On November 14, 2023 Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage 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 third quarter ended September 30, 2023 and provided recent corporate updates (Press release, Galera Therapeutics, NOV 14, 2023, View Source [SID1234637624]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Given the considerable time and investment required for additional clinical trials, we find it prudent to explore strategic options," said Mel Sorensen, M.D., Galera’s President and CEO. "We also made the difficult decision to discontinue our GRECO trials, which we believe is an appropriate step towards cash conservation and maximizing value for our shareholders. We extend our gratitude to all the patients, research teams, and dedicated employees who have contributed to the advances we have made."

Recent Corporate Updates

Radiotherapy-Induced Severe Oral Mucositis (SOM)

In August 2023, the Company announced that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) for 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. 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, the Company held a Type A meeting 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 is exploring potential strategic alternatives, as it is not feasible to conduct an additional trial with the Company’s current resources.
Cisplatin-Related Chronic Kidney Disease (CKD)

In November 2023, the cisplatin-related CKD data from the Phase 3 ROMAN trial was presented 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," described significant reduction in the incidence of CKD one year following treatment with cisplatin in the avasopasem arm, reducing CKD by 50% compared to placebo. This was a prospectively defined endpoint of the ROMAN trial, and the reduction was seen with both cisplatin dosing schedules and across all stages of CKD. The result paralleled improvements in preservation of kidney function across the entire population observed from three months through one year. Lower incidence of renal adverse events was also observed in the avasopasem arm compared to placebo during the treatment phase.
Locally Advanced Pancreatic Cancer (LAPC)

In October 2023, the Company decided to halt the Phase 2b GRECO-2 trial of rucosopasem manganese (rucosopasem) in patients with LAPC, following a futility analysis of the trial. The analysis indicated that the trial was unlikely to succeed as designed. GRECO-2 is a randomized, double-blind, placebo-controlled trial evaluating rucosopasem or placebo in combination with stereotactic body radiation therapy (SBRT) in patients with LAPC. Overall survival was the trial’s primary endpoint. The trial was designed to enroll 220 patients with final analysis at 120 events (deaths). The trial had enrolled 177 patients by the date of the decision to halt the trial, and the futility analysis was conducted based on 35 deaths with a data cutoff of October 9, 2023.
Non-Small Cell Lung Cancer (NSCLC)

In October 2023, the Company decided to halt the randomized, placebo-controlled Phase 1/2 GRECO-1 trial of rucosopasem in patients with NCSLC, following the futility analysis of the GRECO-2 trial.
In October 2023, a poster featuring rucosopasem preclinical data was presented at the 2023 American Society for Radiation Oncology (ASTRO) Annual Meeting, which took place October 1-3, 2023, in San Diego, CA. The poster, titled "Enhanced Peroxide Fluxes and Radiosensitization in Colorectal Tumors but Not Normal Enterocytes from the Combination of Superoxide Dismutase Mimetics and Pharmacological Ascorbate," noted that rucosopasem in combination with pharmacological ascorbate may sensitize tumors to clinically relevant radiotherapy doses while maintaining their normal tissue sparing effects.
General Corporate Updates

In connection with the CRL announcement in August 2023, the Company further announced a reduction in force, which reduced the Company’s workforce by 22 employees, or approximately 70%, as of August 9, 2023 ("Workforce Reduction"). The decision was based on cost-reduction initiatives intended to reduce operating expenses.
To maximize value for shareholders, the Company engaged Stifel, Nicolaus & Company, Inc. to undertake a comprehensive review of strategic alternatives for both the Company and its portfolio of dismutase mimetics. These strategic alternatives may include, but are not limited to, mergers, asset sales, divestiture, licensing arrangements, or other strategic transactions. If the Company is unable to undertake any strategic alternative, it may be required to cease operations. The Company has not set a timetable for completion of this evaluation process and does not intend to disclose further updates unless and until it is determined that further disclosure is appropriate or necessary.
Third Quarter 2023 Financial Highlights

Research and development expenses were $6.1 million in the third 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 reduced personnel-related expenses due to the Workforce Reduction, partially offset by an increase in rucosopasem development costs.
General and administrative expenses were $5.0 million in the third quarter of 2023, compared to $4.9 million for the same period in 2022. The increase was primarily attributable to avasopasem commercial preparations and increased legal expenses, largely offset by reduced personnel-related expenses due to the Workforce Reduction and reduced insurance expense.
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 $(15.1) million, or $(0.33) per share, for the third quarter of 2023, compared to a net loss of $(16.0) million, or $(0.60) per share, for the same period in 2022.
As of September 30, 2023, Galera had cash, cash equivalents and short-term investments of $28.4 million. Galera expects that its existing cash, cash equivalents and short-term investments, taking into account the discontinuation of the GRECO-1 and GRECO-2 trials, will enable Galera to fund its operating expenses and capital expenditure requirements into 2025.

On November 14, 2023 Fortress Biotech, Inc. (Nasdaq: FBIO) ("Fortress"), an innovative biopharmaceutical company focused on efficiently acquiring, developing and commercializing or monetizing promising therapeutic products and product candidates, reported its financial results and recent corporate highlights for the third quarter ended September 30, 2023 (Press release, Fortress Biotech, NOV 14, 2023, View Source [SID1234637623]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Lindsay A. Rosenwald, M.D., Fortress’ Chairman, President and Chief Executive Officer, said, "In the third quarter of 2023, Fortress and our partner companies and subsidiaries continued to advance our diverse portfolio of drug candidates. Our total consolidated net revenue this quarter was $34.8 million, which includes an upfront payment of $19 million that Journey Medical received upon entering into an exclusive license agreement with Maruho for commercialization of Qbrexza in additional territories in Asia. We’re looking forward to multiple significant near-term milestones, including potentially up to four New Drug Application ("NDA") and Biologics License Application ("BLA") submissions to the U.S. Food and Drug Administration ("FDA") between 2023 and 2025, one of which is a NDA for DFD-29 to treat rosacea around the end of this year. We are also anticipating the PDUFA goal date of January 3, 2024, for cosibelimab, our investigational anti-PD-L1 antibody, as a treatment for patients with metastatic or locally advanced cutaneous squamous cell carcinoma ("cSCC")."

Recent Corporate Highlights2:

Marketed Dermatology Products

● Journey Medical Corporation (Nasdaq: DERM) ("Journey Medical"), our partner company, primarily focuses on selling and marketing of prescription dermatology products.
● In September 2023, Journey Medical entered into an exclusive license agreement with Maruho Co., Ltd. ("Maruho"), a Japanese company specializing in dermatology as well as Journey’s exclusive licensing partner that developed and is commercializing Qbrexza (Rapifort) in Japan. Under the terms of the Agreement, Journey Medical received a $19 million nonrefundable upfront payment
1 Includes CAEL-101, an asset in development at Caelum Biosciences (a former subsidiary now 100% owned by AstraZeneca’s Alexion) with respect to which Fortress remains eligible to receive substantial milestone payments.

2 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 which 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.

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 in the third quarter of 2023 were $34.5 million, an increase of $18.4 million, or 114%, compared to total net revenues of $16.1 million in the third quarter of 2022.
● Journey Medical’s total product net revenues were $15.3 million for the third quarter of 2023, compared to third quarter 2022 total product net revenues of $16.0 million.

Fortress, through its subsidiaries, partner companies and partners, has a broad and diverse pipeline of clinical-stage programs being evaluated in over 20 ongoing clinical trials. We look forward to potential filings of up to four NDA and BLA submissions from 2023 through 2025, including:

● DFD-29 – modified release oral minocycline for rosacea
● Cosibelimab – anti-PD-L1 antibody for solid tumors
● CUTX-101 – copper histidinate for Menkes disease
● CAEL-101 – light chain fibril-reactive monoclonal antibody for AL amyloidosis

We also expect to continue to advance our early-to-mid-clinical-stage candidates, some of which may begin pivotal trials during the next twelve to eighteen months, including:

● Dotinurad – urate transporter (URAT1) inhibitor for gout and hyperuricemia
● IV tramadol – intravenous small molecule for acute post-operative pain
● MB-106 – CD20-targeted CAR-T cell therapy for hematologic malignancies
● Triplex – multi-antigen, modified vaccinia Ankara-based (MVA) vaccine for cytomegalovirus (CMV)
● MB-117 – ex vivo lentiviral gene therapy for newborns with XSCID (X-linked severe combined immunodeficiency)
● MB-217 – ex vivo lentiviral gene therapy for previously transplanted children and young adults with XSCID
● AJ201 – Nrf1 and Nrf2 activator, androgen receptor degradation enhancer for spinal and bulbar muscular atrophy (SBMA), also known as Kennedy’s Disease
● BAER-101 – GABAA α2/3 positive allosteric modulator for refractory epilepsies
● MB-109 – IL13Rα2-targeted CAR-T cell therapy combined with HSV-1 oncolytic virus for recurrent glioblastoma and high-grade astrocytomas

General Corporate:

● In November 2023, Fortress raised approximately $10.0 million in gross proceeds 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.

Financial Results:

● As of September 30, 2023, Fortress’ consolidated cash, cash equivalents and restricted cash totaled $74.7 million, compared to $89.2 million as of June 30, 2023, a decrease of $14.5 million during the quarter.
● Fortress’ consolidated cash, cash equivalents and restricted cash, totaling $74.7 million as of September 30, 2023, includes $37.7 million attributable to Fortress and private subsidiaries, $0.2
million attributable to Avenue, $1.8 million attributable to Checkpoint, $10.3 million attributable to Mustang Bio and $24.7 million attributable to Journey Medical.
● Subsequent to the end of the third quarter, in November 2023, Fortress raised approximately $10.0 million in gross proceeds in a public offering and Avenue raised approximately $5.0 million in gross proceeds in a public offering. In addition, in October 2023, Checkpoint raised approximately $11.1 million in gross proceeds from the exercise of warrants and Mustang Bio raised approximately $4.4 million in gross proceeds in a registered direct offering.
● Fortress’ consolidated net revenue totaled $34.8 million for the third quarter of 2023, which included $15.3 million in net product revenue generated from our marketed dermatology products. This compares to consolidated net revenue totaling $16.5 million for the third quarter of 2022, which included $16.0 million in net product revenue generated from our marketed dermatology products.
● Consolidated research and development expenses including license acquisitions were $20.3 million for the third quarter of 2023, compared to $29.9 million for the third quarter of 2022.
● Consolidated selling, general and administrative expenses were $21.7 million for the third quarter of 2023, compared to $30.1 million for the third quarter of 2022.
● Consolidated net loss attributable to common stockholders was $7.1 million, or $0.94 per share, for the third quarter of 2023, compared to consolidated net loss attributable to common stockholders of $24.5 million, or $4.11 per share for the third quarter of 2022.

On November 14, 2023 Eli Lilly and Company (NYSE: LLY) reported that it will attend the sixth annual Evercore ISI HealthCONx Conference on Nov. 28, 2023. Daniel Skovronsky, M.D., Ph.D., executive vice president, chief scientific and medical officer, and president of Lilly Research Laboratories, will participate in a fireside chat at 3:50 p.m., Eastern time (Press release, Eli Lilly, NOV 14, 2023, https://investor.lilly.com/news-releases/news-release-details/lilly-participate-evercore-isi-healthconx-conference-0 [SID1234637622]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

On November 14, 2023 Defence Therapeutics Inc. ("Defence" or the "Company"), (CSE: DTC, USOTC: DTCFF, FSE: DTC), one of the leading Canadian biotechnology companies working in the field of immune-oncology reported that it has successfully submitted on November 9, 2023 an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its ACCUM-002TM Dimer CDCA-SV40 commonly named "AccuTOX", an injectable anticancer molecule, for the treatment of solid cancer tumors (Press release, Defence Therapeutics, NOV 14, 2023, View Source;utm_medium=rss&utm_campaign=defences-successful-submission-of-an-investigational-new-drug-ind-application-for-accutox-as-an-injectable-anticancer-treatment-for-solid-tumors [SID1234637621]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

AccuTOX is a derivative of the initial Accum molecule, which has been reported to target cancer on multiple fronts. AccuTOX disrupts endosomal membranes resulting in impaired intracellular transport mechanisms. AccuTOX also triggers genotoxic effects, blocks DNA repair mechanisms normally used by cancer cells to repair its damaged genome and induces a form of immunogenic cell death capable of turning "ON" the immune system. When previously tested in preclinical animal models under the supervision of Dr. Moutih Rafei, AccuTOX impaired tumor growth resulting in "70-100% survival" of animals with solid T-cell lymphoma, melanoma or breast cancer.

The IND application includes data, reports and overview summaries of numerous studies to evaluate the pharmacology, pharmacokinetics, and toxicology of AccuTOX both in vitro and in vivo, including cancer models. In addition, the application describes the manufacture of the drug substance and drug product to be used in human clinical trials. The main purpose of the IND is to share with the FDA the extensive non-clinical data supporting an acceptable safety profile when AccuTOX will be first administered to humans. The FDA will review the application and determine the acceptability of the data before Defence begins the Phase I clinical trial, which could be as early as Q1-Q2 2024.

"We are thrilled and excited that Defence has achieved a successful submission on its first IND, which represents an important milestone towards advancing AccuTOX into the clinic. We look forward to work with clinical investigators at City of Hope to study this important and novel candidate for the treatment of melanoma and potentially other solid tumors," said Sébastien Plouffe, President & CEO of Defence Therapeutics. "With the continued difficulties encountered in the oncology clinic, we believe that the therapeutic use of AccuTOX provides a novel and powerful approach to combat cancer," he added.

The primary objective of this upcoming Phase I clinical trial, when approved, is to identify the best therapeutic dosing range that would allow clinicians to co-administer the AccuTOX compound with Opdulag, a BMS product containing both anti-LAG3 and anti-PD-1. Several other secondary parameters including therapeutic efficacy will be monitored in treated patients in preparation for a Phase IIa trial on a basket of tumors.

According to Precedence Research, the global cancer therapeutics market size is expected to be worth around US$ 393.61 billion by 2032 from at US$ 164 billion in 2022, growing at a CAGR of 9.20% during the forecast period 2023 to 2032.

ON November 14, 2023 CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of life-threatening diseases and conditions, reported financial results for the third quarter and nine months ended September 30, 2023 and provided an update on recent business events (Press release, CorMedix, NOV 14, 2023, View Source [SID1234637620]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Recent Corporate Highlights:

The FDA completed a preapproval inspection for CorMedix’s primary finished dosage CMO site in September and the Company is pleased with the outcome. In addition, the FDA has also conducted preapproval inspections for DefenCath at suppliers of active pharmaceutical ingredients, contract laboratories involved in testing of product specifications and packaging facilities, and CorMedix is not aware of any outstanding deficiencies or review issues at this time.
CorMedix has intensified preparations for commercial launch with a number of new hires in market access, sales training, pricing and contracting, commercial operations, field medical, and drug safety and pharmacovigilance.
In the event CorMedix receives final FDA approval over the coming days, the Company is targeting a commercial launch before the end of the first quarter of 2024.
The United States Patent and Trademark Office (USPTO) issued a patent with claims directed to the composition of a catheter lock solution for preventing infection and reduced blood flow in central venous catheters. This Patent reflects the unique and proprietary nature of DefenCath, and will extend the Company’s intellectual property protection with an expiration date of April 15, 2042.
Cash and short-term investments, excluding restricted cash, at September 30, 2023 amounted to $86.6 million.
Joe Todisco, CorMedix CEO, commented, "I am very pleased with the Company’s progress on all fronts, most notably the outcomes of the preapproval inspections at our primary CMO facility and other key vendors, as well as our preparations toward commercial readiness. We have accelerated multiple work streams aimed at commercial launch, and made a number of key hires over the past quarter that are essential to ensuring a successful commercial launch following a potential FDA approval of DefenCath. We look forward to providing additional updates over the coming days as we anticipate a response from FDA on or around our target action date."

Third Quarter and Nine Month 2023 Financial Highlights

For the third quarter of 2023, CorMedix recorded a net loss of $9.7 million, or $0.17 per share, compared with a net loss of $6.9 million, or $0.17 per share, in the third quarter of 2022. The higher net loss recognized during the third quarter of 2023 compared with 2022 was attributable to net increase in operating expenses primarily due to increased pre-launch commercial activities for DefenCath.

Operating expenses in the third quarter of 2023 increased approximately 50% to $10.5 million, compared with $7.0 million in the third quarter of 2022. R&D expense increased approximately 14% to $2.7 million compared to $2.3 million in the third quarter of 2022, mainly due to a net increase in costs related to medical affairs activities and increase in personnel related expenses due to additional hires in 2023 as compared with 2022. SG&A expense increased approximately 69% to $7.8 million compared with $4.6 million in the third quarter of 2022. This increase was driven primarily by an increase in costs related to market research studies and pre-launch activities in preparation for the potential marketing approval of DefenCath and as a result of additional hires in 2023 as compared with 2022.

For the nine months ended September 30, 2023, CorMedix recorded a net loss of $31.6 million, or $0.65 per share, compared with a net loss of $21.5 million, or $0.54 per share, in the first nine months of 2022. The increase in net loss in the first nine months of 2023 was driven primarily by an increase in operating expenses.

Operating expenses in the first nine months of 2023 were $33.3 million compared with $22.3 million during the comparable period in 2022, an increase of approximately 49%. This increase was primarily attributable to costs related to market research studies and pre-launch activities in preparation for the potential marketing approval of DefenCath, and an increase in personnel expenses due to additional hires in 2023.

Total cash on hand and short-term investments as of September 30, 2023 was $86.6 million, excluding restricted cash of $0.2 million. The Company believes that, based on the Company’s cash resources at September 30, 2023, it has sufficient resources to fund operations at least twelve months from the filing of its Quarterly Report on Form 10-Q for the quarter ended September 30, 2023.

Conference Call Information

The management team of CorMedix will host a conference call and webcast today, November 14, 2023, at 4:30 PM Eastern Time, to discuss recent corporate developments and financial results. Call details and dial-in information is as follows:

Domestic: 1-888-886-7786
International: 1-416-764-8658
Conference ID: 16013968
Webcast: Webcast Link