On May 15, 2023 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported financial results for the first quarter ended March 31, 2023 and provided a corporate update (Press release, Decibel Therapeutics, MAY 15, 2023, View Source [SID1234631713]).

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"2023 is a very important year for Decibel as we work to advance the CHORD global Phase 1/2 trial for DB-OTO, our gene therapy pipeline and DB-020. The initiation of our first gene therapy clinical study is the first of several important inflection points that we expect to occur in 2023, including dosing the initial patients and early clinical readouts from the CHORD clinical trial. We believe DB-OTO has the potential to be a best-in-class gene therapy for individuals with otoferlin-related hearing loss," said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. "The multiple challenges of severe hearing loss continue to drive our vision. Congenital hearing loss is recognized as a potential neurodevelopmental emergency and with our product candidates we are aiming to provide durable, physiological hearing to address this."

Pipeline Highlights and Upcoming Milestones:

Gene Therapies for Congenital, Monogenic Hearing Loss

Initiated CHORD Phase 1/2 Clinical Trial of DB-OTO; Received European Orphan Drug Designation for DB-OTO:
DB-OTO is an AAV-based, dual-vector, gene therapy product candidate designed to be administered a single time to selectively express functional otoferlin (OTOF) in the inner hair cells of individuals with OTOF deficiency with the goal of enabling the ear to transmit sound to the brain and enable durable, physiological hearing. Decibel is developing DB-OTO in collaboration with Regeneron Pharmaceuticals, and Decibel retains global commercial rights to the product candidate.
In May 2023, Decibel announced approval from the Spanish Agency of Medicines and Medical Devices (AEMPS) and in January 2023, Decibel announced approval from the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) for its Clinical Trial Applications (CTAs) for its CHORD clinical trial of DB-OTO. The Company has previously announced clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application for CHORD. Decibel expects that the first two patients in CHORD in the U.S. will be as young as seven years of age and that participants in the U.K. and Spain will be infants two years of age and younger.
In March 2023, the European Medicines Agency (EMA) Committee on Orphan Medicinal Products (COMP) and European Commission (EC) issued orphan drug designation for DB-OTO. Orphan drug designation is granted by the EC for medicines in development to treat rare conditions affecting no more than five in 10,000 people in the European Union, provided there is no other satisfactory treatment option or the medicine can be of significant benefit to those affected by a specific condition. Medicines that are granted orphan drug designation by the EC qualify for financial and regulatory incentives including protocol assistance, possible exemptions or reductions in certain regulatory fees, and, if approved for marketing, ten years of market exclusivity in the European Union.
Announced Presentations Related to DB-OTO and AAV.103 at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting; Presented New Non-Clinical Safety Data from DB-OTO at the 46th Annual Association for Research in Otolaryngology (ARO) MidWinter Meeting:
In May 2023, Decibel announced it will deliver podium presentations related to DB-OTO and AAV.103 at ASGCT (Free ASGCT Whitepaper). Decibel will also present posters related to its preclinical pipeline and its platform.
In February 2023, Decibel presented new non-clinical data supporting the clinical development of DB-OTO. In Good Laboratory Practices studies, Decibel did not observe any adverse DB-OTO-related findings in otic or non-otic tissues across any evaluation of OTOF-deficient mice or non-human primates.
Selected a Product Candidate for AAV.103 GJB2-Related Hearing Loss Program and Initiated Manufacturing Activities to Support IND-Enabling Studies: In February 2023, Decibel presented preclinical data at ARO on its AAV.103 gene therapy product candidate designed to restore hearing in individuals with mutations in the gap junction beta-2 (GJB2) gene, the leading cause of autosomal recessive, non-syndromic, congenital hearing loss worldwide. Results demonstrated that selective expression of GJB2 in non-sensory cells that normally express GJB2 resulted in robust and durable hearing restoration in a mouse model of GJB2 deficiency. Decibel is developing the AAV.103 program in collaboration with Regeneron Pharmaceuticals and has achieved a pre-IND milestone in April 2023 related to initiation of manufacturing under the collaboration agreement. Decibel retains global commercial rights to the product candidate.
Otoprotection Therapeutic

DB-020 Phase 1b Clinical Trial Data to Be Presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2023 Annual Meeting: Decibel will present data from the Phase 1b clinical trial of DB-020, its product candidate for prevention of cisplatin-induced hearing loss in patients receiving cisplatin chemotherapy. In the interim results from the trial, DB-020 demonstrated reduced ototoxicity, a favorable safety and tolerability profile and no observed effect on systemic cisplatin pharmacokinetics (PK) in such patients. The Company is seeking advice from regulatory agencies concerning the future development of DB-020.
Corporate Update:

Board of Directors Update: In March 2023, Decibel announced the appointment of Matthew Kapusta, MBA, to its Board of Directors. Mr. Kapusta serves as the CEO of uniQure, a gene therapy company.
First Quarter 2023 Financial Results:

Cash Position: As of March 31, 2023, cash, cash equivalents and available-for-sale securities were $87.9 million, compared to $104.6 million as of December 31, 2022.
Research and Development Expenses: Research and development expenses were $7.9 million for the first quarter of 2023, compared to $7.5 million for the same period in 2022. The increase in research and development expenses for the first quarter of 2023 was primarily due to costs to prepare for the Company’s Phase 1/2 clinical trial of DB-OTO, higher research costs related to its other preclinical gene therapy programs and higher personnel-related costs due to increased headcount and wages.
General and Administrative Expenses: General and administrative expenses were $6.2 million for the first quarter of 2023, compared to $6.6 million for the same period in 2022. The decrease in general and administrative expenses for the first quarter of 2023 was primarily due to lower professional fees including external consulting, advisory, legal and audit services.
Financial Guidance:

Based on its current operating and development plans, Decibel believes that its existing cash, cash equivalents and available-for-sale securities will fund its planned operating expenses into the first half of 2024.

On May 15, 2023 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on developing innovative therapies for liver and other chronic diseases, reported corporate updates and financial results for the first quarter ended March 31, 2023 (Press release, CymaBay Therapeutics, MAY 15, 2023, View Source [SID1234631712]).

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Sujal Shah, President and CEO of CymaBay, stated, "In a year with RESPONSE pivotal phase 3 data fast approaching, we have already achieved a number of key goals thus far in 2023. In January, we licensed rights to Kaken Pharmaceutical Company to develop and commercialize seladelpar for patients with PBC in Japan and strengthened our balance sheet with a successful financing. In April, we announced that the ENHANCE phase 3 trial results were published in Hepatology, a preeminent medical journal in the field of liver disease. Last week, we announced the appointment of Harish Shantharam as Chief Financial Officer. These accomplishments set up the remainder of 2023 as we look to share data from RESPONSE, manufacture commercial supplies of seladelpar, prepare regulatory filings and advance our pre-commercial planning. We look forward to sharing updates across all of these activities through the remainder of the year."

Corporate Updates:

Appointed Harish Shantharam to the role of Chief Financial Officer in May 2023. Harish brings nearly 20 years of experience leading financial functions in biotech with a strong emphasis on setting strategy and building financial planning and accounting operations to prepare companies for commercial drug launch.
ENHANCE manuscript titled "Seladelpar efficacy and safety at 3 months in patients with primary biliary cholangitis: ENHANCE, a phase 3, randomized, placebo-controlled study" published in April 2023 in Hepatology, a preeminent journal in the field of liver disease.
Entered into a collaboration and license agreement with Kaken Pharmaceutical Co., Ltd. in January 2023 for the development and commercialization in Japan of CymaBay’s investigational drug seladelpar for the treatment of PBC.
Financial Updates:

Completed a public equity offering in January 2023, in which we sold 11,821,428 shares of common stock at $7.00 per share and pre-funded warrants to purchase 2,142,857 shares of common stock at $6.9999 per share. Net proceeds of the offering were $92.4 million after deducting underwriting commissions and other offering expenses.

Held $236.4 million in cash, cash equivalents and investments as of March 31, 2023. We believe that cash and investments on hand are sufficient to fund CymaBay’s operating plan through the third quarter of 2024.
First Quarter Ended March 31, 2023 Financial Results

Research and development expenses for the three months ended March 31, 2023, and 2022 were $18.6 million and $18.4 million, respectively. Research and development expenses for the three months ended March 31, 2023 had a moderate increase compared to the corresponding period in 2022 primarily due to lower project costs spending on completion of enrollment of our RESPONSE trial and in drug manufacturing and development for PBC offset by higher employee compensation as we continued to hire additional personnel to support our clinical studies.

General and administrative expenses for the three months ended March 31, 2023 and 2022 were $8.3 million and $6.1 million, respectively. General and administrative expenses for the three months ended March 31, 2023 were higher than the corresponding period in 2022 as we continued to add administrative personnel and expand our infrastructure to support our corporate growth.

Net loss for the three months ended March 31, 2023 and 2022 was $28.8 million and $27.8 million, or ($0.29) and ($0.32) per share, respectively. Net loss for the three months ended March 31, 2023 was higher than the corresponding period in 2022 due primarily to an increase in general and administrative expenses, partially offset by higher interest income earned on our investments. Overall, we expect operating expenses to increase in the future as we continue to execute our development and pre-commercialization plans for seladelpar in PBC.

On May 15, 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 first quarter ended March 31, 2023 and provided an update on recent business developments (Press release, CorMedix, MAY 15, 2023, View Source [SID1234631711]).

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Recent Corporate Highlights:

● As previously guided, CorMedix is resubmitting its DefenCath New Drug Application, or NDA, to the U.S. Food and Drug Administration ("FDA"). The Company anticipates that the NDA will be classified as a Type 2 resubmission with a 6-month review cycle with a target action date in November.

● The Company also recently announced that the US Patent & Trademark office has issued a notice of allowance of patent claims directed to the composition of a catheter lock solution for preventing infection and reduced blood flow in central venous catheters. This newly allowed U.S. Patent Application reflects the unique and proprietary nature of DefenCath, and will extend our current intellectual property protection to an anticipated expiration date in 2042.

● CorMedix recently announced that following our submission of a duplicate New Technology Add-On Payment, or NTAP, application to the Centers for Medicare & Medicaid Services, CMS has issued the Inpatient Prospective Payment System 2024 proposed rule that includes an NTAP of up to $17,111 per hospital stay for DefenCath. This NTAP is conditioned upon the DefenCath NDA obtaining final FDA approval prior to July 1, 2024.

● Cash and short-term investments, excluding restricted cash, at March 31, 2023 amounted to $55.6 million.

Joe Todisco, CorMedix CEO, commented, "we are excited to announce today that our DefenCath NDA is ready to be resubmitted to FDA. The resubmission reflects the hard work of the CorMedix regulatory and technical teams and our strategic partners, and we will now increase our focus on preparations for a potential commercial launch in early 2024. We look forward to providing additional updates as we progress toward potential approval in 2023 and aim to deliver on our commitment to reducing the risk of infections in patients receiving hemodialysis via central venous catheters."

First Quarter 2023 Financial Highlights

For the first quarter of 2023, CorMedix recorded a net loss of $10.6 million, or $0.24 per share, compared with a net loss of $7.0 million, or $0.18 per share, in the first quarter of 2022, an increase of $3.5 million, driven primarily by an increase in operating expenses.

Operating expenses in the first quarter 2023 were $11.0 million, compared with $7.0 million in the first quarter of 2022, an increase of approximately 57%. The increase was driven by higher SG&A expenses which increased approximately 60% to $7.6 million, primarily driven by costs related to market research studies and pre-launch activities in preparation for the potential approval of DefenCath, and an increase in personnel expenses and non-cash charges for stock-based compensation. R&D expenses also increased by 49% to $3.4 million, primarily due to net increases in personnel expenses and non-cash charges for stock-based compensation. Additionally, there were also increases in medical affairs activities in preparation for the potential marketing approval of DefenCath and costs related to the manufacturing of DefenCath prior to its potential marketing approval.

The Company reported cash and short-term investments of $55.6 million at March 31, 2023, excluding restricted cash. The Company believes that it has sufficient resources to fund operations at least through the first half of 2024.

On May 15, 2023 Compugen Ltd. (Nasdaq: CGEN) (TASE: CGEN) a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported financial results for the first quarter ended March 31, 2023 and provided a corporate update on key events since the start of 2023 (Press release, Compugen, MAY 15, 2023, View Source [SID1234631710]).

"I am excited that we are leading the way with a unique triple combination immuno-oncology approach with potential to be the next generation of immunotherapies in a very large market with high unmet need", said Anat Cohen-Dayag, Ph.D., President, and Chief Executive Officer of Compugen. "We have already shown preliminary clinical benefit in two tumor types, typically not responding to immunotherapy, microsatellite stable colorectal cancer and platinum resistant ovarian cancer. To confirm this signal, we have initiated two proof-of-concept studies evaluating the triple blockade of PVRIG, TIGIT and PD-1 with COM701, COM902 and pembrolizumab in these indications and are on track to report initial findings by the end of the year. We believe this strategy provides the fastest route in building a path to registration and de-risk our lead assets, COM701 and COM902."

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Dr. Cohen-Dayag continued, "We continue to feed our own pipeline by leveraging our pioneering computational discovery platform. We presented new data on our lead pre-clinical asset, COM503, in an oral presentation at the recent CIMT (Free CIMT Whitepaper) annual meeting. COM503 is a differentiated, novel anti-IL-18BP antibody based approach to harness cytokine biology for anti-cancer therapeutics. By freeing the body’s own interleukin-18 to stimulate the immune system to fight cancer at the tumor site, COM503 has the potential advantage of avoiding the typical systemic toxicity associated with cytokine therapeutics".

Dr. Cohen-Dayag concluded, "We are driven by our strong belief in the potential of our differentiated assets, internal and partnered, and the opportunity to transform the lives of patients with cancer. Our $74.3 million cash balance as of March 31, 2023 re-affirms our financial discipline, with bold execution on our DNAM-1 axis hypothesis and our newly disclosed lead pre-clinical asset advancing towards IND submission next year. We have cash runway at least through the end of 2024, to support operations, reach milestones and de-risk our lead clinical assets, COM701 and COM902."

Financial Results
As of March 31, 2023, cash, cash equivalents, short-term bank deposits and restricted cash totalled approximately $74.3 million, compared with approximately $83.7 million as of December 31, 2022. The Company expects its existing cash and cash related balances to be sufficient to fund its operations at least through the end of 2024, based on its current plans.

Compugen does not have any debt.

R&D expenses for the first quarter of 2023, were approximately $7.4 million compared with approximately $7.2 million for the comparable period in 2022.

General and administrative expenses for the first quarter of 2023, were approximately $2.6 million, compared with approximately $2.6 million for the comparable period in 2022.

Net loss for the first quarter of 2023, was approximately $9.3 million, or $0.11 per basic and diluted share, compared with a net loss of approximately $9.7 million, or $0.11 per basic and diluted share, in the first quarter of 2022.

Full financial tables are included below.

Conference Call and Webcast Information
The Company will hold a conference call today, May 15, 2023, at 8:30 AM ET to review its first quarter 2023 results. To access the live conference call by telephone, please dial 1-866-744-5399 from the U.S., or +972-3-918-0644 internationally. The call will be available via live webcast through Compugen’s website, located at the following link. Following the live webcast, a replay will be available on the Company’s website.

On May 15, 2023, Coherus BioSciences, Inc. (the "Company") reported to have reduced the amount of shares that could be issued and sold pursuant to its "at-the-market" program ("ATM") with Cowen and Company, LLC ("Cowen") in an amount equal to $86.25 million (Filing, Coherus Biosciences, MAY 15, 2023, View Source [SID1234631709]). The reduction in the amount of shares that can be issued and sold under the ATM was effected pursuant to the Amendment No. 1 to Sales Agreement ("Amendment No. 1"), which amended the Company’s Sales Agreement with Cowen, dated November 8, 2022 (the "Agreement"), to reduce the aggregate offering price under the Agreement from $150.0 million to $63.75 million.

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The foregoing description of Amendment No. 1 is not a complete description thereof, and is qualified in its entirety by reference to the full text of the Amendment No. 1, which will be filed with the Securities and Exchange Commission as an exhibit to the Company’s Quarterly Report on Form 10-Q for the quarter ending June 30, 2023.