On April 22, 2024 Biohaven Ltd. (NYSE: BHVN), a biopharmaceutical company focused on the discovery, development, and commercialization of life-changing treatments in key therapeutic areas, including immunology, neuroscience, and oncology, reported the closing of its underwritten public offering of 6,451,220 of its common shares, which includes the full exercise of the underwriters’ option to purchase 841,463 additional common shares, at a public offering price of $41.00 per share (Press release, Biohaven Pharmaceutical, APR 22, 2024, View Source [SID1234642217]). The gross proceeds from the offering were approximately $264.5 million before deducting underwriting discounts and commissions and offering expenses payable by Biohaven. Biohaven intends to use the net proceeds received from the offering for general corporate purposes.

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J.P. Morgan, Morgan Stanley, TD Cowen, and Piper Sandler & Co. acted as the joint lead book-running managers of the offering. Cantor acted as a book-runner of the offering. Baird also acted as a book-runner of the offering.

The offering was made only by means of a prospectus supplement and the accompanying prospectus, copies of which may be obtained from the offices of the following: J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204, or by email at [email protected], Morgan Stanley & Co. LLC, Attention: Prospectus Department, 180 Varick Street, 2nd Floor, New York, New York 10014, TD Securities (USA) LLC, 1 Vanderbilt Avenue, New York, NY 10017, by telephone at (855) 495-9846 or by email at [email protected], and Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, Attention: Prospectus Department, by telephone at (800) 747-3924 or by email at [email protected].

The shares were issued pursuant to an effective shelf registration statement on Form S-3. Copies of the registration statement can be accessed through the SEC’s website at www.sec.gov. This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the applicable securities laws of such state or jurisdiction.

On April 22, 2024 Replicate Bioscience, a clinical-stage company pioneering novel self-replicating RNA (srRNA) technology for use in infectious disease, oncology, autoimmune disease, and more, reported acceptance of two oral presentations at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, May 7-11 in Baltimore, Maryland (Press release, Replicate Bioscience, APR 22, 2024, View Source;cell-therapy-asgct-annual-meeting-302123737.html [SID1234642216]).

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Presentation Details:

The full abstracts are now available through the ASGCT (Free ASGCT Whitepaper) conference website.

Title: Novel Self-Replicating RNA Vectors Broaden Therapeutic Window and Expand Use Outside of Vaccines
Session Title: Emerging Viral Vectors
Location and Time: Ballroom 4, Thursday, May 9, 5:15 PM – 5:30 PM ET
Abstract: 204
Presenter: Parinaz (Paris) Aliahmad
Summary: Ultra-low doses of optimized srRNA vectors achieved immunization with protective antibody titers with minimal reactogenicity in a preclinical influenza model. This broadened therapeutic window presents the opportunity to use these vectors for many clinical applications.

Title: Single and Low Dose Self-Replicating RNA Vaccine Provides Effective Immune Protection Against Rabies in Healthy Volunteers
Session Title: Vector Product Engineering, Development, and Manufacturing (excluding AAV)
Location and Time: Room 314-317, Saturday, May 11, 10:15 AM – 10:30 AM ET
Abstract: 408
Presenter: Zelanna Goldberg
Summary: RBI-4000, an srRNA vector encoding the rabies glycoprotein encapsulated in a lipid nanoparticle, is the first clinical demonstration of next-generation srRNA technology. Interim analysis of a Phase 1 trial demonstrate that all doses were well tolerated and that RBI-4000 was immunogenic at all dose levels tested, conferring protection with a single, low dose.

On April 22, 2024 Carisma Therapeutics Inc. (Nasdaq: CARM) ("Carisma" or the "Company"), a clinical-stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, reported the acceptance of two abstracts at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 Annual Meeting being held May 7-11, 2024, in Baltimore, MD (Press release, Carisma Therapeutics, APR 22, 2024, View Source [SID1234642214]).

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"We are excited to present new preclinical data highlighting the potential of our engineered macrophage cell therapies for the treatment of liver and lung fibrosis, both of which represent significant unmet medical needs with limited available therapies for patients," said Michael Klichinsky, PharmD, PhD, Co-founder and Chief Scientific Officer of Carisma. "We look forward to presenting these data and are excited to expand our macrophage engineering platform beyond oncology."

Poster Presentation Details:

Title: Genetically Engineered Macrophage Cell Therapy Reverses Liver and Lung Fibrosis in Preclinical Models
Abstract #: 852
Session: Other Cellular and Regenerative Therapies
Date & Time: Wednesday, May 8, 2024; 12:00 PM ET
Location: Exhibit Hall

Title: Redirected Soluble Modulators (RSM) – A Novel Engineering Strategy to Enhance Immune Receptor Signaling
Abstract #: 812
Session: Immune Targeting and Approaches with Genetically-Modified Cells and Cell Therapies
Date & Time: Wednesday, May 8, 2024; 12:00 PM ET
Location: Exhibit Hall

The posters presented at ASGCT (Free ASGCT Whitepaper) 2024 will be available online in the "Publications" section of Carisma’s website at View Source following the start of the respective poster sessions.

On April 22, 2024 Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS), a commercial-stage biotechnology company, reported that the first patient was dosed in its Phase 1 clinical trial (KYANITE-1) evaluating inhaled KB707, a modified HSV-1 vector designed to deliver genes encoding both human interleukin-12 (IL-12) and interleukin-2 (IL-2) to the lung, for the treatment of patients with locally advanced or metastatic solid tumors of the lung (Press release, Krystal Biotech, APR 22, 2024, View Source [SID1234642213]).

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"Cytokine therapy holds significant potential for the treatment of solid tumors but its utility has been limited by a lack of safe and effective delivery options," said David Chien, M.D., Senior Vice President of Clinical Development at Krystal Biotech. "Cytokine delivery via inhalation is a first-of-its-kind therapeutic approach made possible by the unique attributes of Krystal’s HSV-1-based vector platform. Together with intratumoral KB707, inhaled KB707 has the potential to significantly expand the clinical utility of cytokine therapy to treat a wide range of otherwise difficult-to-treat and standard of care refractory solid tumors. Dosing the first patient in KYANITE-1 is an exciting step toward our goal of delivering a new class of cancer immunotherapies."

The KYANITE-1 clinical trial is an open-label, multicenter, dose escalation and expansion study to evaluate inhaled KB707 monotherapy in patients with advanced solid tumor malignancies affecting the lungs. Details of the KYANITE-1 study can be found at www.clinicaltrials.gov under NCT identifier: NCT06228326.

"Dosing the first patient in our inhaled KB707 trial is another important milestone for our oncology program and for Krystal," said Suma Krishnan, President, Research & Development, Krystal Biotech. "KYANITE-1 is our second active clinical trial evaluating KB707 and along with KB407 and KB408, our inhaled genetic medicine candidates for the treatment of cystic fibrosis and alpha-1 antitrypsin deficiency, respectively, KB707 is our third clinical stage drug candidate delivered via inhalation. With a deep pipeline of active clinical trials, we are looking forward to data readouts starting later in 2024, which we expect will showcase the breadth and potential of our proprietary, HSV-1 based gene delivery platform."

In February 2024, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for inhaled KB707 for the treatment of patients with solid tumors with pulmonary metastases that are relapsed or refractory to standard of care therapy. This is the second Fast Track Designation for the KB707 program. In July 2023, the FDA granted intratumoral KB707 Fast Track Designation for the treatment of anti-PD-1 relapsed/refractory locally advanced or metastatic melanoma.

About Fast Track Designation
Fast Track Designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and treat a serious or unmet medical need, enabling drugs to reach patients sooner. Clinical programs with Fast Track Designation may benefit from early and frequent communication with the FDA throughout the regulatory review process, and such clinical programs may be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met.

About IL-2, IL-12, and KB707
IL-2 and IL-12 are secreted cytokines with complementary functions promoting cell-mediated immunity in humans. Both IL-2 and IL-12 have been shown to elicit anti-tumor immune responses in preclinical models and in clinical settings and have been extensively studied for their potential in cancer immunotherapy. Despite promising signs of efficacy, it has proven difficult to effectively harness IL-2 and IL-12 for therapeutic benefit, as systemic administration is often poorly tolerated, and their inherently short half-lives necessitate high dose levels and extremely frequent dose intervals. KB707 is a modified HSV-1 vector designed to deliver genes encoding both human IL-12 and IL-2 directly to a patient’s tumor(s) and promote systemic immune-mediated tumor clearance. KB707 targets solid tumors that are accessible via intratumoral injection or inhalation.

On April 22, 2024 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a clinical stage biotechnology company whose proprietary INTASYL siRNA gene silencing technology is designed to make immune cells more effective in killing tumor cells, reported it is presenting new data about its lead clinical product candidate, PH-762, an INTASYL compound (Press release, Phio Pharmaceuticals, APR 22, 2024, View Source [SID1234642212]).

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Preclinical studies demonstrate:

PH-762 is rapidly taken up by cells and robustly silences PD-1 mRNA and protein in lymphocytes within the tumor microenvironment (TME)
Intratumoral injection of murine PH-762 (mPH-762) significantly inhibits tumor growth in murine tumor models and is well tolerated
mPH-762-mediated silencing of PH-762 within the TME may generate memory-specific T cells, promoting IFN-γ release in the TME
Studies in non-human primates demonstrate that PH-762 is well-tolerated and does not induce release of cytokines associated with cytokine release syndrome (CRS)
These finding support the ongoing clinical trial of PH-762’s safety and efficacy as a neoadjuvant therapy for treatment of cSCC, melanoma, or Merkel cell carcinoma
The data, authored by Melissa Maxwell, Linda Mahoney, and Dr. Mary Spellman, will be presented at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) on May 8th in Baltimore, Maryland.

Presentation Details are as follows:
Title: INTASYL PH-762: PD-1 Intratumoral Immunotherapy
Abstract Number: 774
Session Title: Cancer-Immunotherapy and Cancer Vaccines
Authors: Melissa Maxwell, Linda Mahoney, Mary Spellman, M.D.
Date and Time: May 8, 2024 12:00 PM
Location: Exhibit Hall