On October 23, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, reported the closing of its previously announced underwritten public offering of 9,833,334 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 1,500,000 shares, at a price to the public of $30.00 per share, and, to certain investors in lieu of common stock, pre-funded warrants to purchase 1,666,722 shares of its common stock at a purchase price of $29.999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.001 exercise price per share of each pre-funded warrant (Press release, Ultragenyx Pharmaceutical, OCT 23, 2023, View Source [SID1234636262]). The estimated net proceeds to the company, after deducting underwriting discounts and commissions and other offering expenses payable by the company, were approximately $326.1 million.

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J.P. Morgan, Goldman Sachs & Co. LLC, BofA Securities, and TD Cowen acted as joint book-running managers for the offering.

The securities described above were offered by Ultragenyx Pharmaceutical Inc. pursuant to a registration statement previously filed with the Securities and Exchange Commission that became automatically effective on February 12, 2021. A final prospectus supplement and accompanying prospectus were filed with the SEC and available for free on the SEC’s website at View Source Copies of the final prospectus supplement and the accompanying prospectus related to the offering may be obtained from J.P. Morgan Securities LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, by telephone at 866-803-9204, or by email at prospectus- [email protected]; Goldman Sachs & Co. LLC, Prospectus Department, 200 West Street, New York, NY 10282, telephone: 1-866-471-2526, facsimile: 212-902-9316 or by emailing [email protected]; BofA Securities, NC1-002-02-25, 201 North Tryon Street, Charlotte, NC 28255-0001, Attention: Prospectus Department, or by email at [email protected]; and Cowen and Company, LLC, 599 Lexington Avenue, New York, NY 10022, by email at [email protected] or by telephone at (833) 297-2926.

This press release shall 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 registration or qualification under the securities laws of any such state or jurisdiction.

On October 23, 2023 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), a clinical stage immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported a trial-in-progress poster from the Phase 1/2 clinical trial for SNS-101, a conditionally active, human monoclonal antibody targeting the immune checkpoint VISTA (V-domain Ig suppressor of T cell activation), at the European Society for Medical Oncology Congress (ESMO) (Free ESMO Whitepaper) 2023 (Press release, Sensei Biotherapeutics, OCT 23, 2023, View Source [SID1234636261]).

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The multi-center Phase 1/2 clinical trial is a first-in-human, open-label, dose escalation study evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of SNS-101 as both a monotherapy and in combination with Regeneron’s PD-1 inhibitor Libtayo in patients with advanced solid tumors. To date, four patient cohorts have been enrolled in the monotherapy arm. Sensei expects to report initial pharmacokinetic and safety monotherapy data in the fourth quarter of 2023, and topline monotherapy data in 2024. The company has also initiated the combination arm of the study and expects to report initial combination safety and pharmacokinetic data in the first quarter of 2024, with preliminary anti-tumor activity combination data in 2024.

The full poster is available for viewing on Sensei’s website in the "Events & Presentations" section of the Company’s Investor Relations website at www.senseibio.com.

On October 23, 2023 Revolution Medicine presented its corporate presentation (Press release, Revolution Medicines, OCT 23, 2023, View Source [SID1234636260]).

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On October 23, 2023 Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology that leverages a patient’s own biology to deliver rare disease therapeutics, reported the successful completion of its acquisition of EryDel S.p.A., a privately-held, late-stage biotech company (Press release, Quince Therapeutics, OCT 23, 2023, View Source [SID1234636259]). Quince’s newly acquired Phase 3 lead asset, EryDex, targets a rare neurodegenerative disease, Ataxia-Telangiectasia (A-T). Currently, there are no approved treatments for A-T and the market represents a $1+ billion estimated peak sales opportunity globally. EryDex utilizes a highly differentiated and proprietary technology platform for autologous intracellular drug encapsulation (AIDE), which is designed to optimize the biodistribution of dexamethasone sodium phosphate (DSP; a pro-drug) by using an A-T patient’s own red blood cells to deliver the sustained therapeutic over a once monthly treatment period.

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"The successful closing of the EryDel acquisition is an exciting step forward in fulfilling our vision to build a leading rare disease biotechnology company," said Dirk Thye, M.D., Quince’s Chief Executive Officer. "We are dedicated to developing treatments utilizing our proprietary AIDE technology platform that hold the potential to help children and families affected by rare and debilitating diseases such as A-T. Quince’s priority is to advance the Phase 3 clinical trial of EryDex to evaluate its safety and efficacy for the treatment of A-T, then expand our development efforts into other potential indications that leverage our proprietary AIDE technology platform."

Quince is well-capitalized into 2026 and intends to focus its development expertise and financial resources toward advancing a single global Phase 3 NEAT (Neurologic Effects of EryDex on Subjects with A-T) clinical trial, which is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the neurological effects of EryDex on patients with A-T. Enrollment for the Phase 3 NEAT trial is expected to begin in the second quarter of 2024. The company plans to enroll approximately 86 A-T patients aged six to nine years-old and approximately 20 additional A-T patients aged 10 years or older. This pivotal clinical trial will be conducted under a Special Protocol Assessment (SPA) that has been agreed with the U.S. Food & Drug Administration (FDA), which should allow for the submission of a New Drug Application (NDA) following completion of this single study, assuming positive results.

Quince’s integrated senior leadership team holds extensive development, clinical, regulatory, and commercial expertise, and includes:

Dirk Thye, M.D. – Chief Executive Officer and member of Quince’s Board of Directors
Charles Ryan, J.D., Ph.D. – President
Guenter R. Janhofer, M.D., Ph.D. – Chief Medical Officer
Brendan Hannah, M.B.A. – Chief Business Officer and Principal Financial Officer
Giovanni Mambrini, MSc – Chief Technology Officer
Thomas Sabia, M.B.A. – Chief Commercial Officer
Pamela Williamson, RAC, FRAPS, M.B.A. – Head of Regulatory Affairs
EryDel’s former Chief Executive Officer, Luca Benatti, Ph.D., also joins Quince’s Board of Directors.

The acquisition of EryDel was completed with no upfront cash payment, using a stock-for-stock exchange and potential downstream milestone cash payments. EryDel stockholders now own 15.2% of Quince’s outstanding shares and may be issued up to an additional 725,036 shares of the company’s common stock (equal to 16.6% of Quince’s currently outstanding shares – inclusive of the shares issued) upon the first anniversary of the transaction closing. EryDel stockholders also will be entitled to up to $485 million in potential total downstream cash payments, including up to $5 million in development milestones, $25 million at NDA acceptance, $60 million in approval milestones, and $395 million on market and sales milestones, with no royalties paid to EryDel stockholders. The transaction includes the assumption of EryDel’s $13 million (€10 million in principal) European Investment Bank (EIB) loan with scheduled payments beginning in the second half of 2026.

On October 23, 2023 PDS Biotechnology Corporation (Nasdaq: PDSB) (PDS Biotech or the Company), a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer immunotherapies and infectious disease vaccines based on the Company’s proprietary T cell activating platforms, reported immune response data from a preliminary analysis of a subset of patients in VERSATILE-002, the Phase 2 clinical trial evaluating the safety and efficacy of PDS0101 in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in patients with human papillomavirus (HPV)16-positive recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) (Press release, PDS Biotechnology, OCT 23, 2023, View Source [SID1234636258]). The data presented during the European Society for Medical Oncology Congress 2023 (ESMO Congress 2023) provided preliminary insight to the pre-existing immune responses in advanced HPV-positive HNSCC patients and potential changes to the immune profile after treatment.

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"Generation of multifunctional, anti-tumor T cells with the relevant cytokine and chemokine profiles are necessary for effective long-term control of tumor growth and clinical outcomes. This initial study focused on understanding the immunological profile of advanced head and cancer patients in the blood," said Kevin Harrington, PhD, Professor of Biological Cancer Therapies, The Royal Marsden. "This preliminary study suggests that PDS0101 may promote a TH1 predominant cytokine profile as well as induction of important T cell activating chemokines. Such studies could be helpful in providing further insight into how PDS0101 in combination with KEYTRUDA alters T cell cytokine and chemokine profiles to promote improved clinical outcomes."

The data presented at ESMO (Free ESMO Whitepaper) 2023 included 18 patients with a median age of 63 years (range 46-83) and all had confirmed HPV16-positive tumors. The immunological profiles were assessed at three timepoints: pre-treatment,12 (cycle 5), and 36 weeks (cycle 13) following four and five cycles of combination therapy, respectively.

Highlights of the analysis include:

•
Combination of PDS0101 and KEYTRUDA appears to lead to changes towards a TH1 predominant cytokine profile reported to be associated with improved killer T cell activity

•
Combination of PDS0101 and KEYTRUDA resulted in increased polyfunctionality reflected in T cells expressing 5 or more cytokines. Increased polyfunctionality is typically associated with enhanced killing function and anti-tumor activity

"This analysis provides preliminary insights into how PDS0101 in combination with KEYTRUDA may be impacting specific cytokines and chemokines in CD8 and CD4 T cell populations," said Lauren V. Wood, MD, Chief Medical Officer of PDS Biotech. "The investigational combination appears to be promoting a predominant TH1 immunologic profile that is associated with decreases in CD8 T cells in peripheral blood. We are encouraged that these observations align with other Phase 2 studies reporting PDS0101-induced polyfunctional CD8 T cells traffic to tumors and we look forward to continued investigation in our VERSATILE-003 Phase 3 study."

1 Klopp A, et al. 2022. IMMUNOCERV, an ongoing Phase II trial combining PDS0101, an HPV-specific T cell immunotherapy, with chemotherapy and radiation for treatment of locally advanced cervical cancers. Presented at: Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper); November 8-12, 2022. Boston, MA. Abstract: 674.

About PDS0101
PDS0101, PDS Biotech’s lead candidate, is a novel investigational human papillomavirus (HPV)-targeted immunotherapy that stimulates a potent targeted T cell attack against HPV-positive cancers. PDS0101 is given by subcutaneous injection alone or in combination with other immunotherapies and cancer treatments. In a Phase 1 study of PDS0101 in monotherapy, the treatment demonstrated the ability to generate multifunctional HPV16-targeted CD8 and CD4 T cells with minimal toxicity. Interim data suggests PDS0101 generates clinically active immune responses and the combination of PDS0101with other treatments can demonstrate significant disease control by reducing or shrinking tumors, delaying disease progression, and/or prolonging survival. The combination of PDS0101 with other treatments does not appear to compound the toxicity of other agents.

About VERSATILE-002
VERSATILE-002 is a single-arm Phase 2 trial evaluating the safety and efficacy of PDS0101, an HPV16-targeted investigational T cell-activating immunotherapy that leverages PDS Biotech’s proprietary Versamune technology, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab). The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve and ICI-refractory patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC) and was granted Fast Track designation by the Food and Drug Administration in June 2022.