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.

On May 16, 2022 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported the appointment of Phuong Khanh (P.K.) Morrow, M.D., FACP, as Chief Medical Officer, effective May 23, 2022 (Press release, CRISPR Therapeutics, MAY 16, 2022, View Source [SID1234614634]). Dr. Morrow brings more than a decade of leadership experience in global drug development and joins CRISPR Therapeutics to lead the Company’s global clinical development and regulatory operations.

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"P.K.’s leadership experience, deep expertise in oncology drug development, and her track record in bringing novel medicines to patients will be invaluable as we continue to advance our broad portfolio of innovative gene-edited therapies," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "She will play an integral role in shaping our clinical development strategy, and in building and strengthening our organization, and we look forward to her contributions as we continue on our mission to transform medicine."

"I am excited to join CRISPR Therapeutics’ leadership team at this important time in its growth and evolution," said Dr. Phuong Khanh (P.K.) Morrow. "The potential of the Company’s pre-eminent gene editing platform combined with the broad pipeline creates a tremendous opportunity to bring several novel and potentially transformative therapies to patients in need."

During her biopharmaceutical career, Dr. Morrow has demonstrated outstanding leadership in bringing novel medicines through all phases of clinical development and global regulatory approval. Specializing in the therapeutic areas of oncology and hematology, she has been responsible for end-to-end development of numerous drug candidates and for the implementation of strategic partnerships with academic institutions, key opinion leaders and biopharmaceutical co-collaborators to facilitate the successful execution of clinical trials. Dr. Morrow most recently served as Vice President and Global Therapeutic Area Head of Hematology, GI Oncology, GU Oncology, and Bone at Amgen, where she was responsible for guiding and accelerating late development activities addressing marketed hematology programs, Blincyto and Kyprolis, and guiding the late development strategy for programs that focus upon FLT3 and MCL-1. She also led the medical launch activities for Imlygic, Kyprolis, Neulasta Onpro and Blincyto; served as the Global Product General Manager for three early-stage oncology molecules focused upon MCL-1 and KRAS G12C; and led a cross-functional team in the development and registration of Neulasta Onpro and the successful submission of the Neupogen and Neulasta Acute Radiation Syndrome (ARS) sBLAs, leading to the regulatory approval of both products for the ARS indication. In addition, Dr. Morrow was appointed by the U.S. Food and Drug Administration (FDA) to be the industry representative to the Oncology Drug Advisory Committee (ODAC) for a four-year term, ending in 2019.

Previously, Dr. Morrow was Assistant Professor, Department of Breast Medical Oncology at the University of Texas MD Anderson Cancer Center. She co-led the development of the first multidisciplinary breast cancer survivorship clinic at MD Anderson and served as the principal investigator of multiple drug studies. Dr. Morrow received an M.D. from the University of Texas Medical School at Houston, with honors, and completed her Internal Medicine Residency at Baylor College of Medicine and Hematology/Oncology Fellowship at the University of Texas MD Anderson Cancer Center, where she also served as a Chief Fellow. She received a B.S. in Pharmacy from the University of Houston.

On May 16, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to creating novel solutions that treat urothelial and specialty cancers, reported that highlights new data on real-world experience utilizing the antegrade approach via nephrostomy tube for administration of JELMYTO (mitomycin) for pyelocalyceal solution (Press release, UroGen Pharma, MAY 16, 2022, View Source [SID1234614633]). This data adds to a growing body of evidence on the safety and efficacy profile of the antegrade method of administration for JELMYTO. These data were presented during a podium presentation at the 2022 American Urological Association (AUA) annual meeting in New Orleans, Louisiana.

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"JELMYTO is efficacious as a chemoablative agent in adult patients with low grade upper tract urothelial cancer, and while it’s FDA approved for both antegrade and retrograde administration, prior reports are limited to the retrograde experience," said Kyle Rose, MD, Urologic Oncology Fellow at Moffitt Cancer Center in Tampa, Fla., and study investigator. "These data provide additional evidence that instillation via a nephrostomy tube is an effective instillation method with a safety profile that offers an encouraging option to appropriate patients."

Dr. Rose presented the abstract Antegrade Administration of Reverse Thermal Mytomycin Gel for Primary Chemoablation of Upper Tract Carcinoma via Percutaneous Nephrostomy Tube: A Multi-Institutional Real-World Experience (Abstract PD58-06) during a podium presentation at the AUA annual meeting on Monday, May 16.

"All 71 patients in the Phase 3 OLYMPUS trial utilized the retrograde approach to administer JELMYTO, therefore we are pleased to see real-world evidence that supports the utilization of the antegrade approach giving physicians and patients more options to administer JELMYTO based on their preference and experience," said Mark Schoenberg, MD, Chief Medical Officer, UroGen.

About This Study

The real-world data from this retrospective analysis was pooled from Moffitt Cancer Center, Tampa, FL; University of Missouri School of Medicine, Columbia, MO; The University of Texas MD Anderson Cancer Center, Houston, TX; and Mayo Clinic, Rochester, MN.

Twenty-six patients received nephrostomy tube administration of JELMYTO, six patients (23%) had solitary kidneys. Nine patients (35%) went on to receive at least one dose of maintenance therapy. Ureteral stenosis occurred in four patients (15%). Other adverse events included fatigue (27%), flank pain (19%), urinary tract infection (12%), sepsis (8%) and hematuria (8%). No patients had impaired renal function during follow-up and no deaths occurred.

Thirteen patients (50%) exhibited a complete response at post-induction ureteroscopy while 12 patients (46%) had a partial response. One patient experienced progression to invasive disease and required a nephroureterectomy. At a median follow-up of seven months (IQR 3-9) post-induction, no patients who experienced a complete response recurred.

The limitations of this study include the retrospective nature, small sample size, and pooled reporting of results. There is a need for larger studies with longer follow-up to study more conclusively any potential advantages of antegrade JELMYTO administration when compared to retrograde instillation.

About the Pivotal OLYMPUS Study

OLYMPUS (Optimized DeLiverY of Mitomycin for Primary UTUC Study) was an open-label, single-arm Phase 3 clinical study of UGN-101 JELMYTO (mitomycin) for pyelocalyceal solution, to evaluate the safety, tolerability and tumor ablative effect of JELMYTO in patients with low-grade Upper Tract Urothelial Cancer UTUC (LG UTUC). Seventy-one patients were treated at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of JELMYTO administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a Primary Disease Evaluation (PDE) to determine Complete Response (CR), the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer and for cause biopsy. Patients who achieved a CR at the PDE timepoint were eligible for the maintenance phase of the trial, during which they could receive monthly maintenance instillations for up to 12 months and were assessed to determine the durability of response with JELMYTO.

In the OLYMPUS study, data was generated for the retrograde administration of JELMYTO. In that study population ureteric obstruction was reported in 58% (n=41) of patients receiving JELMYTO, including 17% (n=12) of patients who experienced Grade 3 obstruction.

About LG UTUC

LG UTUC is a rare disease managed by endoscopic methods and radical nephroureterectomy. Endoscopic resection and laser ablation attempt to preserve the kidney, though there is a high risk of recurrence that may eventually necessitate removal of the kidney. Although kidney removal is the gold standard for treatment of high-grade UTUC, it may be over-treatment in LG UTUC, as kidney removal offers similar five-year survival as kidney-sparing procedures but is associated with significant morbidity. JELMYTO is efficacious as a primary chemoablative therapy in patients with LG UTUC.

About JELMYTO

JELMYTO (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel indicated for primary chemoablative treatment of LG UTUC in adults. It is recommended for primary treatment of biopsy-proven LG UTUC in patients deemed appropriate candidates for renal-sparing therapy. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

APPROVED USE FOR JELMYTO

JELMYTO is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO.
Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose.

Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.

are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
Tell your healthcare provider if you take water pills (diuretic).
How will I receive JELMYTO?

Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
JELMYTO is given to your kidney through a tube called a catheter.
During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.
After receiving JELMYTO:

JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.
JELMYTO may cause serious side effects, including:

Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.

You are encouraged to report negative side effects of prescription drugs to the U.S. Food and Drug Administration. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

On May 16, 2022 Eli Lilly and Company (NYSE: LLY) reported that it will attend the UBS Global Healthcare Conference on Tuesday, May 24, 2022 (Press release, Eli Lilly, MAY 16, 2022, View Source [SID1234614632]). Michael Mason, senior vice president, president of Lilly Diabetes, will participate in a fireside chat at 8:30 a.m., Eastern time.

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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.