On January 9, 2024 Nerviano Medical Sciences S.r.l. (NMS), a part of NMS Group S.p.A. (NMS Group) and Nerviano Medical Sciences, Inc. (NMS-US), a wholly owned subsidiary of NMS Group, focused on the discovery and development of oncology drugs and the largest oncological R&D company in Italy, reported that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for its next-generation FLT3 inhibitor NMS-03592088 for the treatment of Acute Myeloid Leukemia (AML) (Press release, Nerviano Medical Sciences, JAN 9, 2024, View Source [SID1234639161]).

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NMS-03592088 is a potent inhibitor of FLT3, KIT and CSF1R that is being assessed as new therapeutic option for AML patients who are relapsed or refractory after treatment with prior standard of care drugs including prior FLT3 inhibitors. Preclinical characterization demonstrated that NMS-03592088 has superior features with respect to approved FLT3 inhibitor drugs supporting its positioning as next generation FLT3 inhibitor. Notably, in addition to the higher biochemical and cellular potency and superior in vivo efficacy, NMS-03592088 showed potent activity in the presence of the gatekeeper resistance mutation F691L, reported as cause of relapse after treatment with first generation FLT3 inhibitors opening the potential for treatment of patients who failed prior FLT3 inhibitor treatment.

The clinical efficacy of NMS-03592088 is being evaluated as monotherapy in a Phase I/II study in Europe and the US. Preliminary evidence of activity was observed during Phase I dose escalation with efficacy demonstrated in patients who had failed prior standard of care including patients who failed prior FLT3 treatments (LINK: AACR (Free AACR Whitepaper)2023). The trial is currently enrolling patients in Phase II with different FLT3 positive AML settings explored (NCT03922100).

"The orphan drug designation for NMS-03592088 is a significant achievement for NMS. It underscores the potential of NMS-03592088 and our dedication to making a difference in the lives of patients with FLT3 AML. We are proud to be at the forefront of innovation and remain steadfast in our mission to address unmet medical needs" said Lisa Mahnke, MD, PhD, CMO of NMS | CEO and Managing Director of NMS-US.

"Receiving orphan drug designation for NMS-03592088 is a critical milestone in our development journey. This recognition not only highlights the scientific merit of our approach but also reinforces our commitment to bringing novel therapies to those who need them most. We look forward to advancing NMS-03592088 through further clinical development and regulatory milestones" said Elena Ardini, MSc, Asset Leader of NMS.

The FDA’s Office of Orphan Products Development grants ODD status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. ODD provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include potential for market exclusivity for seven years upon FDA approval eligibility for tax credits for qualified clinical trials, and waiver of Prescription Drug User Fee Act Application fee.

On January 9, 2024 Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, and NEC Corporation (NEC; TSE: 6701), a leader in IT, network and AI technologies, reported the signing of a further development collaboration agreement to continue the clinical evaluation of the individualized neoantigen cancer vaccine TG4050 (Press release, NEC, JAN 9, 2024, View Source [SID1234639160]).

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TG4050 is currently being evaluated in a randomized multicenter Phase I trial as a single agent in the adjuvant treatment of HPV-negative head and neck cancers. Based on promising data obtained in this Phase I* trial (new windowNCT04183166), Transgene and NEC are preparing a randomized Phase I/II extension of this trial slated to start in 2024. This new trial builds on compelling first signs of efficacy and induction of specific T-cell responses with the aim of generating a comprehensive set of immunological and clinical data to further demonstrate the potential of TG4050.

Transgene and NEC expect to present additional immunological and clinical data from the Phase I trial at a scientific conference in the first half of 2024.

TG4050 is based on Transgene’s viral vector based myvac platform and powered by NEC’s cutting-edge AI capabilities for the identification and prediction of the most immunogenic neoantigens.

Alessandro Riva, Chairman and CEO of Transgene, commented: "We are pleased to announce the extension of our agreement with NEC, which marks a significant milestone in our collaboration. We are looking forward to continuing to treat patients with our individualized cancer vaccine TG4050. The compelling initial Phase I data presented with NEC at ASCO (Free ASCO Whitepaper) 2023 showed that all evaluable patients treated with TG4050 monotherapy developed a specific immune response and remained disease-free.

"Our joint clinical development plan builds on these promising data in a setting where there is no approved treatment to prevent patient relapse after adjuvant chemoradiotherapy. We believe that TG4050, by combining a powerful and immunogenic viral vector with an extremely sophisticated neoantigen selection tool, has the potential to address major medical needs in the adjuvant treatment of solid tumors."

Masamitsu Kitase, Corporate SVP, Head of Healthcare and Life Sciences Division, NEC Corporation, commented: "Transgene has been our trusted partner in developing our joint neoantigen asset TG4050. I am excited that the positive results from the Phase I study have encouraged us to further collaborate on this very promising asset for treating head and neck cancers. We are happy that our state-of-the-art artificial intelligence (AI)/ machine learning (ML) models help in predicting clinically meaningful neoantigens which impact patient outcomes. NEC’s Healthcare and Life Sciences Division is committed to bringing novel AI-based treatments to patients across the globe and achieving meaningful advances in the pharmaceutical industry."

About myvac
myvac is a viral vector (MVA – Modified Vaccinia Ankara) based, individualized immunotherapy platform that has been developed by Transgene to target solid tumors. myvac-derived products are designed to stimulate the patient’s immune system to recognize and destroy tumors using their own cancer specific genetic mutations. Transgene has set up an innovative network that combines bioengineering, digital transformation, established vectorization know-how and unique manufacturing capabilities. Transgene has been awarded "Investment for the Future" funding from Bpifrance for the development of its platform myvac. TG4050 is the first myvac-derived product being evaluated in clinical trials.
Click new windowhere to watch a short video on myvac.

About TG4050
TG4050 is an individualized immunotherapy being developed for solid tumors that is based on Transgene’s myvac technology and powered by NEC’s longstanding artificial intelligence (AI) and machine learning (ML) expertise. This virus-based therapeutic vaccine encodes neoantigens (patient-specific mutations) identified and selected by NEC’s Neoantigen Prediction System. The prediction system is based on more than two decades of expertise in AI and has been trained on proprietary data allowing it to accurately prioritize and select the most immunogenic sequences.
TG4050 is designed to stimulate the immune system of patients in order to induce a T-cell response that is able to recognize and destroy tumor cells based on their own neoantigens. This individualized immunotherapy is developed and produced for each patient.

About the clinical trial
TG4050 is being evaluated in a Phase I clinical trial for patients with HPV-negative head and neck cancers (new windowNCT04183166). An individualized treatment is created for each patient after they complete surgery and while they receive adjuvant therapy. Half of the participants received their vaccine immediately after completing adjuvant treatment. The other half were given TG4050 as an additional treatment at the time of recurrence of the disease as an additional treatment to standard of care (SoC). This randomized study is evaluating the treatment benefits of TG4050 in patients who are at risk of relapse. Thirty-two patients have been included in this trial under way in France, the UK, and the USA. The principal investigator of the trial is Prof. Christian Ottensmeier, MD, PhD, Consultant Medical Oncologist at the Clatterbridge Cancer Centre and Professor of Immuno-Oncology at the University of Liverpool. In France, the clinical trial is conducted at Institut Curie by Prof. Christophe Le Tourneau, MD, PhD, Head of the Department of Drug Development and Innovation (D3i), and at the IUCT-Oncopole, Toulouse by Prof. Jean-Pierre Delord, MD, PhD. In the USA, the trial is being led by Yujie Zhao, MD, PhD, at the Mayo Clinic. Endpoints of the trial include safety, feasibility, and biological activity of the therapeutic vaccine. Initial immunological and clinical data presented at AACR (Free AACR Whitepaper) 2023 and ASCO (Free ASCO Whitepaper) 2023 are very encouraging.

On January 9, 2024 MimiVax, Inc., a biotechnology company focused on the development of SurVaxM, a survivin-targeted vaccine therapy for glioblastoma and other cancers, reported new investments from the venture philanthropy arms of leading brain tumor patient organizations, VC, private and existing investors (Press release, MimiVax, JAN 9, 2024, View Source;utm_medium=rss&utm_campaign=new-investments-in-mimivax [SID1234639159]).

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The investments from the Brain Tumor Investment Fund (BTIF), an affiliate of the National Brain Tumor Society (NBTS), the Sontag Innovation Fund, LLC, a subsidiary of the Sontag Foundation, MEDA Angels, LLC, Varia Ventures and private investors will support the ongoing phase 2b clinical study, subsequent registration-related work and prepare the company for commercialization of SurVaxM for ndGBM. Glioblastoma is a rare but deadly cancer with median overall survival of only about 16 months. SurVaxM was developed to bring a paradigm shift to a field that has seen little advancement in decades.

MimiVax is rapidly enrolling subjects in its SURVIVE trial [NCT05163080], a randomized, blinded, placebo-controlled Phase 2b clinical trial of SurVaxM among patients with ndGBM at 11 cancer centers across the USA. This follows the successful Phase 2a study published in the Journal of Clinical Oncology which met its primary endpoint, achieving a median overall survival (mOS) of 25.9 months – and found that 41% of the patients receiving SurVaxM survived at least 3 years, with several long term progression-free survivors.

Investment funding is designed to help MimiVax achieve its goal of revolutionizing the landscape of brain tumor treatment. "We are honored to receive significant investments from the Brain Tumor Investment Fund, the Sontag Innovation Fund, MEDA Angels, Varia Ventures and our private investors. "Their confidence in our mission further propels our efforts to make a meaningful impact in the lives of those affected by brain tumors," said Michael Ciesielski, CEO, MimiVax, Inc.

The Brain Tumor Investment Fund, known for its dedication to funding pioneering solutions in neuro-oncology, expressed enthusiasm about the partnership. "Brain tumor patients and caregivers desperately need better outcomes. The results seen in previous trials of SurVaxM make us optimistic for the SURVIVE Phase 2b trial and the potential for a new treatment on the horizon" said John Higgins, Managing Director of BTIF.

MEDA Angels, (part of the Tech Coast Angels (TCA) network) are a health-care focused investor group founded by physician, payers and scientist-angel investors, supported by a team of regulatory, reimbursement, legal and financial advisors. "We knew the grave unmet need for game-changing therapy for glioblastoma, a devasting brain cancer with median overall survival of just 16 months. After review of MimiVax’ approach, novel survivin targeting and strong data through their Phase 2a, we became confident they were well worth backing." -MEDA Angels

The Sontag Innovation Fund, building on the reputation of the Sontag Foundation as one of the largest private funders of brain tumor research in North America, is proud to have MimiVax as a portfolio company. "There is an extraordinary unmet need for new treatments that make a major impact in the brain tumor space. We are excited to play a role in the development of promising therapies like SurVaxM" said Scott Davis, Managing Director of the Sontag Innovation Fund.

MimiVax extends its gratitude to the Brain Tumor Investment Fund, the Sontag Innovation Fund, MEDA Angels, Varia Ventures and our private investors for their support and confidence in the company’s vision. This investment fortifies MimiVax’s position as a key player in the quest for innovative solutions in the fight against brain tumors.

On January 9, 2024 Keros Therapeutics, Inc. ("Keros" or "we") (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta ("TGF-ß") family of proteins, reported the closing of the previously announced underwritten public offering of 4,025,000 shares of common stock on January 8, 2024, at a public offering price of $40.00 per share, inclusive of the underwriters’ exercise in full of their option to purchase up to an additional 525,000 shares of common stock at the public offering price (the "Option") (Press release, Keros Therapeutics, JAN 9, 2024, View Source [SID1234639158]). The net proceeds to Keros from the offering, before deducting offering expenses, were approximately $151.3 million. All of the common stock was offered by Keros. Based on its current operating plan, as a result of the full exercise of the Option, Keros believes that the net proceeds from this offering, together with its cash and cash equivalents as of September 30, 2023 and net proceeds of approximately $68.5 million from the sale of shares of its common stock pursuant to its at-the-market sales agreement subsequent to September 30, 2023, will enable Keros to fund its planned operating expenses and capital expenditure requirements into 2027.

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Goldman Sachs & Co. LLC, Leerink Partners, Piper Sandler and Truist Securities acted as joint book-running managers for the offering.

The offering was made pursuant to a shelf registration statement on Form S-3 that was filed with the Securities and Exchange Commission ("SEC") on May 3, 2021 and was effective upon filing. A final prospectus supplement and accompanying prospectus relating to the offering was filed with the SEC on January 4, 2024, and is available on the SEC’s website at www.sec.gov. Copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained by visiting the SEC’s website or by contacting: Goldman Sachs & Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, by telephone at (866) 471-2526 or by email at [email protected]; Leerink Partners LLC, Attention: Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, by telephone at 1-800-808-7525, ext. 6105, or by email at [email protected]; Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, Minnesota 55402, by telephone at (800) 747-3924, or by email at [email protected]; or Truist Securities, Inc., Attention: Prospectus Department, 3333 Peachtree Road NE, 9th floor, Atlanta, GA 30326, by telephone at (800) 685-4786, 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 offer or 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 such state or jurisdiction.

On January 9, 2024 Illumina, Inc. (Nasdaq: ILMN) ("Illumina" or the "company") reported unaudited preliminary financial results for the fourth quarter and fiscal year 2023 ahead of its presentation at the 42nd Annual J.P. Morgan Healthcare Conference on January 9, 2024 at 9:00am Pacific Time (12:00pm Eastern Time) (Press release, Illumina, JAN 9, 2024, View Source [SID1234639157]). The webcast can be accessed through the Investor Info section of Illumina’s website at investor.illumina.com.

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Preliminary financial results
•Consolidated revenue of approximately $1,115 million for Q4 2023, up 3% from Q4 2022, and approximately $4,497 million for fiscal year 2023, down 2% from fiscal year 2022
•Core Illumina revenue of approximately $1,090 million for Q4 2023, up 2% from Q4 2022, and approximately $4,431 million for fiscal year 2023, down 3% from fiscal year 2022
•Shipped 79 NovaSeq X instruments in Q4 2023 and 352 instruments for fiscal year 2023
•Consolidated GAAP operating margin of approximately (15.5%) for Q4 2023 and approximately (24.0%) for fiscal year 2023
•Consolidated non-GAAP operating margin of approximately 3.8% for Q4 2023 and approximately 5.3% for fiscal year 2023
•Core Illumina GAAP operating margin of approximately 2.4% for Q4 2023 and approximately 12.3% for fiscal year 2023
•Core Illumina non-GAAP operating margin of approximately 18.0% for Q4 2023 and approximately 19.8% for fiscal year 2023

As previously announced, the company expects to report its full fourth quarter and fiscal year 2023 results following the close of market on Thursday, February 8, 2024. The unaudited results in this press release are preliminary and subject to the completion of accounting and annual audit procedures and are therefore subject to adjustment.

Statement regarding use of non-GAAP financial measures
The company reports non-GAAP results for diluted earnings per share, net income, gross margin, operating expenses, including research and development expense, selling general and administrative expense, and from time to time, as applicable, legal contingencies and settlement, and goodwill and intangible impairment, operating income (loss), operating margin, gross profit (loss), other income (expense), tax provision, constant currency revenue growth, and free cash flow (on a consolidated and, as applicable, segment basis for our Core Illumina and GRAIL segments) in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The company’s financial measures under GAAP include substantial charges such as amortization of acquired intangible assets among others that are listed in the itemized reconciliations between GAAP and non-GAAP financial measures included in this press release, as well as the effects of currency translation. Management has excluded the effects of these items in non-GAAP measures to assist investors in analyzing and assessing past and future operating performance, including in the non-GAAP measures related to our Core Illumina and GRAIL segments. Additionally, non-GAAP net income and diluted earnings per share are key components of the financial metrics utilized by the company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation.

The company encourages investors to carefully consider its results under GAAP, as well as its supplemental non-GAAP information and the reconciliation between these presentations, to more fully understand its business. Reconciliations between GAAP and non-GAAP results are presented in the tables of this release.