On April 22, 2024 Cidara Therapeutics, Inc. (Nasdaq: CDTX) (the Company), a biotechnology company using its proprietary Cloudbreak platform to develop drug-Fc conjugate (DFC) immunotherapies designed to save lives and improve the standard of care for patients facing serious diseases, reported financial results for the fourth quarter and full year ended December 31, 2023 and provided an update on its corporate activities and product pipeline (Press release, Cidara Therapeutics, APR 22, 2024, View Source [SID1234642192]).

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"2023 included significant accomplishments throughout our business within both our Cloudbreak drug-Fc conjugate (DFC) and our REZZAYO (rezafungin) programs," said Jeffrey Stein, Ph.D., president and chief executive officer of Cidara. "With respect to our DFC platform, we continue to generate and present compelling data from our oncology programs as well as our CD388 influenza program partnered with Janssen. We have multiple key catalysts expected this year, including the filing of an Investigational New Drug Application (IND) in mid-2024 for CBO421, a potential best-in-class inhibitor of CD73. We recently presented promising new data on CD73/PD-1 multispecific DFC, CCR5-targeting DFC and CBO421 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting."

Dr. Stein continued, "Most recently, REZZAYO was approved in the European Union (EU) and United Kingdom (UK) for the treatment of invasive candidiasis in adults, earning milestone payments of approximately $11.1 million and $2.8 million, respectively. In addition, enrollment was completed in the Phase 3 ReSTORE trial in China evaluating the efficacy and safety of rezafungin as a treatment for candidemia and invasive candidiasis, bringing us one step closer to making this important drug available to an even broader global patient population."

Recent Corporate Highlights

Presented at AACR (Free AACR Whitepaper) Annual Meeting 2024: In April 2024, Cidara presented promising new data on novel Cloudbreak DFC candidates at the AACR (Free AACR Whitepaper) Annual Meeting. The Company delivered four poster presentations highlighting the data on the Company’s multispecific CD73/PD-1 DFC, its CCR5-targeting DFC, and CBO421, its lead oncology DFC candidate targeting CD73.
Presented at IDWeek 2023: In October 2023, Cidara presented new preclinical and clinical data on Novel Cloudbreak Influenza Drug-Fc Conjugate CD388 at IDWeek 2023.
Presented at ESMO (Free ESMO Whitepaper) Immuno-Oncology Annual Congress: In December 2023, Cidara presented new preclinical data on its novel Cloudbreak, multi-specific CD73/PD-1 targeting DFC candidate at the ESMO (Free ESMO Whitepaper) Immuno-Oncology (IO) Annual Congress. The Company also presented new preclinical data on Cloudbreak CBO421, its first-in-class CD73-targeting DFC.
Presented at SITC (Free SITC Whitepaper)’s 38th Annual Meeting: In November 2023, Cidara presented new preclinical data on Cloudbreak CBO421 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 38th Annual Meeting.
Received EU and UK approval for REZZAYO for the treatment of invasive candidiasis in adults: In December 2023 and January 2024, Cidara and Mundipharma announced REZZAYO had been approved in the EU and UK, respectively, for the treatment of invasive candidiasis in adults. Cidara received a milestone payment of approximately $11.1 million from Mundipharma in February 2024 for the EU approval of rezafungin and received a milestone payment of approximately $2.8 million from Mundipharma in April 2024 for the UK approval of rezafungin, in accordance with the terms of the Collaboration and License Agreement, dated September 3, 2019, by and between the Company and Mundipharma.
Completed enrollment in the Phase 3 ReSTORE trial of rezafungin in China: In December 2023, Cidara and Mundipharma announced the completion of enrollment in the Phase 3 ReSTORE trial in China evaluating the efficacy and safety of rezafungin as a treatment for candidemia and invasive candidiasis. The portion of the trial conducted in China included 52 patients diagnosed with candidemia and/or invasive candidiasis. ReSTORE (NCT03667690) is a global, randomized, double-blind, controlled Phase 3 pivotal clinical trial evaluating the efficacy and safety of once-weekly intravenous dosing of rezafungin compared to once-daily dosing of caspofungin, the current standard of care, to treat patients with candidemia and/or invasive candidiasis. Data from this study are expected in the second quarter of 2024.
Named San Diego Metro Area Top Workplace: In November 2023, Cidara was named a Top Workplace by The San Diego Union-Tribune for the sixth year, ranking among the top 100 companies.
Fourth Quarter and Full Year 2023 Financial Results

Revenue totaled $17.6 million and $63.9 million for the three months and full year ended December 31, 2023, respectively, compared with $10.2 million and $64.4 million for the same periods in 2022.
Revenue for the year ended December 31, 2023 related to the achievement of milestones and ongoing research and development and clinical supply services provided to Mundipharma, Janssen and Melinta of $16.4 million, $23.3 million and $19.7 million, respectively, $0.2 million in royalty revenue recognized following initiation of the commercial launch of REZZAYO in the U.S. on July 31, 2023, as well as product revenue of $4.3 million related to shipments of REZZAYO naked vials to Melinta and Mundipharma.

Revenue for the year ended December 31, 2022 included $25.9 million of revenue recognized upon transfer of an intellectual property license to Melinta in August 2022. The remaining revenue for the year ended December 31, 2022 related to the achievement of milestones and ongoing research and development and clinical supply services provided to Mundipharma, Janssen and Melinta of $14.3 million, $23.5 million and $0.8 million, respectively.

Cash and cash equivalents totaled $35.8 million as of December 31, 2023, compared with $32.7 million as of December 31, 2022.
As of December 31, 2023, Cidara had 90,601,999 shares of common stock outstanding, and 2,104,472 shares of Series X Convertible Preferred Stock outstanding, which are convertible into 21,044,720 shares of common stock.
Cost of product revenue was $1.5 million for the year ended December 31, 2023 and primarily consisted of direct material costs, third-party manufacturing costs and indirect overhead costs associated with the manufacture, quality assessment and delivery of REZZAYO naked vials shipped to Melinta and Mundipharma. Prior to regulatory approval, all direct and indirect manufacturing costs were charged to research and development expense in the period incurred.
Research and development expenses were $14.7 million and $68.5 million for the three months and full year ended December 31, 2023, respectively, compared to $20.1 million and $77.4 million for the same periods in 2022. The decrease in research and development expenses for the full year ended December 31, 2023 compared to the full year ended December 31, 2022 is primarily due to lower clinical expenses associated with the rezafungin clinical trials and lower consulting and personnel costs, offset by higher clinical expenses associated with our Cloudbreak platform.
Selling, general and administrative (SG&A) expenses were $5.4 million and $18.3 million for the three months and full year ended December 31, 2023, respectively, compared to $3.6 million and $20.5 million for the same periods in 2022. The decrease in SG&A expenses for the full year ended December 31, 2023 compared to the full year ended December 31, 2022 is primarily due to lower consulting, personnel and legal costs, and lower amortization of contract costs related to obtaining the Melinta License Agreement, offset by higher selling expenses related to REZZAYO.
Net loss for the three months ended December 31, 2023 was $3.2 million, compared to a net loss of $13.6 million for the three months ended December 31, 2022. Net loss for the full year ended December 31, 2023 was $22.9 million, compared to a net loss of $33.6 million for the year ended December 31, 2022.
Restatement of Consolidated Financial Statements

The financial results included herein and the financial statements included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2023 (the Form 10-K) have been restated to account for indirect taxes that led to understatements of accrued liabilities and operating expenses, as more fully described in our Current Report on Form 8-K filed with the United States (U.S.) Securities and Exchange Commission (the SEC) on April 16, 2024 and in the Form 10-K.

On April 22, 2024 Celularity Inc. (NASDAQ: CELU) ("Celularity"), a regenerative and cellular medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, reported that it will host an on-site and virtual Investor and Analyst Research & Development (R&D) Day (Press release, Celularity, APR 22, 2024, View Source [SID1234642191]). The Investor and R&D Day, scheduled for Tuesday, May 21, 2024, from 12:00 p.m. to 4:00 p.m. ET in Florham Park, NJ, will showcase progress and advancements of Celularity’s placental technology platform and clinical development pipeline and future growth expectations for its commercial advanced biomaterials business. The event will feature members of Celularity’s executive management team, including:

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Celularity Executive Management:

Robert J. Hariri, M.D., Ph.D., – Celularity Chairman, CEO and Founder
Stephen Brigido, DPM – President, Degenerative Diseases
Adrian Kilcoyne M.D., M.P.H., M.B.A.- Chief Medical Officer
Ramji Krishnan, Ph.D.-Chief Technology Officer
A live webcast of the Investor and Analyst R&D Day can be accessed by visiting the Investor Relations section of Celularity’s website at www.celularity.com. A replay will also be available for 30 days following the event. An invited panel of expert medical speakers will provide insights into the technology and programs as well.

"We are very excited to host our first on-site and virtual Investor and Analyst R&D Day at our world-class headquarters, research, and manufacturing center. We will showcase the capabilities resident at Celularity and share the progress made in our development programs and commercial business. Our unique bifurcated regenerative and cellular medicine business model, which leverages both clinical and commercial stage technologies, will be presented and toured during the day," added Celularity CEO and Founder. Dr. Robert J. Hariri.

On April 22, 2024 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that it will present first data exploring novel TALEN editing processes in hematopoietic stem and progenitor cells (HSPCs) at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) being held on May 7-11, 2024 (Press release, Cellectis, APR 22, 2024, View Source [SID1234642190]).

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"These two posters showcase the potential and versatility of the TALEN technology to promote efficient gene insertion in HSPCs. We show that circular single strand DNA templates can be efficiently delivered to HSPCs and enable unprecedented efficiency of gene insertion without compromising the viability, fitness and differentiation capacity of edited cells" commented Julien Valton, Ph.D., Vice President of Gene Therapy at Cellectis.

"We also illustrate a novel TALEN mediated-DNA template insertion approach that rewires the natural ability of myeloid cells to cross the blood brain barrier to efficiently vectorize a genetically encoded-therapeutic protein to the brain. This approach is, by essence, versatile and could be used to vectorize an array of therapeutic proteins to the brain and potentially address multiple neurological disorders."

Poster presentation: Intron Editing of HSPC Enables Lineage-Specific Expression of Therapeutics

Gene therapy using edited hematopoietic and progenitor stem cells (HSPCs) has the potential to provide a lifelong supply of genetically encoded therapeutics.

Today, most therapies are impacted with the difficulty to cross the blood-brain barrier (BBB). The BBB is a continuous endothelial membrane that, along with pericytes and other components of the neurovascular unit, limits the entry of toxins, pathogens, protein and small molecules to the brain.

Cellectis has developed a TALEN mediated promoter-less intron editing technology that enables the expression of a therapeutic transgene exclusively by monocyte derived from edited HSPCs.

The edited cells containing genetically encoded therapeutic proteins have the capacity to cross the blood-brain barrier and secrete the corresponding therapeutic within the brain.

This novel editing approach is an important addition to the HSPC gene editing toolbox that might unlock new strategies for the treatment of metabolic and neurological diseases.

Research data showed that:

o Intron editing can be performed within B-cell, T-cell, Monocyte-specific endogenous genes (CD20, CD4 and CD11b, respectively)

o Intron editing allows expression of transgenes in a lineage-specific manner without markedly impacting the expression of the endogenous gene targeted

o Editing of CD11b intron using a therapeutic transgene encoding IDUA (the enzyme missing in Type-1 Mucopolysaccharidosis patients) enables to restrict the expression of IDUA to the myeloid lineage.

o Edited HSPCs efficiently engraft in the bone-marrow of immunodeficient mice and differentiate into edited myeloid cells that can cross the BBB and populate the brain.

o The intron editing strategy described in this work is versatile and could be potentially used to vectorize multiple genetically encoded-therapeutic proteins to the brain and thus address multiple metabolic and neurological disorders.

Title: Intron Editing of HSPC Enables Lineage-Specific Expression of Therapeutics

Presenter: Julien Valton, Ph.D., Vice President Gene Therapy at Cellectis

Session Date/Time: May 5, 2024 at 12PM ET
Session Title: Gene Targeting and Gene Correction New Technologies
Presentation Room: Exhibit Hall
Final Abstract Number: 721

Poster presentation: Circularization of Non-Viral Single-Strand DNA Template for Gene Correction and Gene Insertion Improves Editing Outcomes in HSPCs

Today, most of the gene insertion approaches used to edit HSPCs ex vivo are hampered by the low efficiency of DNA template delivery into their nucleus.

Cellectis has developed and optimized a novel gene editing process, leveraging the TALEN technology and circular single strand DNA template delivery, enabling highly efficient gene insertion in HSPCs.

Research data showed that:

o Non-viral single strand DNA delivery associated to TALEN technology allows gene insertion in long-term repopulating hematopoietic stem cells

o Circularization of the single strand DNA further increases the rates of gene insertion without impacting cellular viability and fitness of HSPCs, facilitating the development of next generation of ex vivo cell therapies

Title: Circularization of Non-Viral Single-Strand DNA Template for Gene Correction and Gene Insertion Improves Editing Outcomes in HSPCs

Presenter: Alex Boyne, Gene Editing Platform Manager at Cellectis

Session Date/Time: May 9, 2024 at 12PM ET
Session Title: Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates
Presentation Room: Exhibit Hall
Final Abstract Number: 1235

Full abstracts and poster presentations will be available on Cellectis’ website following the event: View Source

On April 22, 2024 Bristol Myers Squibb (NYSE: BMY) and Cellares, the first Integrated Development and Manufacturing Organization (IDMO) dedicated to clinical and industrial-scale cell therapy manufacturing, reported a worldwide capacity reservation and supply agreement for the manufacture of CAR T cell therapies in a transaction valued up to $380M in upfront and milestone payments (Press release, Bristol-Myers Squibb, APR 22, 2024, View Source [SID1234642189]). As part of the agreement, Cellares will optimize, automate, and tech-transfer select Bristol Myers Squibb CAR T cell therapies onto its automated and high-throughput manufacturing platform, the Cell Shuttle. Cellares will dedicate multiple Cell Shuttle and Cell Q systems with fully automated, high-throughput quality control for Bristol Myers Squibb’s exclusive use. The Cell Shuttles and Cell Qs will be deployed in Cellares’ Smart Factories in the U.S., EU, and Japan.

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Manufacturing cell therapies is both operationally and technically complex. Because cell therapies are rapidly transforming the way many different diseases are treated, the demand for these treatments is increasing significantly. This collaboration enables Bristol Myers Squibb to expand its manufacturing capacity, meeting the growing demand for its diverse range of cell therapies through a platform that is scalable and has the potential to improve turnaround time, bringing the promise of cell therapies to more patients faster.

"The agreement with Cellares is our latest step forward in support of our comprehensive strategy to unlock the full potential of CAR T therapy to deliver transformative treatments to as many patients as possible, as quickly as possible," said Lynelle B. Hoch, president, Cell Therapy Organization, Bristol Myers Squibb. "Our collaboration with Cellares strengthens our existing internal manufacturing capabilities for CAR T cell therapies by giving us access to the first end-to-end fully automated cell therapy manufacturing platform, to help ensure we meet the high demand for these differentiated treatments, now and in the future."

This agreement expands upon the existing collaborations between Bristol Myers Squibb and Cellares. In August 2023, Bristol Myers Squibb participated in Cellares’ Series C financing to launch the first IDMO Smart Factory in an effort to meet the demand for cell therapies globally. That same month, Bristol Myers Squibb joined Cellares’ Technology Adoption Partnership (TAP) Program to evaluate the Cell Shuttle’s automated manufacturing capabilities.

"This agreement with Bristol Myers Squibb is aligned with our strategy of establishing a global network of high-throughput, automated Smart Factories to meet the growing and worldwide demand for cell therapies," said Fabian Gerlinghaus, CEO and co-founder of Cellares. "We look forward to demonstrating how our innovative technology’s emphasis on standardization will accelerate commercial-scale manufacturing and worldwide deployment. Our collaboration with Bristol Myers Squibb and our collective expertise furthers our mission to accelerate access to life-saving cell therapies for patients globally."

On April 22, 2024 Azitra, Inc. (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision dermatology, reported preclinical data from the Company’s platform and pipeline (Press release, Azitra, APR 22, 2024, View Source [SID1234642188]). The data will be presented on Friday, May 10, 2024, in two oral sessions entitled "Engineered Staphylococcus Epidermidis as a Protein Delivery System for Treating Skin Diseases" and "Staphylococcus epidermidis Strain Expressing LEKTI-D6 (ATR12-351) for Netherton Syndrome."

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"We are thrilled to announce new preclinical data for our precision dermatology platform that demonstrate proof-of-concept data supporting the use of genetically engineered skin commensals to deliver proteins to the skin," said Travis Whitfill, Azitra’s co-founder and COO. "These data show the robust preclinical activity of ATR-12 in Netherton syndrome models and further supports the rationale behind our Phase 1b clinical trial in Netherton syndrome patients."

ATR-12 is an engineered strain of S. epidermidis that expresses a fragment of human lympho-epithelial Kazal-type-related inhibitor (LEKTI) protein, which is missing in patients with Netherton syndrome, a chronic and sometimes fatal disease of the skin estimated to affect approximately one to nine in every 100,000. ATR-12 has been engineered to deliver missing LEKTI protein when applied topically to Netherton syndrome patients. Azitra has an open IND for a Phase 1b clinical trial in adult patients (NCT06137157).

The data in the abstracts released online today show that topical application of ATR-12 in preclinical models reduced produced reduced IL-36γ by 93% compared to skin extracts induced to overexpress IL-36γ. Additionally, topical application of ATR-12 significantly reduced protease activity in skin samples compared to a Netherton syndrome model skin (p<0.01). Finally, ATR-12 produced higher amounts of LEKTI compared to topical application of LEKTI protein alone (6.0 µg vs. 2.3 µg, respectively, p<0.01) after 24 hours and resulted in deeper skin penetration of LEKTI.