On December 9, 2022 The Janssen Pharmaceutical Companies of Johnson & Johnson reported the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for talquetamab for the treatment of patients with relapsed or refractory multiple myeloma (Press release, Johnson & Johnson, DEC 9, 2022, View Source [SID1234624998]). Talquetamab is an investigational, off-the-shelf (ready to use), bispecific T-cell engager antibody targeting both GPRC5D, a novel drug target that is on some normal cells but overexpressed on myeloma cells, and separately targets CD3 on T cells.1

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"Despite the therapies that have been developed for the treatment of multiple myeloma, there remains persistent unmet needs for patients who relapse or become refractory," said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. "Through our discovery and development of talquetamab, a novel GPRC5DxCD3 bispecific antibody, we remain relentlessly committed to the investigation of innovative therapies for patients and oncologists. We look forward to working closely with the FDA in their review of the talquetamab submission."

This BLA is supported by data from the Phase 1/2, first-in-human MonumenTAL-1 study of talquetamab (Phase 1: NCT03399799; Phase 2: NCT04634552) in patients with relapsed or refractory multiple myeloma who have received more than three prior lines of therapy.1 The first presentation of Phase 2 results from the MonumenTAL-1 study will be highlighted at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 10, 2022 in an oral scientific session (Abstract #157)2 and featured as part of the ASH (Free ASH Whitepaper) Press Briefing.

About Talquetamab
Talquetamab is a first-in-class, off-the-shelf (ready to use), investigational bispecific T-cell engager antibody targeting both GPRC5D, a novel multiple myeloma target, and CD3, a primary component of the T-cell receptor.1 CD3 is involved in activating T cells, and GPRC5D is highly expressed on multiple myeloma cells.3,4

Talquetamab, which is given by subcutaneous injection, is currently being evaluated in the Phase 1/2 MonumenTAL-1 clinical study for the treatment of relapsed or refractory multiple myeloma (NCT03399799), and in combination studies RedirecTT-1 (NCT04586426), TRIMM-2 (NCT04108195), TRIMM-3 (NCT05338775), MonumenTAL-2 (NCT05050097) and MonumenTAL-3 (NCT05455320).

Talquetamab received Breakthrough Therapy designation from the U.S. FDA in June 2022 for the treatment of adult patients with relapsed or refractory multiple myeloma, who have previously received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. In May 2021 and August 2021, talquetamab received an Orphan Drug designation for the treatment of multiple myeloma by the U.S. FDA and the European Commission, respectively. In January 2021, talquetamab received a PRIME designation by the European Commission.

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.5 In multiple myeloma, these plasma cells change, spread rapidly and replace normal cells in the bone marrow with tumors.6 In 2022, it is estimated that more than 34,000 people will be diagnosed with multiple myeloma, and more than 12,000 people will die from the disease in the U.S.7 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems or infections.

On December 9, 2022 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA testing, reported that the publication of a new study in JCO Precision Oncology highlighting the clinical utility of its personalized and tumor-informed molecular residual disease test, Signatera, for postoperative risk stratification and prediction of recurrence in patients with stage I-III esophageal and gastric cancers (EGCs) (Press release, Natera, DEC 9, 2022, View Source [SID1234624997]). The full study can be found here.

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EGCs are the sixth most common cancers worldwide,1 affecting approximately 47,000 new patients per year in the U.S.2 In patients with localized disease, despite treatment with curative-intent therapy, over 50% recur within three years.3-5 Today, clinical practice guidelines support either adjuvant therapy or observation post surgery, so there is a strong unmet need for better risk stratification tools to help inform these risk-based management decisions. In addition, due to the high rate of recurrence, guidelines recommend frequent monitoring for recurrence with imaging, endoscopic evaluation or tumor markers.6

This real-world study, one of the largest reported EGC studies to date, included 943 plasma samples collected from 295 patients across more than 70 institutions. The primary analysis focused on the 212 patients with stage I-III disease. Signatera ctDNA status was evaluated at diagnosis (prior to neoadjuvant treatment), post surgery and then serially during routine surveillance, with median clinical followup of 417 days.

Key takeaways from the study include:

Pre-treatment: ctDNA was detectable in 96% (23/24) of patients with preoperative samples.
Post surgery (within 16 weeks): ctDNA was detected post-surgically in 23.5% (16/68) of patients. ctDNA-positive patients experienced a higher rate of recurrence (81.2%) in comparison to ctDNA-negative patients (13.5%). ctDNA-positive patients experienced inferior RFS (HR: 10.7, 95% CI: 4.3-29.3, p<0.0001).
Surveillance setting (at least 2 weeks after completion of adjuvant treatment): the recurrence rate in patients with a ctDNA-positive result was 95.2% (20/21) compared to 7.9% (5/63) in patients who remained ctDNA-negative. ctDNA-positive patients experienced inferior RFS (HR: 17.7, 95% CI: 7.3-50.7, p<0.0001).
Multivariate analysis: ctDNA was the strongest prognostic factor compared to all other clinicopathological risk factors including disease stage, location, and MSI status (HR: 11.82, 95% CI: 6.18-22.6, p<0.001).
"This study shows how longitudinal assessment using Signatera allows for accurate post operative risk stratification and adjuvant therapy or surveillance monitoring in patients with esophagogastric cancers in a real-world setting," said Samuel Klempner, M.D., associate professor at Massachusetts General Hospital and corresponding author of the study. "Unfortunately, many gastroesophageal cancers recur after definitive treatment, and patients with advanced disease have a poor prognosis. This study highlights the potential for Signatera to better identify patients at highest risk of recurrence and help develop novel treatment approaches."

About Signatera

Signatera is a custom-built circulating tumor DNA (ctDNA) test for treatment monitoring and molecular residual disease (MRD) assessment in patients previously diagnosed with cancer. The test is available for both clinical and research use, and has been granted three Breakthrough Device Designations by the FDA for multiple cancer types and indications. The Signatera test is personalized and tumor-informed, providing each individual with a customized blood test tailored to fit the unique signature of clonal mutations found in that individual’s tumor. Signatera is intended to detect and quantify cancer left in the body, at levels down to a single tumor molecule in a tube of blood, to identify recurrence earlier and to help optimize treatment decisions.

On December 9, 2022 UroGen Pharma Ltd. (Nasdaq: URGN) a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported the grants of inducement restricted stock units ("RSUs") to 12 new employees in connection with their employment with UroGen (Press release, UroGen Pharma, DEC 9, 2022, View Source [SID1234624996]). These new team members will support the ongoing commercial launch of Jelmyto (mitomycin) for pyelocalyceal solution, UroGen’s first approved product, and the continued development of the Company’s pipeline.

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Up to 20,800 shares of UroGen’s common stock are issuable upon the vesting and settlement of the RSUs. The RSUs will vest equally over three years, with one third of the underlying shares vesting each year on the anniversary of the vesting date, subject in each case to the employee’s continued service relationship with UroGen.

The RSUs are subject to the terms and conditions of UroGen’s 2019 Inducement Plan and RSU grant notice and agreement thereunder. The RSU grants were granted as an inducement material to each employee entering into employment with UroGen in accordance with Nasdaq listing Rule 5635(c)(4).

On December 6, 2022 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel small-molecule therapeutics to benefit people with diseases of the immune system, reported that it has commenced an underwritten public offering of shares of its common stock (or pre-funded warrants to purchase its common stock in lieu thereof) and accompanying warrants to purchase shares of its common stock (or pre-funded warrants) (Press release, X4 Pharmaceuticals, DEC 9, 2022, View Source [SID1234624995]). In addition, X4 expects to grant the underwriters a 30-day option to purchase additional shares of its common stock and/or warrants to purchase shares of its common stock at the public offering price, less the underwriting discounts and commissions. All of the securities in the offering will be sold by X4. 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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Piper Sandler, Stifel and Cantor are acting as joint book-running managers for the offering.

The offering will be made only by means of a written prospectus and related prospectus supplement forming part of a shelf registration statement on Form S-3 that was originally filed with the Securities and Exchange Commission (SEC) on October 19, 2020 and declared effective by the SEC on October 26, 2020. A preliminary prospectus supplement and accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available at the SEC’s website located at www.sec.gov, copies of which may be obtained, when available, from: Piper Sandler & Co., 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, Attention: Prospectus Department, by telephone at (800) 747-3924 or by email at [email protected]; Stifel, Nicolaus & Company, Incorporated, Attention: Syndicate, One Montgomery Street, Suite 3700, San Francisco, California 94104, or by telephone at 415-364-2720 or by email at [email protected]; or Cantor Fitzgerald & Co., Attention: Capital Markets, 499 Park Avenue, 4th Floor, New York, New York 10022, or by email at [email protected].

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

On December 9, 2022 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported a clinical collaboration with Gilead Sciences, Inc. (Nasdaq: GILD) to evaluate the safety and anti-leukemia activity of pivekimab sunirine (pivekimab) in combination with magrolimab, a potential, first-in-class, investigational CD47 inhibitor, in patients with relapsed or refractory (R/R) CD123-positive acute myeloid leukemia (AML) (Press release, ImmunoGen, DEC 9, 2022, View Source [SID1234624994]).

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"AML is the most common form of acute leukemia among adults and accounts for the largest number of deaths from leukemia in both the United States and Europe," said Anna Berkenblit, MD, Senior Vice President and Chief Medical Officer of ImmunoGen. "Given the potentially complementary mechanisms of action, the limited overlap in safety profiles, and the encouraging data seen with each agent in combinations to date, we are excited to explore this novel doublet in patients with relapsed/refractory AML, where few effective treatment options exist."

Expected to initiate in 2023, the collaboration will be a new cohort in ImmunoGen’s 802 study and will evaluate pivekimab in combination with magrolimab in up to 42 patients with R/R CD123-positive AML. The primary endpoint for this cohort is complete response (CR) rate. ImmunoGen’s 802 study is an open-label, multicenter, Phase 1b/2 trial to determine the safety and tolerability of pivekimab and assess the anti-leukemia activity of the agent when administered in combination with Vidaza (azacitidine) and/or Venclexta (venetoclax) in patients with relapsed and frontline CD123-positive AML.

ABOUT PIVEKIMAB SUNIRINE

Pivekimab sunirine is a CD123-targeting ADC in clinical development for hematological malignancies, including blastic plasmacytoid dendritic cell neoplasm (BPDCN), acute myeloid leukemia (AML), and other CD123+ hematologic malignancies. Pivekimab is currently being evaluated as monotherapy for patients with BPDCN and in combination with Vidaza (azacitidine) and Venclexta (venetoclax) for patients with untreated and relapsed/refractory AML. Pivekimab uses one of ImmunoGen’s novel indolinobenzodiazepine (IGN) payloads, which alkylate DNA and cause single strand breaks without crosslinking. IGNs are designed to have high potency against tumor cells, while demonstrating less toxicity to normal marrow progenitors than other DNA-targeting payloads. The European Medicines Agency (EMA) granted orphan drug designation to pivekimab for the treatment of BPDCN in June 2020. Pivekimab also holds this designation in the U.S. In October 2020, the FDA granted pivekimab Breakthrough Therapy designation in relapsed/refractory BPDCN.

ABOUT MAGROLIMAB

Magrolimab is a potential, first-in-class, investigational treatment designed to inhibit the CD47 protein. CD47 binds to receptors on macrophages to transmit a "don’t eat me" signal used by cancer cells to escape natural cell death, called phagocytosis. Targeting the CD47 protein, magrolimab is intended to restore phagocytosis. Magrolimab is being studied in several hematologic cancers as well as solid tumor malignancies. Granted Fast Track Designation by the FDA for the treatment of myelodysplastic syndrome (MDS), AML, diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma, magrolimab has also been granted Orphan Drug Designation by the FDA for MDS and AML and by the European Medicines Agency for AML. More information about clinical trials with magrolimab is available at www.clinicaltrials.gov.

Magrolimab is an investigational product and is not approved by any regulatory authority for any use; its safety and efficacy have not been established.

ABOUT ACUTE MYELOID LEUKEMIA (AML)

AML is a cancer of the bone marrow cells that produce white blood cells. It causes the marrow to increasingly generate abnormal, immature white blood cells (blasts) that do not mature into effective infection-fighting cells. The blasts quickly fill the bone marrow, impacting the production of normal platelets and red blood cells. The resulting deficiencies in normal blood cells leave the patient vulnerable to infections, bleeding problems, and anemia. It is estimated that, in the U.S. alone, more than 20,000 people will be diagnosed with AML this year and more than 11,000 will die from the disease.