On December 5, 2024 Pfizer Inc. (NYSE: PFE) reported that it will highlight the latest advancements from its growing hematology and breast cancer portfolios at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (December 7-10) and the San Antonio Breast Cancer Symposium (SABCS, December 10-13) (Press release, Seagen, DEC 5, 2024, View Source [SID1234648829]). Data from more than 100 company-sponsored, investigator-sponsored, and collaborative research abstracts, including 13 oral presentations and four poster spotlights, will be shared across the company’s approved medicines and expanding portfolio of potential breakthroughs for patients with blood and breast cancers, as well as rare blood disorders.

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"Our robust presence at ASH (Free ASH Whitepaper) and SABCS reinforces Pfizer’s legacy of scientific innovation for people living with blood disorders and breast cancer," said Chris Boshoff, Chief Oncology Officer and Executive Vice President, Pfizer. "We are pleased to share the latest updates for some of our key approved medicines, including ADCETRIS, ELREXFIO, and IBRANCE, which continue to generate compelling data as foundations of care in their respective indications. We are also excited to present new results in hemophilia and from our expanding pipeline of innovative, next-generation therapy candidates for both blood and breast cancers, including new data on combination approaches across our core scientific modalities."

Key ASH (Free ASH Whitepaper) Presentations

Data from more than 75 company-sponsored, investigator-sponsored, and collaborative research abstracts will be presented at ASH (Free ASH Whitepaper), including updated analyses from the pivotal ECHELON-3 trial supporting the clinical benefit of ADCETRIS (brentuximab vedotin) in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). New data for ELREXFIO (elranatamab-bcmm) in relapsed/refractory multiple myeloma (RRMM) will also be presented from the pivotal MagnetisMM-3 trial, as well as Phase 1 combination data from the MagnetisMM-20 trial. Pfizer will also present updates from its growing Hematology-Oncology pipeline, which includes next-generation CD30 antibody-drug conjugates and other novel and differentiated molecules, including the first presentation of combination data for SEA-CD70 in high-risk myelodysplastic syndromes.

Additionally, Pfizer will present results across its portfolio of investigational and approved medicines in benign hematology.

ADCETRIS: Additional analyses from the Phase 3 ECHELON-3 trial will be presented, highlighting the durability of complete responses and consistent benefit of ADCETRIS in combination with lenalidomide and rituximab in patients with relapsed/refractory DLBCL, including enrollment of elderly patients, those who are refractory to most recent treatment, and those who have received prior CAR-T therapy. These findings also underscore the overall survival (OS) advantage over lenalidomide and rituximab plus placebo in patients who have received at least two prior lines of therapy. In addition, updated two-year follow-up data from a Phase 2 study investigating the combination of ADCETRIS, nivolumab, doxorubicin, and dacarbazine in newly diagnosed early-stage classical Hodgkin lymphoma (cHL) will be presented highlighting promising efficacy and safety of this investigational novel combination.
ELREXFIO: A post hoc analysis of the Phase 2 MagnetisMM-3 trial continues to show deep and durable responses after longer-term follow-up of nearly three years, and these responses were also maintained with a reduction to once-monthly dosing in RRMM. Data will also be shared from the ongoing Phase 1b MagnetisMM-20 trial that indicate encouraging clinical efficacy and predictable safety signals with ELREXFIO in combination with carfilzomib and dexamethasone after a median of two prior lines of therapy (range: one to three).
SEA-CD70 (PF-08046040): Encouraging preliminary data from the ongoing Phase 1 study with PF-08046040, also known as SEA-CD70, a nonfucosylated monoclonal antibody targeting CD70 that is designed to enhance effector function, will be shared for the first time from the combination dose-optimization cohort with azacitidine in patients with higher-risk myelodysplastic syndromes (MDS). SEA-CD70 is being developed with the goal of being a best-in-class foundational medicine either alone or as combination treatment in myeloid malignancies.
Key SABCS Presentations

Data from 30 company-sponsored, investigator-sponsored, and collaborative research abstracts will be presented at SABCS, including nine real-world analyses affirming IBRANCE (palbociclib) as a first-line standard-of-care treatment for hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer (MBC). The company will also present new data from its expanding pipeline of innovative, next-generation therapy candidates that have the potential to address critical unmet patient needs across all subtypes and stages of breast cancer, including new and updated Phase 1 data for atirmociclib, vepdegestrant, and the novel KAT6 inhibitor, PF-07248144.

IBRANCE : In P-VERIFY, the largest-ever real-world comparative overall survival analysis of first-line CDK4/6 inhibitors plus aromatase inhibitor (AI) therapy in HR+/HER2- MBC, numerically similar overall survival rates were observed across CDK4/6 inhibitor groups at 12, 24, and 30 months.
Atirmociclib (PF-07220060): Updated data will be presented from a Phase 1/2a study of atirmociclib, a next-generation, highly selective CDK4 inhibitor, in combination with letrozole as a potential first-line treatment for patients with HR+/HER2- MBC. Atirmociclib is being developed as a potential future CDK inhibitor backbone therapy in HR+ MBC, and a Phase 3 study in the first-line setting is anticipated to start by early 2025.
Vepdegestrant: For the first time, initial Phase 1b data will be shared from the Phase 1b/2 TACTIVE-U trial evaluating the combination of vepdegestrant, a potential first-in-class PROteolysis TArgeting Chimera (PROTAC) estrogen receptor (ER) degrader, in combination with abemaciclib in patients with ER+/HER2- locally advanced or MBC. These data reinforce the potential of vepdegestrant as a new backbone endocrine therapy, and Pfizer and Arvinas anticipate topline Phase 3 data evaluating vepdegestrant as a monotherapy in the first quarter of 2025.
KAT6 (PF-07248144) : Updated efficacy and safety data will be presented from a Phase 1 dose-expansion study of PF-07248144, a novel KAT6 inhibitor, in heavily pretreated ER+/HER2- MBC. These early data continue to provide strong clinical proof of concept for this novel target as a potential new treatment approach for ER+/HER2- MBC, and Pfizer anticipates initiating a Phase 3 study for PF-07248144 in the post-CDK4/6 inhibitor setting in mid-2025.
Additional information on key Pfizer-sponsored abstracts at ASH (Free ASH Whitepaper) and SABCS, including date and time of presentation, follow in the chart below. A complete list of Pfizer-sponsored accepted abstracts is available here: View Source

Blood Cancers

Updated Analysis of Brentuximab Vedotin, Nivolumab, Doxorubicin, and Dacarbazine for Nonbulky, Early-Stage Classical Hodgkin Lymphoma (Abstract #460)

Abramson J

Oral Presentation

Sunday, December 8, 9:30-11:00 AM PST

Presentation Time: 10:15 AM PST

Efficacy of Elranatamab (ELRA) in Combination with Carfilzomib (CFZ) and Dexamethasone (DEX) in the Phase 1b MagnetisMM-20 Trial in Relapsed or Refractory Multiple Myeloma (RRMM) (Abstract #1024)

Tomasson H

Oral Presentation

Monday, December 9, 4:30-5:30 PM PST

Presentation Time: 5:15 PM PST

PF-08046040 (SEA-CD70), a Nonfucosylated CD70-Directed Antibody, in Combination with Azacitidine for Patients with Myelodysplastic Syndromes (MDS): A Phase 1 Dose-Finding and Dose Expansion Study (Abstract #1840)

Poster Presentation

Saturday, December 7, 5:30-7:30 PM PST

Durability of Complete Responses in Patients from the ECHELON-3 Study (Abstract #3101)

Yasenchak C

Poster Presentation

Sunday, December 8, 6:00-8:00 PM PST

MagnetisMM-3: Long-Term Update and Efficacy and Safety of Less Frequent Dosing of Elranatamab in Patients with Relapsed or Refractory Multiple Myeloma (Abstract #4738)

Prince M

Poster Presentation

Monday, December 9, 6:00-8:00 PM PST

Outcomes in Older Patients with Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL) from the ECHELON-3 Study (Abstract #4483)

Bartlett N

Poster Presentation

Monday, December 9, 6:00-8:00 PM PST

Outcomes by Refractory Status and Prior Therapies Received in Patients with Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL) from the ECHELON-3 Study (Abstract #4489)

Hahn U

Poster Presentation

Monday, December 9, 6:00-8:00 PM PST

Hemophilia

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults with Moderately Severe to Severe Hemophilia Α: Primary Analysis Results from the Phase 3 AFFINE Gene Therapy Trial (Abstract #1053)

Leavitt AD

Oral Presentation

Monday, December 9, 4:30-6:00 PM PST

Presentation Time: 5:00 PM PST

Descriptive Characterization of Bleeding Events in Participants with Severe Hemophilia Α or B without Inhibitors, Receiving Prophylactic Marstacimab Treatment (Abstract #716)

Matino D

Oral Presentation

Monday, December 9, 10:30 AM-12:00 PM PST

Presentation Time: 10:45 AM PST

Sickle Cell Disease

Qualitative Interview Study to Characterize the Treatment Experiences of Participants with Sickle Cell Disease and Assess Perceptions of Red Blood Cell Transfusions (Abstract #3691)

Kosa K

Poster Presentation

Sunday, December 8, 6:00-8:00 PM PST

Breast Cancer

Comparative overall survival of CDK4/6is plus an aromatase inhibitor (AI) in HR+/HER2- MBC in the US real-world setting

Rugo et al

Poster Spotlight Presentation (PS2-03)

Thursday, December 12, 7:00-8:30 AM CST

PF-07248144, a first-in-class KAT6 inhibitor, in patients with HR+ HER2− metastatic breast cancer: Updated results from phase 1 dose expansion study

Mukohara et al

Poster Presentation (P4-10-28)

Thursday, December 12, 5:30-7:00 PM CST

Vepdegestrant, a PROteolysis TArgeting Chimera (PROTAC) Estrogen Receptor (ER) Degrader, Plus Abemaciclib in ER-Positive/Human Epidermal Growth Factor Receptor 2 (HER2)-Negative Advanced or Metastatic Breast Cancer: TACTIVE-U Preliminary Phase 1b Results

Hilton et al

Poster Presentation ​(P4-12-03)

Thursday, December 12, 5:30-7:00 PM CST

The next-generation CDK4-selective inhibitor atirmociclib (PF-07220060) in combination with letrozole as first-line treatment in patients with HR+/HER2+ metastatic breast cancer

Giordana et al

Poster Presentation (P5-07-28)

Friday, December 13, 12:30-2:00 PM CST

On December 5, 2024 Purple Biotech Ltd. ("Purple Biotech" or the "Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class therapies that overcome tumor immune evasion and drug resistance, reported the closing of its previously announced registered direct offering of 472,668 of the Company’s American Depositary Shares ("ADSs"), each ADS representing 200 ordinary shares, at a purchase price of $6.00 per ADS (Press release, Purple Biotech, DEC 5, 2024, View Source [SID1234648828]).

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H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.

The gross proceeds to the Company from the offering are approximately $2.8 million, before deducting the placement agent’s fees and other offering expenses payable by the Company. Purple Biotech intends to use the net proceeds from the offering to fund the development of its oncology therapeutic candidates and for general working capital and corporate purposes.

The ADSs described above were offered by Purple Biotech pursuant to a "shelf" registration statement on Form F-3 (File No. 333-268710) previously filed with the U.S. Securities and Exchange Commission (the "SEC") on December 8, 2022, and declared effective by the SEC on May 22, 2023. The offering of the ADSs in the offering was made only by means of a prospectus, including a prospectus supplement, forming a part of the effective registration statement. A final prospectus supplement and accompanying prospectus relating to the offering was filed with the SEC. Electronic copies of the final prospectus supplement and accompanying prospectus may be obtained on the SEC’s website at View Source or by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (212) 865-5711 or e-mail at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On December 5, 2024 Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, reported results from its ongoing Phase 2 open-label ADVANCED-2 trial (Press release, Protara Therapeutics, DEC 5, 2024, View Source [SID1234648827]). The trial is assessing intravesical TARA-002, the Company’s investigational cell-based therapy, in high-risk Non-Muscle Invasive Bladder Cancer (NMIBC) patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-Unresponsive or BCG-Naïve. The complete response (CR) rate across BCG exposures was 72% (13/18) at six months and 70% (14/20) at any time with 100% (9/9) of patients maintaining a CR from three months to six months. In addition, two of three patients maintained a CR at nine months. These results will be featured today during a poster session at the 25th Annual Meeting of the Society of Urologic Oncology (SUO) in Dallas, Texas.

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The dataset includes 20 patients who were evaluable at three months, 18 patients who were evaluable at six months and three patients who were evaluable at nine months with a data cutoff of November 19, 2024. In the pivotal cohort of the ADVANCED-2 trial in BCG-Unresponsive patients, the CR rate was 100% (4/4) at six-months and 80% (4/5) at any time. In the proof-of-concept cohort of BCG-Naïve patients, the CR rate was 64% (9/14) at six months and 67% (10/15) at any time. TARA-002 demonstrated a favorable safety and tolerability profile with no Grade 2 or greater treatment-related adverse events (TRAEs), and no patients discontinued due to adverse events.

"These impressive TARA-002 results demonstrate meaningful activity in a difficult to treat patient population," said Brian Mazzarella, MD, Vice President of Research for Urology America, and ADVANCED-2 study investigator. "The activity of TARA-002 across BCG exposures, coupled with its ease of use and low procedural burden for physicians, make it an exciting potential treatment option for NMIBC patients."

"We are thrilled with these positive six-month data, which reinforce TARA-002’s potential in NMIBC, while offering a compelling product profile for physicians and patients," said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. "We believe these encouraging data together with our international site expansion will accelerate patient enrollment, and we look forward to reporting initial data from 12-month evaluable patients in mid-2025."

The majority of adverse events were Grade 1 and transient with no Grade 2 or greater TRAEs as assessed by study investigators. No patients discontinued treatment due to adverse events. The most common adverse events were in line with typical responses to bacterial immunopotentiation, such as flu-like symptoms. The most common urinary symptoms reflect urinary tract instrumentation effects, such as bladder spasm, burning sensation, and urinary tract infection. Most bladder irritations resolved shortly after administration or within a few hours to a few days.

Conference Call and Webcast

Protara will host a conference call and webcast to discuss the data today at 8:30 am ET. The live call can be accessed by registering as a participant here. Upon registration, participants will receive conference call dial-in information. A live webcast of the event can be accessed by visiting the Events and Presentations section of the Company’s website: View Source The webcast will be archived for a limited time following the presentation.

About ADVANCED-2

The Phase 2 open-label ADVANCED-2 trial is assessing intravesical TARA-002 in NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-Unresponsive (n≈100) and BCG-Naïve (n=27). The BCG-Unresponsive cohort has been designed to be registrational in alignment with the FDA’s 2024 BCG-Unresponsive Non-muscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment Draft Guidance for Industry.

About TARA-002

TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil in Japan and approved in Taiwan by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432.

When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma, IL-1b, IL-6, IL-12, granulocyte-macrophage colony-stimulating factor (GM-CSF) and natural killer cells. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer is the sixth most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses. Approximately 65,000 patients are diagnosed with NMIBC in the United States each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle.

On December 5, 2024 NextCure presented its corporate presentation (Presentation, NextCure, DEC 5, 2024, View Source [SID1234648826]).

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On December 5, 2024 Monte Rosa Therapeutics, Inc. (Nasdaq: GLUE), a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, reported an update from its ongoing Phase 1/2 open-label, multicenter study of MRT-2359 in patients with MYC-driven solid tumors (Press release, Monte Rosa Therapeutics, DEC 5, 2024, View Source [SID1234648825]). MRT-2359 is an investigational, orally bioavailable, GSPT1-directed MGD discovered and developed by Monte Rosa Therapeutics.

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"These latest interim results from our ongoing Phase 1/2 study of MRT-2359 continue to indicate a favorable safety profile, and degradation of GSPT1 to desired levels in patients with heavily pretreated, solid tumors, including those that express high levels of MYC. Importantly, we believe the MRT-2359 safety profile supports further clinical development, with no signs of hypotension, cytokine release syndrome (CRS), or clinically significant hypocalcemia observed at any dose level and regimen, all of which have been reported as safety limitations of other GSPT1 degraders. We’re pleased to confirm the selection of 0.5 mg daily at a 21 days on, 7 days off drug dosing schedule as our recommended Phase 2 dose, a schedule that enables dosing of MRT-2359 more than twice as frequently per cycle as compared to the 5 days on, 9 days off regimen previously explored in our study and that we also believe to be more patient compliance-friendly," said Markus Warmuth, M.D., Chief Executive Officer of Monte Rosa Therapeutics. "Trial enrollment has been strong and we are working towards completing the biomarker and activity assessment of our monotherapy dose escalation study using the 21 days on, 7 days off schedule, including backfill cohorts. We have started safety assessments of MRT-2359 in combination with enzalutamide in previously treated metastatic prostate cancer patients as well as with fulvestrant in previously treated metastatic estrogen receptor-positive breast cancer patients. We look forward to providing an update on clinical data from the study as well as plans for potential expansion cohorts in the first quarter of next year."

Summary of Interim Data on Enrollment, Safety & Pharmacodynamics

Enrollment Highlights

•
Patients have been dosed with MRT-2359 in 6 dose levels across two dosing schedules, namely a 5 days on, 9 days off drug (5/9) dosing schedule and a 21 days on, 7 days off drug (21/7) dosing schedule.
•
The study has enrolled patients with a diverse set of tumor types, including non-small cell lung cancer (NSCLC), small cell lung cancer (SCLC), neuroendocrine (NE) tumors of the prostate, bladder and other organs of origin, androgen receptor-positive prostate cancer, and estrogen receptor-positive breast cancer.

Safety Highlights

•
Using the 5/9 dosing schedule, doses of 0.5 mg and 1 mg per day were identified as having a generally favorable safety profile, while doses of 1.5 mg or higher were above the maximum tolerated dose (MTD) with thrombocytopenia being a dose limiting toxicity (DLT).
•
Using the 21/7 schedule, both 0.5 and 0.75 mg were identified as having a generally favorable safety profile.
•
0.5 mg using the 21/7 dose schedule was selected as the recommended phase 2 dose (RP2D) for any expansion cohorts of the Phase 1/2 study.
•
Safety assessments of MRT-2359 in combination with enzalutamide in previously treated metastatic prostate cancer as well as with fulvestrant in previously treated metastatic estrogen receptor- positive breast cancer have been initiated.

Pharmacodynamic Highlights

•
Pharmacodynamic effects were assessed utilizing mass spectrometry measurements of GSPT1 protein levels from paired tumor biopsies. The target levels of approximately 60% GSPT1 degradation were observed in tumor biopsies across all dose levels in relevant tumor types, supporting that the dose of 0.5 mg per day provides optimal degradation consistent with its designed activity based on preclinical studies.

Monte Rosa continues to collect and evaluate clinical results from the MRT-2359 Phase 1/2 study and expects to share updated data, including biomarker and activity data, in Q1 2025.

About MRT-2359

MRT-2359 is a potent, highly selective, and orally bioavailable investigational molecular glue degrader (MGD) that induces the interaction between the E3 ubiquitin ligase component cereblon and the translation termination factor GSPT1, leading to the targeted degradation of GSPT1 protein. The MYC transcription factors (c‑MYC, L-MYC and N-MYC) are well-established drivers of human cancers that maintain high levels of protein translation, which is critical for uncontrolled cell proliferation and tumor growth. Preclinical studies have shown this addiction to MYC-induced protein translation creates a dependency on GSPT1. By inducing degradation of GSPT1, MRT-2359 is designed to exploit this vulnerability, disrupting the protein synthesis machinery, leading to anti-tumor activity in MYC-driven tumors.