Ultragenyx Announces Pricing of Public Offering of Common Stock and Pre-Funded Warrants

On October 18, 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 pricing of its underwritten public offering of 8,333,334 shares of its common stock at a price to the public of $30.00 per share (Press release, Ultragenyx Pharmaceutical, OCT 18, 2023, View Source [SID1234636115]). In addition, in lieu of issuing common stock to certain investors, the company is offering 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. The aggregate gross proceeds to the company from this offering is expected to be $300 million, before deducting underwriting discounts and commissions and other offering expenses, and excluding the exercise of any pre-funded warrants. In addition, the company has granted the underwriters of the offering an option for a period of 30 days to purchase up to an additional 1,500,000 shares of the company’s common stock at the public offering price, less the underwriting discount.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The offering is expected to close on or about October 23, 2023, subject to satisfaction of customary closing conditions. J.P. Morgan, Goldman Sachs & Co. LLC, BofA Securities, and TD Cowen are acting as joint book-running managers for the offering.

A registration statement relating to these securities has been filed with the Securities and Exchange Commission and became automatically effective on February 12, 2021. This offering is being made solely by means of a prospectus supplement and accompanying prospectus. When available, 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.

R&D Day

On October 18, 2023 Supernus Pharmaceuticals presented its corporate presentation (Presentation, Supernus, OCT 18, 2023, View Source [SID1234636114]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!


RAPT Therapeutics Announces Two FLX475 Presentations at the Society for Immunotherapy of Cancer (SITC) 2023 Annual Meeting

On October 18, 2023 Prescient Therapeutics (ASX:PTX) reported that its abstract for the results of a Phase 1b study in T-cell lymphomas has been accepted for poster presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December (Press release, RAPT Therapeutics, OCT 18, 2023, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-announces-two-flx475-presentations-society [SID1234636112]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The meeting is the largest and most comprehensive hematology conference in the world.

Prescient Therapeutics managing director and CEO, Steven Yatomi-Clarke, said, "It is a huge honour to have our abstract presented at the world’s most prestigious haematology conference. This is a validation of the quality and relevance of the clinical data we are generating in T-cell lymphomas, which is an area of unmet clinical need. It is also a testament to the hard work by the Prescient team and our clinical collaborators.

"Prescient looks forward to presenting this data at ASH (Free ASH Whitepaper) and will release this data to the market concurrently."

PharmaMar presents new data on lurbinectedin for Small Cell Lung Cancer at ESMO 2023

On October 18, 2023 PharmaMar (MSE:PHM) reported that it will present new data on lurbinectedin in Small Cell Lung Cancer (SCLC) at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) congress, being held in Madrid from October 20-24th (Press release, PharmaMar, OCT 18, 2023, View Source [SID1234636111]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Among the most outstanding studies will be an oral presentation by Dr. Antonio Calles, where final data from the LUPER trial of lurbinectedin in combination with pembrolizumab (immunotherapy) in second-line SCLC will be presented. The presentation called "Lurbinectedin (LUR) in combination with pembrolizumab (PBL) in relapsed small cell lung cancer (SCLC): the phase 1/2 LUPER study" concludes that the combination of lurbinectedin and pembrolizumab is an effective second-line treatment for SCLC in patients who did not receive prior immunotherapy, achieving in some cases deep and durable responses, with a manageable safety profile and no new emerging signs of toxicity when combining the two drugs.

Dr. Antonio Calles, Specialist in Medical Oncology at the Hospital General Universitario Gregorio Marañón, Madrid, Spain, who has led the study, comments "the combination represents an opportunity for those patients with metastatic SCLC, who could not be treated with first-line immunotherapy. The treatment achieved a confirmed response rate of 46.4%, including deep and durable responses that exceeded one year in some patients. Median Progression-Free Survival (PFS) was significantly longer for platinum-sensitive patients compared to platinum-resistant patients, with a PFS of 10 versus3 months, respectively. Currently, the only treatment other than lurbinectedin approved for this indication in the last 2 decades, has a response rate of only ~20% and a PFS of 4 months, in addition to very significant toxicity. However, here we saw no unexpected side effects both drugs could be safely combined at full doses".

Dr. Ali Zeaiter, Vice President and Head of Clinical Development at PharmaMar said: "We are pleased to present new data on the combination of lurbinectedin with immunotherapy. We believe that these data, with the suggested improvements in patients’ outcomes, confirm once again that lurbinectedin is an important treatment option for relapsed SCLC and support the ongoing investigation of lurbinectedin combination with immunotherapy".

In addition to the above presentation, the study entitled "A randomised, multicenter phase-III study comparing doxorubicin (dox) alone versus dox with trabectedin (trab) followed by trab in non-progressive patients (pts) as first-line therapy, in pts with metastatic or unresectable leiomyosarcoma (LMS): Final results of the LMS-04 study" will show the final Overall Survival (OS) data for the combination of doxorubicin and trabectedin of 33.1 months versus 23.8 months for doxorubicin alone. These data confirm that this is the first drug combination that is able to demonstrate benefit in both PFS and OS in a phase III trial in first-line treatment of metastatic leiomyosarcoma. These data have supported further the rationale of also studying lurbinectedin in sarcoma.

Lead investigator, Dr. Patricia Pautier, from the Institut Gustave Roussy in Villejuif, Paris, France, comments "the final results were positive with a median progression-free survival (PFS) statistically improved from 6.2 months with doxorubicin alone to 12.2 months with the combination."

PharmaMar’s abstracts at ESMO (Free ESMO Whitepaper) 2023

PRODUCT TITLE LEAD AUTHOR ABSTRACT
Zepzelca (lurbinectedin) Lurbinectedin (LUR) in combination with pembrolizumab (PBL) in relapsed small cell lung cancer (SCLC): the phase 1/2 LUPER study Dr. Antonio Calles Mini oral presentation ABSTRACT: 1989MO DATE: Saturday, October 21st, 14:50 – 14:55 CET
Lurbinectedin (LRB) pharmacokinetics (PK) and safety when co-administered with itraconazole (ITZ) in patients with advanced solid tumor Dra. Irene Moreno ABSTRACT: 679P DATE: Monday, October 23rd
Supportive measures to control myelosuppression and costs for patients with SCLC with lurbinectedin, CAV or topotecan with or without trilaciclib: a review on the basis of clinical trials Dr. Manuel Domine ABSTRACT: 2024P DATE: Saturday, October 21st
Yondelis (trabectedin) A randomised, multicenter phase-III study comparing doxorubicin (dox) alone versus dox with trabectedin (trab) followed by trab in non-progressive patients (pts) as first-line therapy, in pts with metastatic or unresectable leiomyosarcoma (LMS): Final results of the LMS-04 study Patricia Pautier Proffered Paper presentation ABSTRACT: #2965 DATE: Sunday, October 22nd, 08:30-10:00 CET
TOMAS2: a randomized phase 2 study from the Italian Sarcoma Group (ISG) of trabectedin plus olaparib (T+O) or trabectedin (T) in advanced, metastatic, or unresectable soft tissue sarcomas (STS) after failure of standard treatments. Lorenzo D’Ambrosio Proffered Paper presentation ABSTRACT: #4076 DATE: Sunday, October 22nd, 08:30-10:00 CET.
Tolerability and efficacy of trabectedin plus pegylated liposomal doxorubicin (PLD) in elderly patients with ovarian cancer (OC) – GEICO 105-O Study María Jesús Rubio ABSTRACT: 770P DATE: Sunday, October 22nd
Trabectedin plus radiotherapy on symptomatic advanced sarcoma patients, results from the SYNERGIAS study: a Spanish group for research in sarcoma (GEIS) phase II study Javier Martin Broto ABSTRACT: 1926P DATE: Monday, October 23rd
Targeting Microenvironment and Cellular Immunity in Sarcomas with Trabectedin Combined With Metronomic Cyclophosphamide: The TARMIC study Cheng-Ming Su ABSTRACT: 1924P DATE: Monday, October 23rd

IMUNON Announces First Patient enrolled in Phase 1/2 Clinical Trial of IMNN-001 in Combination with bevacizumab in Advanced Ovarian Cancer

On October 18, 2023 IMUNON, Inc. (NASDAQ: IMNN), a clinical-stage drug-development company focused on developing non-viral DNA-mediated immunotherapy and next-generation vaccines, reported that the first patient in a Phase 1/2 clinical trial evaluating IMUNON’s IMNN-001 in combination with bevacizumab in advanced ovarian cancer has begun treatment (Press release, IMUNON, OCT 18, 2023, View Source [SID1234636108]). IMNN-001 is a DNA-based interleukin-12 (IL-12) immunotherapy currently in Phase 2 clinical development for the localized treatment of advanced ovarian cancer (the OVATION 2 Study).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This Phase 1/2 trial is expected to enroll 50 patients with Stage III/IV advanced ovarian cancer. Patients undergoing frontline neoadjuvant therapy will be randomized 1:1 to receive standard chemotherapy plus bevacizumab vs. chemotherapy plus bevacizumab and IMNN-001. The trial’s primary endpoint is detection of minimal residual disease (MRD) by second look laparoscopy (SLL) and the secondary endpoint is progression-free survival (PFS). Initial SLL data are expected within one year following the completion of enrollment and final PFS data are expected approximately three years following the completion of enrollment. This trial will also include a wealth of translational endpoints aimed at understanding the clonal evolution and immunogenomic features of the MRD phase of ovarian cancer that is currently undetectable by imaging or tumor markers.

The principal investigator of this clinical trial is Amir Jazaeri, M.D., Professor of Gynecologic Oncology and Reproductive Medicine at The University of Texas MD Anderson Cancer Center. The Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology will also be involved in translational analyses using trial samples and animal models of ovarian cancer MRD, including biomarker and genomic analyses, which is expected to expand the Company’s knowledge of the treatment paradigm. These initiatives are a part of the Break Through Cancer Targeting Ovarian Cancer Minimal Residual Disease (MRD) Using Immune and DNA Repair Directed Therapies TeamLab collaboration.

Dr. Corinne Le Goff, President and Chief Executive Officer of IMUNON, said, "This new trial testing the combination of IMNN-001 and bevacizumab in ovarian cancer starts as we have just announced very encouraging interim data for our OVATION 2 program evaluating the benefits of IMNN-001 in the neoadjuvant setting. We believe the combination of IMNN-001 and bevacizumab holds promise based on findings in our earlier pre-clinical animal studies, where the combination showed strong synergies. IMNN-001 as an innovative immunotherapy may transform the current ovarian cancer first line treatment landscape and provide new options to women diagnosed with Stage III/IV disease who face low cure rates of 15% or less."

About Epithelial Ovarian Cancer

Epithelial ovarian cancer (EOC) is the fifth deadliest malignancy among women in the United States. There are approximately 22,000 new cases of ovarian cancer every year and the majority (approximately 70%) are diagnosed in advanced Stages III and IV. EOC is characterized by dissemination of tumor in the peritoneal cavity with a high risk of recurrence (75%, Stages III and IV) after surgery and chemotherapy. Since the five-year survival rates of patients with Stages III and IV disease at diagnosis are poor (41% and 20%, respectively), there remains a need for a therapy that not only reduces the recurrence rate but also improves overall survival. The peritoneal cavity of advanced ovarian cancer patients contains the primary tumor environment and is an attractive target for a regional approach to immune modulation.

About IMNN-001 Immunotherapy

Designed using IMUNON’s proprietary TheraPlas platform technology, IMNN-001 (formerly GEN-1) is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system that enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anticancer immunity acting through the induction of T-lymphocyte and natural killer cell proliferation. The Company previously reported positive safety and encouraging Phase 1 results with IMNN-001 administered as monotherapy or as combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer and completed a Phase 1b dose-escalation trial (the OVATION 1 Study) of IMNN-001 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer. It announced full enrollment in the OVATION 2 Study in September 2022, interim data in September 2023 and expects to report topline data in the second quarter of 2024.