On April 18, 2024 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing innovative treatments for cancer patients, reported that its abstract on the preclinical data for its radiopharmaceutical program MNPR-101-Zr (MNPR-101 conjugated to zirconium-89) submitted to the Society of Nuclear Medicine and Molecular Imaging (SNMMI) 2024 Annual Meeting has been selected for a presentation (Press release, Monopar Therapeutics, APR 18, 2024, View Source [SID1234642153]). SNMMI 2024, held in Toronto, Canada, is recognized as the premier educational, scientific, and research event in the radiopharma space.

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Meeting Details:

Event: Society of Nuclear Medicine and Molecular Imaging (SNMMI) 2024 Annual Meeting

Date: June 8 – June 11, 2024

Location: Metro Toronto Convention Centre, Toronto, ON, Canada

On April 18, 2024 Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported that the European Medicines Agency (EMA) has granted Orphan Drug Designation to Annamycin for the treatment of AML (Press release, Moleculin, APR 18, 2024, View Source [SID1234642152]).

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Annamycin is the Company’s next-generation anthracycline that has been designed to be non-cardiotoxic (unlike currently prescribed anthracyclines) and has shown to be non-cardiotoxic in the 82 subjects treated in multiple studies in the U.S. and in Europe. Furthermore, Annamycin has recently shown in Moleculin’s European clinical study for the treatment of AML using Annamycin in combination with Cytarabine (MB-106) a preliminary 60% composite complete response (CRc) rate in 2nd line subjects (N=10) and an overall interim CRc of 39% in all subjects (N=18), regardless of the prior number of therapies, in the European trial. Durability of the CRc’s is developing. One subject has surpassed the one-year mark with a durable complete response. Recruitment in MB-106 has ended for 2nd line subjects while recruitment for 1st line and 3rd line subjects continue. Annamycin is currently in development for the treatment for AML and STS lung mets, and the Company believes the drug may have the potential to treat additional indications.

"We are pleased to receive Orphan Drug Designation from the EMA for Annamycin, which further supports our ongoing efforts to advance this compelling next-generation anthracycline for the treatment of hard-to-treat cancers," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Combined with the Orphan Drug Designation we already have in the US and with the new composition of matter patent just awarded by the US Patent and Trademark Office with coverage through 2040, we believe the commercial exclusivity of Annamycin is now very well protected. We continue to be encouraged by the growing body of promising interim clinical data demonstrated by Annamycin. We remain focused on advancing our clinical and regulatory strategies toward our next phase of development, including the planned commencement of a pivotal registration study as a 2nd line therapy in AML before year end."

The EMA grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and up to 10 years of potential market exclusivity in the European Union if approved.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of relapsed or refractory AML, in addition to Orphan Drug Designation for the treatment of STS lung mets.

On April 18, 2024 Immix Biopharma, Inc. (Nasdaq: IMMX) ("ImmixBio", "Company", "We" or "Us" or "IMMX"), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and other autoimmune diseases, reported that Immix Biopharma is on track to dose NXC-201 patients in the U.S. with no change in patient enrollment timing (Press release, Immix Biopharma, APR 18, 2024, View Source [SID1234642151]).

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"We are on track to dose relapsed/refractory AL Amyloidosis NXC-201 patients at our New York City lead site and other leading U.S. sites in mid-2024. U.S. site clinical trial agreements have been signed, and site initiation visits are being scheduled," said Ilya Rachman, MD PhD CEO of Immix Biopharma. "Building on our existing NXC-201 clinical dataset, our U.S. relapsed/refractory AL Amyloidosis clinical trial design is focused on patients with adequate cardiac function who are most likely to experience the greatest clinical benefit from NXC-201. We are committed to providing additional treatment alternatives for relapsed/refractory AL Amyloidosis patients, where there are no FDA approved drugs today."

About NEXICART-2

NEXICART-2 (NCT06097832) is an open-label, single-arm, multi-site Phase 1b dose expansion clinical trial in relapsed/refractory AL Amyloidosis for CAR-T NXC-201 in the United States. NEXICART-2 is expected to enroll 40 patients with adequate cardiac function over a period of approximately 18 months from first patient dosing. The objectives are the safety and efficacy of NXC-201. The expected primary endpoints are complete response rate and overall response rate according to consensus recommendations (Palladini et al. 2012).

About NXC-201

We believe NXC-201 (formerly HBI0101) is the only "Single-Day CRS" BCMA-targeted CAR-T cell therapy that is uniquely suited to target AL Amyloidosis and other autoimmune diseases. It is being studied in a comprehensive clinical development program for the treatment of patients with relapsed/refractory AL amyloidosis, and expanding into other autoimmune indications. These trials build on a robust NXC-201 clinical dataset initiated in February 2021. NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma, and awarded EU ODD by the EMA in AL Amyloidosis.

On April 18, 2024 Ferring Pharmaceuticals and SK pharmteco reported an agreement to scale up commercial manufacturing capacity for the drug substance of Ferring’s intravesical gene therapy Adstiladrin (nadofaragene firadenovec-vncg) for ensuring long-term future supply (Press release, Ferring, APR 18, 2024, https://www.ferring.com/ferring-pharmaceuticals-and-sk-pharmteco-enter-into-commercial-gene-therapy-manufacturing-deal/ [SID1234642150]). Following technology transfer, SK pharmteco, a contract development manufacturing organisation (CDMO), will be qualified as another source for manufacturing, testing, and release of the medicine, subject to regulatory approval by the U.S. Food and Drug Administration (FDA).

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Adstiladrin was approved by the FDA in December 2022 for adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

This additional scale up and diversification of drug substance capacity demonstrates Ferring’s commitment to securing future long-term supply of Adstiladrin to meet the projected commercial growth of its non-replicating gene therapy. In addition to this contract manufacturing agreement, dedicated capacity expansion of Adstiladrin is well underway at state-of-the-art facilities in Ferring’s existing site in Finland and its U.S. campus in Parsippany, New Jersey. Ferring will provide updates on the progress of these near-term projects later in 2024.

"Ferring takes its responsibilities to patients with high-risk BCG-unresponsive NMIBC very seriously – especially when pioneering in the emerging field of gene therapy commercial manufacturing," said Bipin Dalmia, Global Head, Uro-Oncology Franchise, Ferring Pharmaceuticals. "Following our January announcement of full availability of Adstiladrin across the U.S. and expansion of our clinical trials program, this agreement with SK pharmteco comes alongside major investments in our own manufacturing capabilities to assure the breadth of our long-term supply base. Stable and sustainable supply is a vital part of our mission to fill this unmet clinical need for the patients we serve."

The need to scale up supply of strategically important medicines for bladder cancer across geographies is demonstrated by the trend of increased disease prevalence worldwide. The World Bladder Cancer Patient Coalition has reported that bladder cancer became the ninth most common cancer in the world (a rise from tenth) according to new data published in February 2024 (IARC GLOBOCAN 2022)1. In the United States, it is the seventh most common cancer, fourth among men, 2-3 and 75% of bladder cancer presents as NMIBC.4

"SK pharmteco is proud to partner with Ferring Pharmaceuticals as a manufacturer of the breakthrough gene therapy Adstiladrin," said Joerg Ahlgrimm, Chief Executive Officer for SK pharmteco. "Our integrated approach, incorporating customizable clinical and commercial GMP manufacturing solutions with comprehensive in-process testing, quality control, and lot release programs, provides unparalleled support to our partners throughout their product lifecycle and is in perfect alignment with our mission of expediting the delivery of potentially life-saving therapies to patients across the globe."

SK pharmteco was selected based on their competencies and alignment on core values across the two companies following a thorough assessment of potential CDMOs with gene therapy development and GMP manufacturing capabilities.

About Adstiladrin
Adstiladrin (nadofaragene firadenovec-vncg) is a gene therapy developed as a treatment for adult patients with BCG-unresponsive NMIBC. It is a non-replicating adenovirus vector-based gene therapy containing the gene encoding interferon alfa-2b protein, administered by catheter into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the interferon gene. The internal cell machinery translates the interferon DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a recombinant analog of the naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patient’s own bladder wall cells into interferon microfactories, enhancing the body’s natural defenses against the cancer. Nadofaragene firadenovec-vncg has been studied in a clinical trial program that includes 157 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).

Nadofaragene firadenovec-vncg has been studied in a clinical trial program that includes 157 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).4

INDICATION
Adstiladrin is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS: Adstiladrin is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.

WARNINGS AND PRECAUTIONS:
Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from Adstiladrin due to low levels of replication-competent adenovirus. Avoid Adstiladrin exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer Adstiladrin by intravesical instillation only. Adstiladrin is not for intravenous use, topical use, or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during Adstiladrin treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during Adstiladrin treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.

Please click to see the full Prescribing Information.

On April 18, 2024Bio-Path Holdings, Inc., (Nasdaq: BPTH) (the "Company" or "BioPath"), a biotechnology company leveraging its proprietary DNAbilize antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, reported that it has entered into a definitive agreement with certain institutional investors for the issuance and sale of 375,000 shares of its common stock at a purchase price per share of $3.225 in a registered direct offering priced at-the-market under Nasdaq rules (Press release, Bio-Path Holdings, APR 18, 2024, View Source [SID1234642149]). Additionally, in a concurrent private placement, Bio-Path has also agreed to issue to such investors unregistered warrants to purchase up to 375,000 shares of common stock at an exercise price of $3.10 per share. The unregistered warrants to be issued in the private placement will become immediately exercisable upon issuance and will expire five years from the date of issuance. The offering is expected to close on or about April 19, 2024, subject to the satisfaction of customary closing conditions.

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Immediately prior to the registered direct offering and the concurrent private placement, the number of outstanding shares of common stock of the Company was 1,190,306. Immediately following the closing of the registered direct offering and the concurrent private placement, the number of outstanding shares of common stock of the Company will be 1,565,306.

H.C. Wainwright & Co., LLC is acting as the exclusive placement agent for the offering.

The gross proceeds to Bio-Path from the offering are expected to be approximately $1.2 million, before deducting the placement agent’s fees and other offering expenses payable by Bio-Path. BioPath currently intends to use the net proceeds from the offering for working capital and general corporate purposes.

The shares of common stock (or common stock equivalents) offered in the registered direct offering (but excluding the unregistered warrants or the shares of common stock underlying such unregistered warrants) described above are being offered and sold by Bio-Path pursuant to a "shelf" registration statement on Form S-3 (Registration No. 333-265282), including a base prospectus, previously filed with the Securities and Exchange Commission ("SEC") on May 27, 2022, and declared effective by the SEC on June 14, 2022. The offering of the shares of common stock (or common stock equivalents) are being made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and an accompanying base prospectus relating to, and describing the terms of, the registered direct offering will be filed with the SEC and will be available on the SEC’s website located at View Source Electronic copies of the prospectus supplement and accompanying base prospectus relating to the registered direct offering, when available, may also be obtained from H.C. Wainwright & Co., LLC at 430 Park Ave., New York, New York 10022, by telephone at (212) 856-5711, or by email at [email protected].

The offer and sale of the unregistered warrants are being made in a transaction not involving a public offering and have not been registered under Section 4(a)(2) of the Securities Act of 1933, as amended (the "Securities Act") and/or Rule 506(b) of Regulation D promulgated thereunder and, along with the shares of common stock underlying such unregistered warrants, have not been registered under the Securities Act or applicable state securities laws. Accordingly, the unregistered warrants and the underlying shares of common stock may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws.

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 jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such jurisdiction.