation efforts following the receipt of the CRL from the FDA for the avasopasem NDA for radiotherapy-induced SOM in August 2023.
Galera reported a net loss of $(5.6) million, or $(0.10) per share, for the fourth quarter of 2023, compared to a net loss of $(16.2) million, or $(0.58) per share, for the same period in 2022.
As of December 31, 2023, Galera had cash and cash equivalents of $18.3 million. Galera expects that its existing cash and cash equivalents will enable Galera to fund its operating expenses and capital expenditure requirements into the second quarter of 2025.
Full Year 2023 Financial Highlights

Research and development expenses were $24.1 million for the year ended December 31, 2023, compared to $31.0 million for the year ended December 31, 2022. The decrease was primarily attributable to a decrease in avasopasem development costs and lower personnel-related expenses due to the Workforce Reduction, partially offset by an increase in rucosopasem development costs.
General and administrative expenses were $22.8 million for the year ended December 31, 2023, compared to $20.2 million for the year ended December 31, 2022. The increase was primarily attributable to avasopasem commercial preparations in 2023, partially offset by a decrease in personnel-related expenses driven by the Workforce Reduction.
As a result of the Workforce Reduction, the Company incurred restructuring-related charges of $2.3 million in the third quarter of 2023, primarily consisting of severance payments, employee benefits and related costs.
Galera reported a net loss of $(59.1) million, or $(1.33) per share, for the year ended December 31, 2023, compared to a net loss of $(62.2) million, or $(2.30) per share, for the year ended December 31, 2022.

On March 28, 2024 Fortress Biotech, Inc. (Nasdaq: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue, reported financial results and recent corporate highlights for the full-year ended December 31, 2023 (Press release, Fortress Biotech, MAR 28, 2024, View Source [SID1234641562]).

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Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "In 2023, we built a significant amount of momentum to position our Company to achieve multiple milestones in 2024. We also generated record consolidated net revenues of $84.5 million in 2023, the majority of which came from the sales and milestone payments from our dermatology and rare disease businesses."

Dr. Rosenwald continued, "We are pleased that the U.S. Food and Drug Administration ("FDA") accepted the New Drug Application ("NDA") filing for DFD-29 earlier this month and look forward to the Prescription Drug User Fee Act ("PDUFA") goal date of November 4, 2024. Across our portfolio, we could receive up to four NDA and Biologics License Application ("BLA") regulatory approvals over the next 18 months, while we continue to advance our 25 development stage programs in 2024."

2023 and Recent Corporate Highlights:

Regulatory Milestones and Updates

· In January 2024, we submitted an NDA to the FDA seeking approval for DFD-29 (Minocycline Hydrochloride Modified Release Capsules, 40 mg) for the treatment of inflammatory lesions and erythema of rosacea in adults. In March 2024, the FDA accepted the NDA and has set a PDUFA goal date of November 4, 2024. If approved, DFD-29 has the potential to become the only oral, systemic therapy to address both inflammatory lesions and erythema (redness) from rosacea, as demonstrated in clinical trials. DFD-29 is currently in development at our partner company, Journey Medical Corporation (Nasdaq: DERM) ("Journey Medical").

1 The development programs depicted in this press release include product candidates in development at Fortress, at Fortress’ private subsidiaries (referred to herein as "subsidiaries"), at Fortress’ public subsidiaries (referred to herein as "partner companies") and at entities with whom one of the foregoing parties has a significant business relationship, such as an exclusive license or an ongoing product-related payment obligation (such entities referred to herein as "partners"). The words "we", "us" and "our" may refer to Fortress individually, to one or more of our subsidiaries and/or partner companies, or to all such entities as a group, as dictated by context.

· We submitted a BLA to the FDA for cosibelimab, our investigational anti-PD-L1 antibody, as a treatment for patients with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or radiation, in January 2023. In December 2023, the FDA issued a complete response letter ("CRL") for the cosibelimab BLA. The CRL only cited findings that arose during a multi-sponsor inspection of a third-party contract manufacturing organization as approvability issues to address in a resubmission. The CRL did not state any concerns about the clinical data package, safety or labeling for the approvability of cosibelimab. We believe we can address the feedback in a resubmission to enable marketing approval in 2024. We also secured additional U.S. patent protection for cosibelimab through at least May 2038. Cosibelimab is currently in development at our partner company, Checkpoint Therapeutics, Inc. (Nasdaq: CKPT) ("Checkpoint").

· Based on its public statements, AstraZeneca plc ("AstraZeneca") has estimated that it expects the FDA to accept its BLA submission of CAEL-101 (anselamimab) to treat AL amyloidosis for review in 2025. In 2021, AstraZeneca acquired Caelum Biosciences, Inc. (founded by Fortress) for an upfront payment of approximately $150 million paid to Caelum shareholders, of which approximately $56.9 million was paid to Fortress. The agreement also provides for additional potential payments to Caelum shareholders, including approximately $148 million to Fortress, payable upon the achievement of regulatory and commercial milestones.

· In December 2023, we completed the asset transfer of CUTX-101 (copper histidinate for Menkes disease) to Sentynl, a wholly owned subsidiary of Zydus Lifesciences Ltd. The CUTX-101 rolling NDA submission is ongoing and is expected to be completed by Sentynl in 2024. Cyprium Therapeutics, Inc. ("Cyprium"), our subsidiary company that developed CUTX-101, will retain 100% ownership over any FDA priority review voucher that may be issued at NDA approval for CUTX-101.

· In October 2023, we announced that the FDA accepted our Investigational New Drug application to initiate a Phase 1 open-label, multicenter clinical trial to assess the safety, tolerability and efficacy of MB-109, a novel combination of MB-101 (IL13Rα2-targeted CAR-T cell therapy) and MB-108 (HSV-1 oncolytic virus), for the treatment of IL13Rα2+ recurrent GBM and high-grade astrocytoma. MB-109 is currently in development at our partner company, Mustang Bio, Inc. (Nasdaq: MBIO) ("Mustang Bio").

Commercial Product Updates

· In September 2023, our partner company, Journey Medical, entered into an exclusive license agreement with Maruho Co., Ltd. ("Maruho"), a Japanese company specializing in dermatology as well as Journey Medical’s exclusive licensing partner that developed and is commercializing Qbrexza (Rapifort) in Japan. Under the terms of a new license agreement, Journey Medical received a $19 million nonrefundable upfront payment and granted Maruho an exclusive license to develop and commercialize Qbrexza (glycopyrronium tosylate hydrate) for the treatment of hyperhidrosis in South Korea, Taiwan, Hong Kong, Macau, Thailand, Indonesia, Malaysia, Philippines, Singapore, Vietnam, Brunei, Cambodia, Myanmar and Laos (the "Territory"). Maruho is responsible for all development and commercialization costs for the program throughout the Territory.

· Journey Medical’s total net revenues for the year ended December 31, 2023, were $79.2 million, an increase of $5.5 million, or 7%, compared to total net revenues of $73.7 million for 2022.

· Journey Medical’s total product net revenues were $59.7 million for the year ended December 31, 2023, compared to total product net revenues of $71.0 million for the year ended December 31, 2022.

General Corporate:

· Throughout 2023 and in January 2024, Fortress raised total gross proceeds of approximately $34.9 million in registered direct offerings priced at-the-market under Nasdaq rules and in a public offering.

· In October 2023, Fortress effected a 1-for-15 reverse stock split of its issued and outstanding common stock to bring the Company into compliance with Nasdaq’s minimum bid price requirement for continued listing.

· In April 2023, we announced the execution of an asset purchase agreement for 4D Molecular Therapeutics ("4DMT") to acquire proprietary rights to our short-form human complement factor H asset for the treatment of complement-mediated diseases. Under the terms of the agreement, 4DMT will make cash payments totaling up to ~$140 million in potential late-stage development, regulatory and sales milestones. A range of single-digit royalties on net sales are also payable. Our short-form human complement factor H asset was in development at our subsidiary company, Aevitas Therapeutics, Inc. ("Aevitas"), prior to the asset purchase agreement with 4DMT.

Financial Results:

· As of December 31, 2023, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $83.4 million, compared to $74.7 million as of September 30, 2023, and $181.0 million as of December 31, 2022, an increase of $8.7 million for the fourth quarter and a decrease of $97.6 million for the full year.

· Fortress’ consolidated cash, cash equivalents and restricted cash, totaling $83.4 million as of December 31, 2023, includes $42.2 million attributable to Fortress and private subsidiaries, $1.8 million attributable to Avenue, $4.9 million attributable to Checkpoint, $7.0 million attributable to Mustang Bio and $27.4 million attributable to Journey Medical.

· Subsequent to the end of the fourth quarter, in January 2024, Fortress raised approximately $11.0 million in gross proceeds in a registered direct offering, Checkpoint raised approximately $14.0 million in gross proceeds in a registered direct offering and Avenue raised approximately $5.0 million in gross proceeds from warrant exercise transactions.

· Fortress’ consolidated net revenue totaled $84.5 million for the full year ended December 31, 2023, which included $59.7 million in net revenue generated from our marketed dermatology products. This compares to consolidated net revenue totaling $75.7 million for the full year ended 2022, which included $71.0 million in net revenue generated from our marketed dermatology products.

· Consolidated research and development expenses including license acquisitions totaled $106.1 million for the full year ended December 31, 2023, compared to $134.9 million for the full year ended December 31, 2022.

· Consolidated selling, general and administrative costs were $94.1 million for the full year ended December 31, 2023, compared to $113.7 million for the full year ended December 31, 2022.

· Consolidated net loss attributable to common stockholders was $(68.7) million, or $(8.47) per share, for the full year ended December 31, 2023, compared to net loss attributable to common stockholders of $(94.6) million, or $(15.97) per share for the full year ended December 31, 2022.

On March 28, 2024 Exact Sciences Corp. (Nasdaq: EXAS), a leading provider of cancer screening and diagnostic tests, in collaboration with Mayo Clinic Comprehensive Cancer Center, reported on data showing high sensitivity and specificity for the non-endoscopic Oncoguard Esophagus test that is currently in development for the detection of esophageal adenocarcinoma (EAC) and its precursors, including Barrett’s esophagus (BE) (Press release, Exact Sciences, MAR 28, 2024, View Source [SID1234641561]). Findings from the test’s algorithm training and testing were recently published in the Clinical Gastroenterology and Hepatology journal.

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EAC is the most common form of esophageal cancer in the U.S. with incidence having increased significantly over the past few decades compared to other, more common types of cancer where rates have either declined or remained flat due to advanced technologies in screening.3,4 The majority of EAC cases are diagnosed in advanced stages, with early EAC typically having no signs or symptoms, making screening in high-risk populations a critical need.5 There are approximately 30 million people in the U.S. who are at high-risk for EAC and should be screened.6

"Esophageal adenocarcinoma (EAC) is quickly becoming one of the deadliest cancers, with over 80% of patients dying within five years of diagnosis.7 Barrett’s esophagus (BE) is the only known precursor to EAC and patients with BE have an increased risk of progressing to EAC if not caught early. Standard methods of screening can be invasive and costly for patients, which is why there has been a recent push to change the screening guidelines to include non-endoscopic tests," said Prasad G. Iyer, MD, Professor of Medicine, Division of Gastroenterology and Hepatology, Mayo Clinic Comprehensive Cancer Center. "In collaborating with Exact Sciences, we are able to study the potential of a minimally invasive, non-endoscopic screening tool currently in development in patients at higher risk for EAC. Our algorithm training and testing study accomplishes the next critical step for the accurate, non-endoscopic detection of BE/EAC."

The study used an encapsulated sponge collection device to collect esophageal specimens from 352 participants in the training set and 125 participants in the test set. These were then blindly assayed for methylated DNA markers (MDMs). Cross-validation of the three-MDM panel demonstrated an overall sensitivity of 82% (CI, 68% to 94%) for BE detection at 90% specificity (CI, 79% to 98%) in the training set and 88% sensitivity (CI, 78% to 94%) at 84% specificity (CI, 70% to 93%) in the test set. In both training and test sets, sensitivity was 100% for EAC and BE with high grade dysplasia (HGD) – both treatment-eligible subsets. The collection method was generally well tolerated by study participants and was conducive to office-based implementation.

"In esophageal cancer, earlier screening and detection will provide an important advance, as we have seen in other cancers. While many experts recommend that people with chronic reflux disease and at least one other risk factor be screened for Barrett’s esophagus, only a small number of eligible patients undergo screening currently,"8 said Paul Limburg, Chief Medical Officer of Screening, Exact Sciences. "At Exact Sciences, our work has always focused on the latest innovations in screening and testing to help patients and clinicians have accurate, less invasive, and cost-effective options. We are collaborating with Mayo Clinic to continue the development of the Oncoguard Esophagus test as a minimally invasive solution for earlier detection of EAC and its precursors."

The Oncoguard Esophagus test is currently under development and features of the test described above are outlining current development goals. This test has not been cleared or approved by the U.S. Food and Drug Administration (FDA) or any other national regulatory authority.

Mayo Clinic and Dr. Iyer have a financial interest in the technology referenced in this press release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.

On March 28, 2024 Crown Bioscience, a global contract research organization (CRO) and a JSR Life Sciences company, reported that it will showcase significant contributions in preclinical and translational oncology research at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2024 meeting (Press release, Crown Bioscience, MAR 28, 2024, View Source [SID1234641560]). Highlighting its latest research, the company will present ten posters covering a broad spectrum of data from the company’s R&D program.

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The data presented will cover a range of innovative techniques in oncology and immuno-oncology drug discovery and development. Posters featured at AACR (Free AACR Whitepaper) include:

The in-depth Characterization of Acute Myeloid Leukemia Patient-Derived Xenograft (PDX) Models with Hotspot Gene Mutations for Therapeutic Evaluation.

The development of an Integrated Pipeline for Immuno-Oncology Drug Testing, using patient-derived tumor organoids with a reconstituted tumor microenvironment and fresh ex vivo patient tissue cultures, aiming to enhance therapeutic evaluation and drug development efficiency with translational patient-derived systems.

The utilization of Large Panel Organoid Drug Testing Combined with Biomarker Analysis, set to deliver insights into mechanisms of action and boost efficiency within the drug development pipeline.

Research on KRAS Inhibitors and PARP Inhibitor-Induced Resistant PDX Ovarian Cancer Models further expands on the knowledge base of drug resistance patterns in cancer while also assessing innovative therapeutic approaches for targeting mutated tumors, including combination therapy.

SynAI, an adaptive AI-driven platform for in silico drug synergism screening, optimizes the exploration of compound interactions in early-stage cancer drug development.

Magnetic Resonance Imaging and Bioluminescence Imaging for Evaluating Tumor Burden in Orthotopic Glioblastoma, merging digital technology with cancer research to forecast a new era of therapeutic strategy development.
Abstracts for all ten poster presentations can be found on a dedicated page, Crown Bioscience AACR (Free AACR Whitepaper) posters 2024. Following the show, all will be available for download.

Attendees are encouraged to visit Booth #1717 to learn more about these studies and talk to Crown Bioscience’s scientists to gain deeper insights into the platforms and services on offer to advance oncology research.