On April 25, 2024 Vincerx Pharma, Inc. reported a proposed underwritten public offering of shares of its common stock and accompanying common stock warrants to purchase shares of its common stock and, to certain investors, pre-funded warrants to purchase shares of common stock and accompanying common stock warrants to purchase shares of its common stock (Press release, Vincerx Pharma, APR 25, 2024, View Source [SID1234642353]). All of the shares, pre-funded warrants and common stock warrants in the proposed offering will be offered by Vincerx. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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Leerink Partners is acting as the sole bookrunning manager for the offering.

The offering is being made pursuant to a shelf registration statement on Form S-3 (File No. 333-262239) that was declared effective by the Securities and Exchange Commission (the "SEC") on January 28, 2022. The offering is being made only by means of a prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement relating to the offering will be filed with the SEC and will be available on the SEC’s website, located at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying prospectus relating to the offering, when available, may also be obtained by contacting Leerink Partners LLC, Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at (800) 808-7525 ext. 6105, or by email at [email protected].

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 April 25, 2024 Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) and Mammoth Biosciences, Inc., reported a collaboration to research, develop and commercialize in vivo CRISPR-based gene editing therapies for multiple tissues and cell types (Press release, Regeneron, APR 25, 2024, View Source [SID1234642352]). Regeneron is developing adeno-associated viral vectors (AAVs) using antibody-based targeting to enhance delivery of genetic medicine payloads to specific tissues and cell types. Mammoth is developing novel ultracompact nucleases and associated gene editing systems, with a variety of editing functionalities at a significantly smaller size than other CRISPR-based systems, including first generation Cas9 nucleases. By leveraging Regeneron’s expertise in AAV and antibody engineering and Mammoth’s expertise in ultracompact gene editing systems, the teams will endeavor to create disease-modifying medicines that can be delivered to tissues beyond the liver, to which most gene editing treatments are currently limited.

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"We believe in the incredible power of gene editing, which we are utilizing in our diverse preclinical and clinical genetic medicines pipeline. After years spent developing our next-generation delivery approaches, we are eager to combine them with Mammoth’s gene editing systems to better match payload, delivery system and disease type," said Christos Kyratsous, Ph.D., Senior Vice President and co-Head of Regeneron Genetic Medicines at Regeneron. "Together, we have the potential to overcome significant delivery hurdles and effectively reach tissues around the body, impact multiple diseases and dramatically increase the number of patients who could benefit from gene editing treatments."

"Mammoth brings over a decade of scientific expertise in CRISPR, beginning with our co-founders’ work in the Doudna Lab at University of California Berkeley. We believe we can further our mission to transform the lives of patients by accelerating the discovery and development of genetic medicines in collaboration with Regeneron. Mammoth’s ultracompact CRISPR systems address the size constraints of viral delivery and complement Regeneron’s targeted AAV technologies. We look forward to working with Regeneron to enable all-in-one AAV delivery and unlock the true potential of in vivo gene editing," said Trevor Martin, Ph.D., co-founder and Chief Executive Officer of Mammoth Biosciences.

"This exciting collaboration pairs two teams with a shared commitment to translating high science into ground-breaking in vivo genetic medicines that can potentially address the needs of more patients and more prevalent conditions," said Jennifer Doudna, Ph.D., co-founder and Chair of the Scientific Advisory Board at Mammoth Biosciences, CRISPR genome editing co-inventor and winner of the 2020 Nobel Prize in Chemistry.

Under the terms of the agreement, Mammoth will receive $100 million inclusive of $95 million in equity investment at signing, and an upfront payment, and is eligible to receive up to $370 million per target in development, regulatory and commercial milestone payments, and royalty rates ranging from single digits to mid-teens on future net sales of all collaboration products. In addition, Mammoth has the right to opt-in to co-funding and sharing profits on a majority of collaboration programs in lieu of receiving milestones and royalties. In exchange, for a period of five and a half years, Regeneron is obtaining broad access to Mammoth’s editing technologies, other than certain excluded targets, with the option to extend such access for an additional two years upon the payment of a research extension fee. The parties will jointly select and research collaboration targets, and then Regeneron will lead development and commercialization.

On April 25, 2024 Purple Biotech Ltd. ("Purple Biotech" or "the Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that harness the power of the tumor microenvironment to overcome tumor immune evasion and drug resistance, reported that interim results from its randomized, controlled, open label, multicenter Phase 2 study of CM24, a first in class immune checkpoint inhibitor, for the treatment of pancreatic ductal adenocarcinoma (PDAC), have been selected as late-breaking abstract poster presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting which will take place on May 31 – June 4, 2024 in Chicago, Illinois (Press release, Purple Biotech, APR 25, 2024, View Source [SID1234642351]).

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The Phase 2 study (NCT04731467) is evaluating CM24 in combination with the Bristol Myers Squibb (BMS) PD-1 inhibitor nivolumab plus standard of care (SoC) chemotherapy in second line PDAC patients compared to SoC chemotherapy alone. The primary endpoint of the study is overall survival (OS), with progression free survival (PFS) and objective response rate (ORR) as secondary endpoints. The study was designed as Bayesian to evaluate the potential benefit of the experimental arm vs SoC and is not powered for hypothesis testing. Approximately 60 patients have been enrolled in the randomized study in 18 centers in the U.S., Spain and Israel. The study is in clinical collaboration with BMS. Purple Biotech retains all worldwide rights to CM24.

The interim data in the CM24/nivolumab plus SoC Nal-IRI/5FU/LV arm vs. the SoC Nal-IRI/5FU/LV control arm suggests a reduced risk of progression or death in the experimental arm, as demonstrated by PFS, supported by higher ORR and disease control rate (DCR) and decreasing CA19-9 in the experimental arm. Full data has been submitted to the ASCO (Free ASCO Whitepaper) Meeting.

Data from the gemcitabine/nab-paclitaxel arm is not yet mature, and OS data continues to mature for both the Nal-IRI/5FU/LV and gemcitabine/nab-paclitaxel arms.

"We are honored to be selected by the ASCO (Free ASCO Whitepaper) committee with our late breaking abstract poster presentation and are looking forward to presenting our interim results from our randomized Phase 2 CM24 study at the ASCO (Free ASCO Whitepaper) 2024 annual meeting." stated Gil Efron, Chief Executive Officer of Purple Biotech. "Topline data are expected by the end of this year."

Abstract LBA4143: Interim results of the Randomized Phase 2 Cohort of Study FW-2020-01 Assessing the Efficacy, Safety and Pharmacodynamics of CM24 in combination with Nivolumab and Chemotherapy in Advanced/metastatic Pancreatic Cancer.

● Date & Time: Sunday, June 1, 1:30 – 4:30 PM CDT.

● Lead Author: Teresa Macarulla, MD, PhD, Hospital Universitario Vall d’Hebron, Barcelona, Spain.

On April 25, 2024 PTC Therapeutics, Inc., (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ending March 31, 2024 (Press release, PTC Therapeutics, APR 25, 2024, View Source [SID1234642350]).

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"We are off to a strong start in 2024, with outstanding commercial performance and achievement of all planned clinical and regulatory milestones for the first quarter," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "We remain on track to achieve the many planned 2024 clinical and regulatory milestones, including global regulatory submissions for sepiapterin."

Key Corporate Updates:

First quarter 2024 total revenue of $210 million
First quarter 2024 revenue for the DMD franchise was $161 million
Translarna (ataluren) net product revenue was $104 million, driven by new patients in existing geographies and continued geographic expansion.
Emflaza (deflazacort) net product revenue was $57 million, resulting from new patient starts and high compliance.
Key Clinical and Regulatory Milestones:

PTC submitted an MAA to the EMA for sepiapterin for the treatment of PKU in March 2024. The company expects to submit an NDA to the FDA for sepiapterin no later than the third quarter of 2024 and to complete regulatory submissions in Japan and Brazil in 2024.
PTC submitted a BLA to the FDA for Upstaza for the treatment of AADC deficiency in March 2024.
Based on FDA feedback, PTC plans to resubmit the NDA for Translarna for the treatment of nmDMD in mid-2024.
Based on FDA feedback, PTC plans to submit an NDA for vatiquinone for the treatment of FA in late 2024.
The interim data update for the PIVOT-HD trial of PTC518 for HD patients remains on schedule for the second quarter of 2024. This update will include 12-month data on the initial group of subjects for whom data were reported in June 2023, as well as 12-week data on a larger number of stage 2 and stage 3 HD patients.
PTC expects to report topline data for the CardinALS trial of utreloxastat for ALS in the fourth quarter of 2024.
First Quarter 2024 Financial Highlights:

Total revenues were $210.1 million for the first quarter of 2024, compared to $220.4 million for the first quarter of 2023.
Total revenue includes net product revenue across the commercial portfolio of $177.6 million for the first quarter of 2024, compared to $187.6 million for the first quarter of 2023. Total revenue also includes collaboration, royalty, and manufacturing revenue of $32.5 million in the first quarter of 2024, compared to $32.8 million for the first quarter of 2023.
Translarna net product revenues were $103.6 million for the first quarter of 2024, compared to $115.1 million for the first quarter of 2023, driven by new patients in existing geographies and continued geographic expansion. The decrease was due to the timing of bulk patient orders.
Emflaza net product revenues were $57.5 million for the first quarter of 2024, compared to $54.6 million for the first quarter of 2023. These results were driven by new patient starts and high compliance.
Roche reported Evrysdi 2024 year-to-date sales of approximately $400 million, resulting in royalty revenue of $31.2 million to PTC for the first quarter of 2024, as compared to $30.8 million for the first quarter of 2023.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $116.1 million for the first quarter of 2024, compared to $195.1 million for the first quarter of 2023. The decrease in research and development expenses reflects strategic portfolio prioritization as the Company continues to focus its resources on its differentiated, high-potential R&D programs.
Non-GAAP R&D expenses were $107.2 million for the first quarter of 2024, excluding $9.0 million in non-cash, stock-based compensation expense, compared to $179.8 million for the first quarter of 2023, excluding $15.3 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $73.3 million for the first quarter of 2024, compared to $86.9 million for the first quarter of 2023. The decrease in selling, general and administrative expenses reflects lower employee costs as a result of the reduction in workforce in 2023.
Non-GAAP SG&A expenses were $63.9 million for the first quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $73.4 million for the first quarter of 2023, excluding $13.5 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was a gain of $0.1 million for the first quarter of 2024, compared to a loss of $2.4 million for the first quarter of 2023.
The net loss was $91.6 million for the first quarter of 2024, compared to a net loss of $139.0 million for the first quarter of 2023.
Cash, cash equivalents, and marketable securities was $884.8 million on March 31, 2024, compared to $876.7 million on December 31, 2023.
Shares issued and outstanding as of March 31, 2024, were 76,653,960.
PTC Reaffirms Full Year 2024 Financial Guidance:

PTC anticipates total revenues for full year 2024 to be between $600 million and $680 million.
PTC anticipates GAAP R&D and SG&A expense for full year 2024 to be between $740 and $835 million.
PTC anticipates Non-GAAP R&D and SG&A expense for full year 2024 to be between $660 and $755 million, including expected R&D expense milestone payments of up to $65 million and excluding estimated non-cash, stock-based compensation expense of $80 million.
PTC anticipates up to $90 million of payments for full year 2024 upon achievement of potential regulatory success-based milestones from previous acquisitions, of which up to $65 million will be recorded as R&D operating expense.

On April 25, 2024 PharmaMar (MSE:PHM) reported that it will present at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), that will take place between the 31st of May and the 4th of June in Chicago, U.S.A., the results of the Phase II PM1183-A-014-15 single-arm basket study, which evaluates lurbinectedin in combination with irinotecan in patients with relapsed Small Cell Lung Cancer (SCLC) after one prior platinum-containing line of treatment (Press release, PharmaMar, APR 25, 2024, View Source [SID1234642349]). The results showed a clear synergy on combining these two compounds that strengthens the use of lurbinectedin.

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PM1183-A-014-15 is a multicenter, open-label Phase I/II Basket study of lurbinectedin in combination with irinotecan in relapsed first lines patients. The study enrolled 101 patients in the SCLC cohort, and the results showed a good outcome in this patient population, included in one arm of the lurbinectedin plus irinotecan combination of the LAGOON study. These results refer to the Overall Response Rate (ORR), Duration of Response (DoR), Progression-Free and Overall Survival (PFS and OS), along with a manageable safety and tolerability profile.

Dr. Luis Paz-Ares Rodríguez, Head of Medical Oncology at the "Hospital Universitario 12 de Octubre" commented: "although both irinotecan and lurbinectedin have demonstrated activity separately in monotherapy in SCLC, these compounds with different mechanisms of action have shown an important synergy when combined".

Luis Mora, Managing Director of the Oncology Business Unit said: "These results are of special importance, given that the same regimen used in this study is being evaluated in the LAGOON Phase III study. The results observed in the relapsed SCLC cohort reinforce the rationale of one of the experimental arms of the LAGOON trial currently in progress".

As SCLC is a tumor addicted to transcription, the combination of an active transcription inhibitor (lurbinectedin) that blocks its promoter region with a topoisomerase I inhibitor (irinotecan) that is necessary to prevent DNA torsional stress during the transcription makes sense scientifically. It has been published in preclinical assays that there is a strong synergy combining lurbinectedin with irinotecan. Now, the synergy observed by combining these two drugs with different mechanisms of action inhibiting the transcription, is also shown in this clinical trial, which makes it very interesting and promising that one of the arms of the LAGOON trial consists of this combination.

The full list of PharmaMar or partner-supported presentations at the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting are:

PRODUCT TITLE LEAD AUTHOR ABSTRACT
Zepzelca (lurbinectedin) Efficacy and safety of lurbinectedin (LUR) with irinotecan (IRI) in patients (Pts) with relapsed small cell lung cancer (SCLC): Results from a phase 2 expansion cohort. Luis G. Paz-Ares PhD ABSTRACT: 8094 TYPE: Poster Session SESSION: Lung Cancer—Non-Small Cell Local-Regional/Small Cell/Other Thoracic Cancers POSTER: 356 DATE: June 6th, 2024 (01:30 PM- 04:30 PM CDT)
Efficacy and safety of lurbinectedin (LUR) with irinotecan (IRI) in a phase 2 expansion cohort of patients (Pts) with synovial sarcoma (SS). Gregory Michael Cote MD, PhD ABSTRACT: 11560 TYPE: Poster Session SESSION: Sarcoma POSTER: 486 DATE: June 1st, 2024 (01:30 PM- 04:30 PM CDT)
Randomized controlled, open-label, phase IIb/III study of lurbinectedin in combination with doxorubicin versus doxorubicin alone as first-line treatment in patients with metastatic leiomyosarcoma. Gregory Michael Cote MD, PhD ABSTRACT: TPS11590 TYPE: Poster Session SESSION: Sarcoma POSTER: 514a DATE: June 1st, 2024 (01:30 PM- 04:30 PM CDT)
A phase II study of lurbinectedin with or without avelumab in small cell carcinoma of the bladder (LASER). Nicholas I. Simon MD, MSc ABSTRACT: TPS4629 TYPE: Poster Session SESSION: Genitourinary Cancer—Kidney and Bladder POSTER: 313a DATE: TBD