On February 10, 2022 Indapta Therapeutics, Inc., a privately held biotechnology company developing an NK (natural killer) cell therapy platform for the treatment of blood and solid tumor cancers, reported it has appointed Mark W. Frohlich, M.D., chief executive officer and raised over $50 million in Series A financing and commitments co-led by RA Capital Management, LP, Vertex Ventures HC and Leaps by Bayer, the impact investment arm of Bayer AG (Press release, Indapta Therapeutics, FEB 10, 2022, View Source [SID1234607992]). The Myeloma Investment Fund, the venture philanthropy subsidiary of the Multiple Myeloma Research Foundation, and Lonza also participated in the round. In connection with the financing, Laura Stoppel, Ph.D., principal at RA Capital Management, LP, Lori Hu, managing director at Vertex Ventures HC, and Fabio Pucci, Ph.D., senior director of venture investments health at Leaps by Bayer, joined Indapta’s board of directors.

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Dr. Frohlich, who also joined the board of directors, succeeds founding chief executive officer and co-founder Guy DiPierro, who has taken on the position of chief strategy officer.

The company will use the proceeds of the financing to grow the team and continue to advance its universal, allogeneic NK cell platform toward an Investigational New Drug Application (IND) and clinical trials. The company’s platform involves the isolation and expansion of a subset of NK cells known as G-NK cells, which have increased potency relative to conventional NK cells. Because the G-NK cells have very active antibody-dependent cellular cytotoxicity, they have the potential to provide substantial clinical benefit to patients when used in combination with monoclonal antibodies. Indapta’s G-NK cells are isolated from healthy donors and do not require genetic engineering, so they can serve as an off-the-shelf treatment that can overcome the cost and inconsistency of autologous T-cell therapies.

Dr. Frohlich has more than 25 years of experience developing cellular immunotherapies to treat cancer. He previously served as executive vice president of portfolio strategy at Juno Therapeutics, which developed the FDA-approved CAR T-cell therapy Breyanzi for large B-cell lymphoma, until the company was sold to Celgene, now part of Bristol Myers Squibb. Before Juno, Dr. Frohlich was chief medical officer and executive vice president at Dendreon Corporation, a pioneer in the development of cellular immunotherapy. At Dendreon, he led the clinical team responsible for the development and U.S. and European regulatory approval of PROVENGE, one of the first commercially available cancer immunotherapies.

"Mark is perfectly suited to lead Indapta as we move our unique G-NK cell therapy closer to clinical trials in patients with multiple myeloma and lymphoma," said Ronald Martell, co-founder and chairman of the board. "His biopharma management pedigree, successful drug development track record and scientific acumen, together with our Series A financing from such an experienced syndicate of investors, will accelerate our ability to bring this innovative therapy to patients. We look forward to Guy’s continued contributions as he transitions to the role of chief strategy officer."

Before joining Dendreon, Dr. Frohlich served as vice president and medical director at Xcyte Therapies, where he led the clinical development program for an autologous activated T-cell therapy to treat cancer. Earlier in his career, he was an assistant adjunct professor of hematology/oncology at the University of California, San Francisco, where he specialized in urologic oncology and conducted laboratory, translational and clinical research. Dr. Frohlich earned a B.S. in electrical engineering and economics from Yale College and an M.D. from Harvard Medical School. At UCSF, he served as chief resident in medicine and completed a fellowship in hematology/oncology and a Howard Hughes postdoctoral fellowship. Dr. Frohlich has served as a strategic advisor to several early-stage immuno-oncology and cell therapy companies and currently serves on the boards of NEUVOGEN and Bioeclipse Therapies.

"I joined Indapta because I believe its NK cell platform is truly differentiated and its preclinical data is particularly compelling. I’m excited to bring this off-the-shelf cell therapy to the clinic, where we have the potential to demonstrate it can benefit patients without the toxicities associated with currently approved cell therapies," said Dr. Frohlich. "I look forward to applying everything I’ve learned over the past two decades to develop this novel therapy."

SVB Leerink acted as Indapta’s exclusive financial advisor for the Series A financing.

This press release does not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

About Indapta’s G-NK Cell Therapy

Indapta Therapeutics is developing a universal, allogeneic G-NK cell therapy designed to substantially improve the cytotoxicity of monoclonal antibody (mAb) therapy in multiple cancers. G-NK cells are a specific and potent subset of NK (natural killer) cells with specialized anti-tumor activity resulting from an epigenetic change rather than engineering. Indapta has further enhanced G-NK cells via specific G-NK cell subset selection and its proprietary manufacturing process, which, when combined, produce a G-NK cell therapy that demonstrates higher efficacy, persistence and enhanced cryopreservation.

When a mAb binds to the tumor target and Indapta’s G-NK cell therapy, it initiates the release of dramatically more cancer-killing compounds than conventional NK cells, allowing for increased efficacy and potentially less frequent dosing. Indapta’s off-the-shelf G-NK cell therapy is further differentiated from other NK cell therapies in that it is a cell banked product with low variability. In vivo studies have demonstrated the safety and efficacy of Indapta’s G-NK cell therapy.

Indapta is partnering with Lonza, a world-leading cell therapy manufacturer, to manufacture G-NK cells for use in clinical trials. Indapta will leverage Lonza’s process development capabilities and expertise to ensure robust, reproducible and scalable clinical good manufacturing processes (cGMP).

On February 10, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to creating novel solutions that treat urothelial and specialty cancers, reported the first published report of real-world experience utilizing the antegrade approach for Jelmyto (mitomycin) for pyelocalyceal solution administration in the Journal of Urology online on February 7, 2022 (Press release, UroGen Pharma, FEB 10, 2022, View Source [SID1234607961]). This report provides a stepwise treatment approach to low-grade Upper Tract Urothelial Cancer (LG UTUC) from initial ureteroscopy to nephrostomy placement, Jelmyto administration, and eventual nephrostomy removal.

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"While Jelmyto is approved for both retrograde and antegrade instillation, the instructions for administration address retrograde instillation, and this is the first time that data on antegrade instillation has been documented in a clinical setting for this chemoablative therapy," says Katie Murray, DO, Division of Urology, Department of Surgery, University of Missouri School of Medicine, Columbia, MO. "This report showed that antegrade instillation provided a well-tolerated and effective method of Jelmyto administration. In our experience we did not see a negative impact on patient comfort. Of note, our experience with antegrade administration in this analysis suggests that this approach, which minimizes manipulation of the ureter during instillation, may help protect against stricture formation which has been associated with repetitive instrumentation of the upper urinary tract. Given the potential benefits of antegrade versus retrograde administration of Jelmyto, we now have a replicable protocol to follow for antegrade administration using a nephrostomy tube."

In both retrograde and antegrade approaches, Jelmyto can be administered as an outpatient procedure in the clinic. Retrograde administration requires administration by a physician via a ureteral catheter, requiring fluoroscopic guidance. Antegrade administration may be performed by trained nursing professionals under clean rather than sterile conditions and does not require fluoroscopy after a nephrostogram confirms placement at the first instillation.

"Choosing the optimal treatment modality for administration of Jelmyto is very important and can have significant implications for successful treatment and recovery," said Mark Schoenberg, MD, Chief Medical Officer, UroGen. "Dr. Murray and her colleagues offer practical guidance for antegrade administration of Jelmyto, which can help reduce some of the complexity stakeholders may experience using retrograde administration."

About the Study

This single-center retrospective study reports the investigator’s technique for antegrade administration along with early outcomes from a cohort of eight patients who have undergone treatment with Jelmyto via nephrostomy tube. All patients underwent follow-up ureteroscopy with complete response in four patients. Three patients reported five adverse events. One patient had two grade-one adverse events (hematuria and fatigue); one patient had a grade-two adverse event (rash requiring oral medication, requiring one week delay of treatment); and one patient had a grade-one adverse event (a palmar rash) and a grade-three adverse event (ureteral stricture). The ureteral stricture was found in the mid-ureter at follow up ureteroscopy and required laser incision. There were no other delays in therapy. In the post hoc review, there were no other ureteral strictures.

The median follow-up was seven months after last instillation (range six weeks – 14 months). At first follow-up ureteroscopy, four patients had a complete response, four patients had a partial response, one of whom (with a history of low-grade bladder cancer) also had a bladder tumor. Four of the seven patients who have had more than one surveillance ureteroscopy had an initial complete response. At four and 14 months, two patients continue to show no evidence of disease. Two patients had a recurrence at 13 and 14 months. All patients with an initial partial response who had a follow-up ureteroscopy underwent complete endoscopic tumor ablation. One patient who could not be completely resected during their pre-instillation ureteroscopy demonstrated partial response to Jelmyto that enabled residual disease to be resected with durable response at five months after last ablation. To date, no patient has required kidney removal. Aside from the patient with bladder tumor at first ureteroscopy, no patient has experienced tumor recurrence in the bladder.

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. Despite these limitations, this study offers a replicable protocol for antegrade administration of Jelmyto.

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 February 10, 2022 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported that Jay Hagan, President and Chief Executive Officer of Regulus, will present at the 11th Annual SVB Leerink Global Healthcare Conference on Friday, February 18, 2022 at 3:00 p.m. ET (Press release, Regulus, FEB 10, 2022, View Source [SID1234607977]).

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A live webcast of the presentation will be available under "Events and Presentations" through the investor relations section of the Company’s website at www.regulusrx.com. A replay of the webcast will be archived for 90 days following the presentation date.

On February 10, 2022 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, reported Gaurav Shah, M.D., Chief Executive Officer, will participate in a fireside chat at the virtual 11th Annual SVB Leerink Global Healthcare Conference on Wednesday, Feb. 16 at 10 a.m. ET (Press release, Rocket Pharmaceuticals, FEB 10, 2022, View Source [SID1234607993]).

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A live audio webcast of the presentation will be available under "Events" in the Investors section of the Company’s website at View Source The webcast replay will be available on the Rocket website following the conference.

On February 10, 2022 Mersana Therapeutics, Inc., (NASDAQ: MRSN) a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported that the Company’s management team will participate in a fireside chat at the 11th Annual SVB Leerink Global Healthcare Conference on Thursday, February 17, 2022 at 10:40 a.m. ET (Press release, Mersana Therapeutics, FEB 10, 2022, View Source [SID1234607962]).

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A live webcast of the presentation will be available on the Investors & Media section of Mersana’s website at www.mersana.com. An archived replay will be available for approximately 30 days following the presentation.