On September 28, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the global leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, reported that it has entered into a Commitment Letter with certain funds managed by Highbridge Capital Management, LLC ("Highbridge"), pursuant to which Highbridge has committed to provide a $25 million senior secured, convertible term loan (the "term loan") (Press release, Gamida Cell, SEP 28, 2022, View Source [SID1234621518]).

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The Commitment Letter does not represent a definitive credit facility and is subject to certain conditions, including the consummation of a Gamida Cell equity offering resulting in gross proceeds of not less than $20 million. The Commitment Letter provides, among other things, for: (i) a maturity date 24 months from the closing date for the term loan; and (ii) an annual interest rate of 7.50%, subject to increase to 12.00% upon the occurrence of certain events, payable on a quarterly basis, and, subject to certain conditions, payable in Gamida Cell’s ordinary shares which will be valued at 95% of the volume weighted average price over a period to be agreed upon. Obligations under the term loan will be secured by substantially all of our assets and the assets of our subsidiaries.

Subject to certain limitations, the lenders will be entitled to convert the term loan, together with a make-whole premium, equal to all accrued and unpaid, and remaining coupons due through the maturity date (the "make whole amount"), into Gamida Cell’s ordinary shares at a conversion price to be equal to a 35% premium to the arithmetic mean of the volume weighted average price of Gamida Cell’s ordinary shares for the three-trading day period commencing on September 28, 2022, which price is subject to adjustment in the event of ordinary share dividends, reclassifications and certain other fundamental transactions affecting the ordinary shares. Subject to certain conditions, the term loan will be immediately callable at 100% of the principal amount plus accrued and unpaid interest to the redemption date, plus the make whole amount, plus a redemption premium of 5%. Commencing four months after the closing date for the term loan, Gamida Cell will begin monthly repayments on the term loan of principal and accrued but unpaid interest on such amount with the make-whole amount. Such installment payments can be paid to Highbridge in either cash or stock.

Gamida Cell expects to pay certain fees and expenses of Highbridge and to enter into a registration rights agreement with Highbridge, pursuant to which Gamida Cell will be required to file a registration statement registering the resale by Highbridge of any ordinary shares of Gamida Cell issuable pursuant to the terms of the term loan within 30 days after the closing date for the term loan.

About Omidubicel

Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.

Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit View Source

About GDA-201

Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy candidate for the potential treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical study data. Preclinical studies have shown that GDA-201 may address key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, these data suggest GDA-201 may improve antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. There are approximately 40,000 patients with relapsed/refractory lymphoma in the US and EU, which is the patient population that will be studied in the currently ongoing GDA-201 Phase 1/2 clinical trial.

For more information about GDA-201, please visit View Source For more information on the Phase 1/2 clinical trial of GDA-201, please visit www.clinicaltrials.gov.

GDA-201 is an investigational cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority.

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

On September 28, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the global leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, reported the pricing of a follow-on public offering of 12,905,000 of its ordinary shares at a public offering price of $1.55 per share for aggregate gross proceeds of $20 million, before deducting underwriting discounts and commissions and estimated offering expenses (Press release, Gamida Cell, SEP 28, 2022, View Source [SID1234621517]). In addition, Gamida Cell has granted the underwriters a 30-day option to purchase up to an additional 1,935,750 ordinary shares at the public offering price, less the underwriting discounts and commissions. The offering is expected to close on or about September 30, 2022, subject to satisfaction of customary closing conditions.

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Gamida Cell intends to use the net proceeds from this offering, together with its existing cash and cash equivalents and trading financial assets: for (i) commercial readiness activities to support potential launch of omidubicel, if approved; (ii) the continued clinical development of its NK product candidates, including GDA-201; and (iii) general corporate purposes, including general and administrative expenses and working capital.

Piper Sandler & Co. and JMP Securities, a Citizens Company, are acting as joint book-running managers for this offering.

A registration statement on Form S-3 (File No. 333-259472) relating to the ordinary shares has been filed with the Securities and Exchange Commission and declared effective on April 1, 2022. This offering will be made only by means of a prospectus supplement. Copies of the final prospectus supplement and the accompanying prospectus related to this offering may be obtained, when available, from: Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, Attention: Prospectus Department, by telephone at (800) 747-3924 or by email at [email protected],or JMP Securities LLC, 600 Montgomery Street, Suite 1100, San Francisco, California 94111, Attention: Prospectus Department, by calling (415) 835-8985, or by e-mail at [email protected]. Investors may also obtain these documents at no cost by visiting the SEC’s website at View Source

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, 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 registration or qualification under the securities laws of any such state or jurisdiction.

On September 28, 2022 Oncotelic Therapeutics, Inc. ("Oncotelic" or the "Company") (OTCQB:OTLC), developer of treatments for rare and orphan indications, including Parkinson Disease, Pancreatic Cancer, and DIPG, reported that the Company will be presenting at the upcoming SITC (Free SITC Whitepaper) 37th Annual Meeting, held November 10-12, 2022, in Boston, MA (Press release, Oncotelic, SEP 28, 2022, View Source [SID1234621516]).

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Abstract Number: 599

Title: Mechanism of Action for OT-101 TGF-ß immunotherapy
Session Date: 11/10/2022 – 11/11/2022,
Presentation time: 9:00 am – 9:00 pm
"Pancreatic cancer is the fourth leading cause of cancer-related mortality with a 5-year survival rate of approximately 10%. Incidence of pancreatic cancer has been steadily increasing despite advances in immunotherapy." said Dr. Vuong Trieu, CEO Oncotelic. "Using deep learning we demonstrated a combinatory approach for treatment of pancreatic cancer leveraging on OT-101 as suppressor of TGF-β. We are initiating late-stage clinical trials around this program and are excite to work with leading thought leaders to bring OT-101 to patients".

About OT-101 Pancreatic Cancer Program

Pancreatic cancer is associated with the poorest prognosis of gastrointestinal cancers and is expected to become the second leading cause of cancer-related mortality in the USA by 2030. Globally, over 400,000 people die of pancreatic cancer each year. Pancreatic cancer is traditionally considered to be an immune-resistant disease. There is a lack of effector T cells, an abundance of myeloid-derived suppressor T cells, and a dearth of key immune effector and regulatory cells. This may be part of the reason why single-agent checkpoint inhibitors are not as effective in comparison to other diseases. Here is where breaking immune tolerance by inhibiting TGF-β with OT-101 will have a significant impact.

The P001 trial was an open-label, multicenter dose-escalation study to evaluate the safety and tolerability of OT-101 (TGF-β2-specific Phosphorothioate Antisense Oligodeoxynucleotide) in adult patients with advanced tumors known to overproduce TGF- β2, which are not or no longer amenable to established therapies. Of the 61 patients treated, 37 had advanced treatment failure pancreas cancer, a very difficult-to-treat cancer with an overall survival rate that is measured in months even with the best available chemotherapy regimens. Disease control (complete response (CR)), partial response (PR) or stable disease (SD)) was achieved in 19 of 35 evaluable pancreas cancer patients (54%). Among liver mets only patients, there are exceptional single-agent activity and survival. Patient 1006 was pushed to complete response (CR) and survived as far out as 77 mos. This patient failed multiple lines of therapies: (1) surgery: Whipple’s procedure, (2) 1st line: 5-FU/LV, Dose 425 mg/m2, (3) 2nd line: 5-FU/LV, Dose 2600 mg/m2/24hr, (4) 3rd line: Gemcitabine, Dose 1000 mg/m2/week, and (5) went on to OT-101with liver mets and complete response. Patient 1022 was pushed to stable disease ("SD") with overall survival of 40 months. This patient had also failed multiple lines of therapies: (1) surgery: Whipple’s procedure, (2) 1st line: radiation therapy (50 Gy), (3) 2nd line: 5FU, and (4) went on to OT-101 with liver mets and SD.

On September 28, 2022 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported the U.S. Food and Drug Administration (FDA) has authorized an extension of the in-use period for JELMYTO (mitomycin) for pyelocalyceal solution admixture from 8 hours to 96 hours (four days) following reconstitution of the product (Press release, UroGen Pharma, SEP 28, 2022, View Source [SID1234621513]). This extension is significant and has implications for all stakeholders including physicians, hospitals, pharmacists and the adult patients who use UroGen’s medicine to treat low-grade, upper-tract urothelial cancer (LG UTUC).

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"We strive to improve the care of patients who depend on our cancer therapy and that includes streamlining logistics that can have a significant impact on the delivery, storage and timing of treatment," said Jeff Bova, Chief Commercial Officer, UroGen. "This extension expands access to JELMYTO and gives our customers greater flexibility in choosing when to mix and schedule instillations. It also prepares us for managing future growth based on increased patient volume."

UroGen encourages healthcare providers to consult the new Prescribing Information and speak to their UroGen representative about this extended in-use period to optimize flexibility when treating patients with JELMYTO.

"This label change for JELMYTO gives physicians and patients the option to treat in the morning, whereas, in most cases, the afternoon was the only option before the change," explains Rian Dickstein, M.D., Chairman of Urology, University of Maryland Baltimore Washington Medical Center (UM BWMC). "Previously, centers with mixing partners had to have JELMYTO mixed and sent by courier in the morning, now this extension provides added convenience of opening up additional scheduling options, which is very important as it enables us to more efficiently manage patient care."

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 September 28, 2022 Prestige Biopharma reported on September 27 through a public disclosure that Octava Fund Limited, the company’s major institutional shareholder, has purchased 57,000 KDR of Prestige Biopharma stock (Press release, Prestige BioPharma, SEP 28, 2022, View Source [SID1234621512]). Tay Lai Wat, Executive Director of Prestige Biopharma, also purchased 5,000 KDR Group to help defend the stock price.

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The company explained that the additional investment from its major institutional shareholder confirmed their steady partnership and trust based on the fundamental and intrinsic value of the company as well as the potential growth of the business.

Recently, Prestige Biopharma withdrew its Marketing Authorisation Application (MAA) for HD201 submitted to the European Medicines Agency (EMA). The company plans to file a new MAA for HD201 to the EMA with supplementation of data and analysis.

Since EMA informed a negative opinion on the Marketing Authorisation Application for the company’s Herceptin Biosimilar in May, major shareholders have been concerned about the sharp drop in stock price and showing support and confidence by purchasing the company’s stock.

In May, Chairman Lisa S. Park and Vice Chairman Michael J. Kim of Prestige Biopharma Group, the largest shareholders of the two companies, bought a total of KRW 7.5 billion worth of stock through Mason Partners, a joint investment company established by Chairman Park and Vice Chairman Kim. In July, Octava Fund had purchased KRW 1.3 billion of Prestige Biopharma stock and KRW 400 million of Prestige Biologics stock, amounting to a total of KRW 1.7 billion.

Tay Lai Wat, Executive Director of Prestige Biopharma, commented: "The recent withdrawal of MAA submitted to the EMA is nothing more than a temporary delay in the company’s plan. The company has no issue with its fundamental value, business competence, and liquidity."

According to the company, a New Drug Submission (NDS) for HD201 is under review by Health Canada and a MAA by the Korea Ministry of Food and Drug Safety. At the same time, the company is on track with the phase 3 clinical trial of its Avastin biosimilar HD204, phase 1 clinical trial of its Humira biosimilar PBP1502 in Europe, and phase 1/2a clinical trial of its first-in-class antibody PBP1510 in Europe and US.