On May 16, 2022 Celularity Inc. (Nasdaq: CELU) ("Celularity" or the "Company"), a clinical-stage biotechnology company developing placental-derived off-the-shelf allogeneic cell therapies, reported financial results for the first quarter ended March 31, 2022, and provided a corporate update (Press release, Celularity, MAY 16, 2022, View Source [SID1234614639]).

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"We have continued to achieve multiple transformational milestones and make significant progress this year with three ongoing Phase 1 clinical trials of two investigational drugs, CYNK-001 and CYNK-101, both of which have been granted Fast Track and Orphan Drug Designations for certain indications," said Robert J. Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity. We expect to have data readouts for all three ongoing Phase 1 programs later this year. As such, we believe we are well-positioned to continue executing our business strategy and developing cellular therapies for cancer, infectious and degenerative diseases that leverage our proprietary placental-based technology platform."

First Quarter Clinical and Regulatory Updates

CYNK-001 for the Treatment of AML and GBM:

CYNK-001 is Celularity’s unmodified cryopreserved human placental hematopoietic stem cell-derived NK cell therapy candidate that is enriched with CD56+/CD3- NK cells and expanded from human placental CD34+ cells. CYNK-001 is currently being investigated in two Phase 1 clinical trials, in AML and in GBM, with data readouts expected in the second half of 2022.
Celularity continues to enroll new cohorts in both arms of the Phase 1 AML study with clinical trial protocol adjustments communicated in December 2021, which include an addition of interleukin-2 (IL-2) to the treatment regimen; a fourth dose on day 21; and an increase in the dose of NK cells.
CYNK-101 for the Treatment of Gastric Cancer:

CYNK-101 is a novel allogeneic off-the-shelf human placental CD34+-derived NK cell product candidate that is genetically modified to express high-affinity and cleavage-resistant CD16 (FCGRIIIA) variant to drive antibody-dependent cell-mediated cytotoxicity. CYNK-101 is currently being investigated in the Phase 1 portion of a Phase 1/2a clinical trial in advanced HER2+ gastric cancer.
In January 2022, the FDA granted Fast Track designation to CYNK-101, which is being developed in combination with standard chemotherapy, trastuzumab and pembrolizumab in first-line locally advanced unresectable or metastatic HER2/neu positive gastric/gastroesophageal (G/GEJ) adenocarcinoma.
In February 2022, the FDA granted Orphan Drug Designation to CYNK-101 for the treatment of G/GEJ cancer.
CYCART-19 for the Treatment of B-Cell Malignancies:

CYCART-19 is an allogeneic Chimeric Antigen Receptor (CAR) engineered human placental-derived T cell that is a potential drug candidate in B-cell malignancies.
Preclinical data demonstrating the feasibility and functionality of expressing a CAR directed to CD19 on placental CD34+derived, cryopreserved, off-the-shelf, allogeneic CYNK cells were presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2021.
Celularity submitted an Investigational New Drug (IND) application to FDA to investigate CYCART-19 for the treatment of B-cell malignancies in the first quarter of 2022 and in April 2022, received email notification from FDA that it will be requesting additional information before Celularity can proceed with the planned Phase 1/2 clinical trial. Celularity anticipates a formal written communication by late May 2022 and plans to work with the FDA in an effort to resolve its questions as promptly as possible and, if the IND is cleared, commence a Phase 1/2 clinical trial of CYCART-19 in B-cell malignancies in the second half of 2022.
First Quarter 2022 Financial Results

Cash and Cash Equivalents: Cash, cash equivalents and marketable securities were $48.0 million as of March 31, 2022, compared to $37.2 million as of December 31, 2021. In March 2022, we amended and restated certain warrants to reduce the exercise price per share to $3.50 per share, among other items, following which the holders exercised such warrants in full for cash for approximately $46.5 million and we issued the holders an aggregate 13,281,386 shares of our Class A common stock.
Total Revenues: Total revenues were $5.9 million for the first quarter of 2022, compared to $2.7 million for the first quarter of 2021. This increase in revenues was primarily driven by sales activity from supply and distribution agreements for Celularity’s degenerative disease products.
Research & Development (R&D) Expenses: R&D expenses were $21.7 million for the first quarter of 2022, compared to $17.0 million for the first quarter of 2021. The increase was primarily driven by higher clinical development expenses.
Selling, General & Administrative (SG&A) Expenses: SG&A expenses were $16.5 million for the first quarter of 2022, compared to $7.6 million for the first quarter of 2021. The increase in SG&A expenses was primarily caused by higher expenses related to being a public company, such as stock-based compensation, insurance and consulting fees.
Net loss: Net loss for the first quarter of 2022 was $62.9 million, or $(0.48) per share for both basic and diluted shares. Net loss for the first quarter of 2021 was $81.5 million, or $(3.40) per share for both basic and diluted shares.

On May 16, 2022 Dynavax Technologies Corporation (Nasdaq: DVAX), a commercial-stage biopharmaceutical company developing and commercializing innovative vaccines, reported that Ryan Spencer, Chief Executive Officer, will present at the H.C. Wainwright Global Investment Conference, being held May 23-26, 2022 (Press release, Dynavax Technologies, MAY 16, 2022, https://investors.dynavax.com/news-releases/news-release-details/dynavax-present-hc-wainwright-global-investment-conference [SID1234614638]).

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The on demand presentation will be available, beginning Tuesday, May 24, 2022 at 7:00 a.m. E.T. and may be accessed through the "Events & Presentations" page on the "Investors" section of the Company’s website at View Source

On May 16, 2022 Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations, reported first-quarter 2022 financial results and provided a corporate update (Press release, Ayala Pharmaceuticals, MAY 16, 2022, View Source [SID1234614637]).

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"We continued to execute on advancing our clinical programs during the first quarter of 2022," said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. "Our highest immediate priority is completing Part A of the RINGSIDE study evaluating AL102 in desmoid tumors and we are on target to announce initial interim data around mid-year. This will be followed by initiation of Part B, the randomized portion of the study, immediately thereafter. We are very encouraged by the positive feedback received from investigators in the study and by early signs of anti-tumor activity. Other important milestones expected this year include clinical updates from the ongoing ACCURACY trial of AL101 in adenoid cystic carcinoma and the planned initiation of a Phase 2 trial of AL102 in T-ALL."

First-quarter 2022 and Recent Business Highlights

Completed enrollment of Part A of the Phase 2/3 RINGSIDE study of AL102 in desmoid tumors: 42 patients have been enrolled in Part A of the RINGSIDE study, which is evaluating the safety and tolerability of AL102, as well as tumor volume by MRI at 16 weeks. Three dosing regimens of AL102 are being tested to determine the optimal dose regimen to advance forward.

Initiated "Window of Opportunity" study of AL101 in adenoid cystic carcinoma (ACC): The study, which is being conducted in collaboration with M.D. Anderson Cancer Center and the Adenoid Cystic Carcinoma (ACC) Research Foundation, is focused on determining the effects of AL101 for the treatment of ACC and other cancers. The goals of the study are to better understand the mechanism of AL101, determine the best treatment regimen and generate data for the future development strategy.
Upcoming Milestones

Initial interim data from pivotal Phase 2/3 RINGSIDE trial in desmoid tumors: Ayala expects to report an initial interim data read-out from Part A of Phase 2/3 RINGSIDE trial of AL102 in desmoid tumors around mid-2022. Part B of the study will be a double-blind placebo-controlled study enrolling up to 156 patients with progressive disease, randomized between AL102 or placebo. The study’s primary endpoint will be progression free survival with secondary endpoints including objective response rates, duration of response and patient reported quality of life measures.
Clinical data from Phase 2 ACCURACY trial of AL101 in ACC: The Phase 2 ACCURACY clinical trial is an open-label, single-arm, multi-center study to assess the clinical activity of AL101 using radiographic assessments of patients with R/M ACC demonstrating disease progression within 6 months prior to dosing. A poster at the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting is expected to feature safety, efficacy, pharmacokinetics, and pharmacodynamics data from the 6mg AL101 cohort in the trial.
Initiate Phase 2 clinical trial evaluating AL102 in T-cell acute lymphoblastic leukemia (T-ALL): Ayala plans to begin a Phase 2 clinical trial evaluating AL101 in R/R T-ALL in the second half of 2022.
First-Quarter 2022 Financial Results

Cash Position: Cash and cash equivalents were $27.4 million as of March 31, 2021, as compared to $37.3 million at December 31, 2021.

Collaboration Revenue: Collaboration revenue was $0.4 million for the first quarter of 2022, as compared to $1.0 million for the corresponding quarter in 2021.

R&D Expenses: Research and development expenses were $7.5 million for the first quarter of 2022, compared to $6.9 million for the corresponding quarter in 2021.

G&A Expenses: General and administrative expenses were $2.4 million for the first quarter of 2022, compared to $2.3 million for the first quarter of 2021.

Net Loss: Net loss was $10.0 million for the first quarter ended March 31, 2022, resulting in basic and diluted net loss per share of $0.66. This compares with a net loss of $9.6 million for the first quarter ended March 31, 2021, or basic and diluted net loss per share of $0.74.

For further details on the company’s financial results, refer to form Quarterly Report on Form 10-Q for the three months ended March 31, 2022, filed with the SEC on May 16, 2022.

On May 16, 2022 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company leveraging the power of the mitochondria and the peptides encoded in its genome to develop potential breakthrough therapeutics targeting chronic and age-related diseases, reported its financial results for the first quarter ended March 31, 2022 and highlighted recent corporate progress (Press release, CohBar, MAY 16, 2022, View Source [SID1234614636]).

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"I am optimistic about the year ahead and the opportunity we have at CohBar to develop therapies for difficult to treat, multi-factorial diseases. In 2022, we remain focused on execution and with a strong team in place, we are well positioned to advance our mitochondrial programs and deliver on our focused strategy," stated Dr. Joseph Sarret, Chief Executive Officer. "During the first quarter we made steady progress across key areas of our business. We aligned our resources to focus on three main areas – the advancement of our IPF program toward the clinic, the discovery and development of additional novel peptide families with our Mito+ platform, and securing a potential partner for CB4211."

Recent Corporate Updates and First Quarter Highlights

Expanded Patent Coverage for CB4211: The company announced today that on May 17, 2022 the United States Patent and Trademark Office will issue a patent, U.S. No. 11,332,497, covering methods of use of CB4211, CohBar’s most advanced candidate, for treating obesity. This patent is expected to be eligible for listing in the FDA Orange Book upon approval of CB4211 as a therapeutic for obesity in the United States. Additionally, the company announced progress in its international patent prosecution strategy, with the issuance of a Japanese patent covering CB4211 and related compositions, as well as a CB4211 medicine for treating NASH.
Received Extension from Nasdaq to Comply with Listing Requirements: On May 12, the company announced that Nasdaq has granted CohBar’s request for a 180-day extension, to November 7, 2022, to achieve compliance with the $1.00 bid price requirement for continued listing on Nasdaq.
Advanced CB5138-3 for Idiopathic Pulmonary Fibrosis (IPF): CohBar is advancing CB5138-3 through IND-enabling studies and is working to optimize drug delivery to increase the likelihood of success in the clinic for this hard-to-treat patient population. CohBar expects to submit an Investigational New Drug Application for this program to the US Food and Drug Administration in the second half of 2023.
Increased Investment in Mito+ Platform: CohBar is investing further resources in its novel platform to discover and develop additional peptide families and identify new product candidates. CohBar’s scientific research team is mining the mitochondrial genome to identify the peptide families that show the most promise to treat multi-factorial diseases involving inflammation, fibrosis and/or metabolic dysregulation.
Strengthened Leadership with Appointment of Nick Vlahakis, MBBS, as Acting Chief Medical Officer: In March, CohBar announced the appointment of Nick Vlahakis, MBBS as acting Chief Medical Officer. Dr. Vlahakis is an experienced pulmonary and critical care clinician and clinical drug developer, having led molecule development strategy and early and late-stage trials in both large and small companies across a wide range of therapeutic areas, including IPF.
First Quarter 2022 Financial Highlights

Cash and Investments: The company had cash and investments of $23.5 million as of March 31, 2022, compared to $26.2 million as of December 31, 2021. The cash burn for the quarter ended March 31, 2022 was approximately $3.1 million.
R&D Expenses: Research and development expenses were $1.5 million for the three months ended March 31, 2022, compared to $2.7 million in the prior year quarter. The decrease in research and development expenses was primarily due to lower preclinical and clinical trial costs primarily due to the timing of those expenses.
G&A Expenses: General and administrative expenses were $1.7 million for the three months ended March 31, 2022, compared to $1.4 million in the prior year quarter. The increase in general and administrative expenses was primarily due to higher stock-based compensation costs and legal fees associated with protecting the company’s intellectual property portfolio.
Net Loss: For the three months ended March 31, 2022, net loss, which included $0.5 million of non-cash expenses, was $3.3 million, or $0.04 per basic and diluted share. For the three months ended March 31, 2021, net loss, which included $0.4 million of non-cash expenses, was $4.0 million, or $0.07 per basic and diluted share.
Details for the Conference Call:

A simultaneous webcast of the call will be accessible via the Investors section of the CohBar website at www.cohbar.com.
For individuals participating in the Investor Call or webcast, please call or login to the conference audio approximately 10 minutes prior to its start.

An audio replay of the call will be available beginning at 8:00 p.m. Eastern Time on May 16, 2022, through 11:59 p.m. Eastern Time on June 6, 2022. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID No. 13728737. The audio recording will also be available at www.cohbar.com during the same period.

On May 16, 2022 TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to defeating cancer using RNA therapeutics, reported financial results for the first quarter ended March 31, 2022, and recent business progress (Press release, TransCode Therapeutics, MAY 16, 2022, View Source [SID1234614635]).

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TransCode co-founder and Chief Technology Officer, Dr. Zdravka Medarova, indicated, "We believe we remain on track to submit an exploratory Investigational New Drug Application (eIND) this year to test our lead therapeutic candidate, TTX-MC138, in a Phase 0 study in cancer patients with advanced solid tumors. We believe this study has the potential to establish proof-of-mechanism for our platform which ultimately could help us build a broad and diverse pipeline of therapeutics and diagnostics that have the potential to reach previously undruggable genetic targets."

"Additionally, we believe we achieved the first milestone related to our Small Business Innovation Research (SBIR) grant during the first quarter and anticipate receiving the next tranche of award funding during the second quarter," said Michael Dudley, co-founder, president and CEO of TransCode. "This non-dilutive funding, combined with capital from our 2021 IPO, provide the resources to drive continued progress across the organization, including key additions to our team who bring valuable expertise, and continued advancement of our lead therapeutic candidate and preclinical development of our other therapeutic candidates. As we move toward the Phase 0 proof-of-mechanism clinical trial, we continue to focus on using the power and versatility of our TTX platform to solve the challenges of RNA delivery in oncology."

Other First Quarter 2022 Highlights

Expanded global RNA oncology patent portfolio with filing of an International PCT Application (PCT/US21/65580) entitled TEMPLATE DIRECTED IMMUNOMODULATION FOR CANCER THERAPY (the ‘580 application). The ‘580 application, expected to be published in June 2022, represents an extension of TransCode’s use of its patented RNA therapeutic platform to include using pattern recognition receptors (PRR) to target tumor cells by activating the RIG-I signaling pathway. Once inside a cell, the selected PRR-activating oligonucleotide encounters a microRNA specific to that tumor where it is expected to activate a type I interferon-driven immune response, leading to programmed tumor cell death.
Published article in the journal "Cancers" titled, Clinical Applications of Short Non-Coding RNA-Based Therapies in the Era of Precision Medicine, in collaboration with teams from Massachusetts General Hospital (MGH), Michigan State University, and Northeastern University. The article describes the development, challenges, and clinical successes of short non-coding RNA-based drugs and details several examples of how these RNA drugs are designed, chemically modified, and delivered to treat cancer, cardiovascular disease, and rare genetic disorders. In addition, the article highlights key similarities and differences between various short non-coding RNA platforms and discusses considerations to maximize treatment efficacy of RNA-based therapeutics. TransCode Co-Founder and scientific advisor, Dr. Anna Moore, was a contributing author to the article. The article was published on March 21, 2022.
Planned Milestones

TransCode’s goals to continue to advance its portfolio include:

TTX-MC138
Submission to FDA of an eIND application for its First-in-Human (FIH) clinical trial.
Completion of a FIH Phase 0 clinical study intended to demonstrate quantifiable evidence of delivery of radiolabeled TTX-MC138 to metastatic lesions in advanced solid tumors; measure pharmacokinetics and biodistribution in vital organs and other tissues; potentially inform therapeutic dose levels based on microdose results; and validate delivery for the TTX pipeline more broadly, potentially opening-up additional relevant RNA targets that have been previously undruggable due to challenges with RNA delivery.
Concurrent completion of IND-enabling studies to support filing an IND application for a Phase I clinical trial of TTX-MC138.
Publication of preclinical results supporting the lead therapeutic candidate, TTX-MC138, in pancreatic cancer and glioblastoma multiforme.
Publication of preclinical results supporting therapeutic candidate, TTX-RIGA.
Continuation of preclinical studies for therapeutic candidates TTX-RIGA, TTX-siPDL1, and TTX-siLIN28B.
Continuation of discussions regarding potential partnerships.
File for Orphan Drug Designation for its lead therapeutic candidate in additional tumor indications.
First Quarter 2022 Financial Highlights

Cash and cash equivalents were $16.9 million at March 31, 2022, compared to $20.8 million at December 31, 2021.
Research and development expense was $1.9 million in the first quarter of 2022, compared to $0.3 million in the first quarter of 2021.
General and administrative expense was $1.6 million in the first quarter of 2022, compared to $0.2 million in the first quarter of 2021.
Operating loss for the three months ended March 31, 2022, was $3.5 million, compared to an operating loss of $0.4 million in the prior year period.
Financial Guidance

TransCode expects that its cash of $16.9 million as of March 31, 2022, together with additional funding expected from the April 2021 SBIR award, are sufficient to fund planned operations into the first quarter 2023 but not for a full 12 months from the date of its financial statements.