Interferon-α(Avadel Pharmaceuticals Plc)

-- Two oral presentations and one poster will feature new data from the Company’s hepatitis B/hepatitis D entry inhibitor, capsid assembly modulator ABI-4334, and interferon-alpha receptor agonist programs -- SOUTH SAN FRANCISCO, Calif., Sept. 19, 2023 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative antiviral therapeutics targeting serious viral diseases, today announced that the company will present new preclinical data from multiple hepatitis B virus (HBV) and hepatitis D virus (HDV) pipeline programs at the 2023 International HBV Meeting taking place in Kobe, Japan, September 19-23, 2023. At the meeting, one oral presentation will highlight progress in the company’s HBV/HDV oral entry inhibitor program including activity, selectivity and pharmacokinetic (PK) data that support advancement towards development candidate nomination. A second oral presentation reports new data for ABI-4334 (4334), Assembly Bio’s most potent capsid assembly modulator (CAM) with a potential best-in-class profile, describing the prevention of HBV DNA integration in vitro. The poster presentation, from the company’s interferon-α receptor (IFNAR) agonist program, characterizes small molecule IFNAR agonists that closely mimic interferon-α’s signaling. “The data we are presenting at the 2023 International HBV Meeting highlight the exciting progress of our preclinical HBV/HDV entry and IFNAR agonist programs, and we anticipate nominating a development candidate from the HBV/HDV entry inhibitor program this year,” said William Delaney, PhD, chief scientific officer of Assembly Bio. “Further, the data reported for 4334 support its potential to prevent HBV DNA integration, a process that has been linked to substantial genetic damage and the development of liver cancer in HBV patients. HBV DNA integration is a pathogenic process that is not directly addressed by nucleos(t)ide analogs and further supports the rationale of targeting the viral core protein through a new class of inhibitors.” HBV/HDV Entry Inhibitor Program HDV is a satellite virus only found in the presence of HBV infection and is considered the most severe form of viral hepatitis. In an oral presentation entitled “Pre-clinical profiling of a novel class of orally bioavailable small molecules potently inhibiting hepatitis B and D virus entry,” the company will present data on a novel class of highly potent, orally bioavailable HBV/HDV entry small molecule inhibitors with favorable drug-like properties. One compound selected for further characterization exhibited potent activity against multiple HBV and HDV genotypes and selective inhibition of sodium taurocholate co-transporting polypeptide (NTCP) compared to other bile acid transporters. This compound further exhibited a favorable PK/pharmacodynamic preclinical pro the potential for once daily dosing. Assembly Bio anticipates nominating a clinical development candidate from the HBV/HDV entry inhibitor program in 2023. Next-Generation HBV CAM Candidate ABI-4334 4334, which has completed Phase 1a evaluation, is a novel, orally bioavailable investigational next-generation CAM that exhibits nanomolar (nM) potency against pgRNA encapsidation and covalently closed circular (ccc)DNA formation in vitro. In patients with chronic HBV infection, HBV DNA integration has been linked to the development of liver cancer. In the oral presentation entitled “ABI-4334, a novel inhibitor of hepatitis B virus core protein, disrupts DL-DNA containing capsids and prevents HBV DNA integration,” data show that in vitro 4334 disrupts RC (relaxed circular)- and DL (duplex linear)-DNA capsid formation at nM levels and inhibits HBV DNA integration in a dose-proportional manner as shown by inverse PCR and next-generation sequencing analyses​. Based on the Phase 1a PK data, the target plasma levels of 4334 required for inhibition of HBV DNA integration in these models are achievable. Interferon-α Receptor Agonist Assembly Bio’s IFNAR agonist program seeks to engage IFNα signaling using an orally bioavailable liver-focused small molecule approach to improve tolerability. The poster entitled “Pre-clinical characterization of novel liver-focused small molecules efficiently inhibiting hepatitis B virus by activating type I interferon signaling,” features preclinical data on a novel class of IFNAR agonists that inhibit HBV infection and replication. In vivo analysis of a compound from the series demonstrates that it closely mimics IFNα activity as measured by activating IFN-stimulated response element signaling and inducing the JAK-STAT pathway in the liver and PBMCs. PK data from the same compound exhibit desirable liver exposure and favorable oral bioavailability. Lead optimization of multiple IFNAR agonists is ongoing. Time and location of the presentations are as follows: HBV/HDV Entry Inhibitor: Oral Presentation: Pre-clinical profiling of a novel class of orally bioavailable small molecules potently inhibiting hepatitis B and D virus entry Presenter: Marc P. Windisch, PhD, Assembly Bio Session: Session IV: Drug discovery in preclinical models Date and Time: September 20, 3:30-5:15 PM JST ABI-4334: Oral Presentation: ABI-4334, a novel inhibitor of hepatitis B virus core protein, disrupts DL-DNA containing capsids and prevents HBV DNA integration Presenter: Nuruddin Unchwaniwala, PhD, Assembly Bio Session: Session VIIII: Integration, pathogenesis, and HCC Date and Time: September 22, 11 AM-1 PM JST Interferon-α Receptor Agonist: Poster Presentation: Pre-clinical characterization of novel liver-focused small molecules efficiently inhibiting hepatitis B virus by activating type I interferon signaling Presenter: Marc P. Windisch, PhD, Assembly Bio Session: Session IV: Drug discovery in preclinical models Date and Time: September 20, 5:15-7:15 PM JST Assembly Bio intends to make the presentations available on the “Events & Presentations” page in the “Investors” section of its website at About Assembly Biosciences Assembly Biosciences is a biotechnology company dedicated to the development of innovative small molecule antiviral therapeutics designed to change the path of serious viral diseases and improve the lives of patients worldwide. Led by an accomplished team of leaders in virologic drug development, Assembly Bio is committed to improving outcomes for patients struggling with the serious, chronic impacts of herpesvirus, hepatitis B virus (HBV) and hepatitis delta virus (HDV) infections. For more information, visit assemblybio.com. Forward-Looking Statements The information in this press release contains forward-looking statements that are subject to certain risks and uncertainties that could cause actual results to materially differ. These risks and uncertainties include: Assembly Bio’s ability to maintain financial resources necessary to continue its clinical studies and fund business operations; Assembly Bio’s ability to initiate and complete clinical studies involving its therapeutic product candidates, including studies contemplated by Assembly Bio’s collaboration agreements, in the currently anticipated timeframes; safety and efficacy data from clinical or nonclinical studies may not warrant further development of Assembly Bio’s product candidates; clinical and nonclinical data presented at conferences may not differentiate Assembly Bio’s product candidates from other companies’ candidates; results of nonclinical studies may not be representative of disease behavior in a clinical setting and may not be predictive of the outcomes of clinical studies; and other risks identified from time to time in Assembly Bio’s reports filed with the U.S. Securities and Exchange Commission (the SEC). You are urged to consider statements that include the words may, will, would, could, should, might, believes, hopes, estimates, projects, potential, expects, plans, anticipates, intends, continues, forecast, designed, goal or the negative of those words or other comparable words to be uncertain and forward-looking. Assembly Bio intends such forward-looking statements to be covered by the safe harbor provisions contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. More information about Assembly Bio’s risks and uncertainties are more fully detailed under the heading “Risk Factors” in Assembly Bio’s filings with the SEC, including its most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Except as required by law, Assembly Bio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Contacts Investor and Corporate: Shannon Ryan SVP, Investor Relations, Corporate Affairs and Alliance Management (415) 738-2992 sryan@assemblybio.com Media: Sam Brown Inc. Hannah Hurdle (805) 338-4752 ASMBMedia@sambrown.com

Company Selects Refractory Advanced Genitourinary Malignancies including Renal Cell Cancer as Second IndicationCompany Selects Refractory Advanced Genitourinary Malignancies including Renal Cell Cancer as Second Indication MILAN, Italy and NEW YORK, July 28, 2023 (GLOBE NEWSWIRE) -- Genenta Science (NASDAQ: GNTA), a clinical-stage immuno-oncology (I/O) company developing a cell-based platform harnessing the power of hematopoietic stem cells to provide durable and safe treatments for solid tumors, today announced that: the Phase 1 dose-ranging clinical trial in Glioblastoma Multiforme (TEM1-GBM) is progressing in line with the development program. All the patients assigned to Cohort 7 (3x10E/kg Temferon™) are dosed.Enrollment of the patients in Cohort 8 (4x10E/kg) is completed.No dose-limiting toxicity has been observed at this point.The median Overall Survival (OS) is currently equal to 17 months. We have selected Refractory Advanced Genitourinary Malignancies including Renal Cell Cancer (RCC) as the second solid tumor indication for Temferon. Late-stage RCC is considered an orphan disease with an unfavorable prognosis for Stage IV RCC patients. The median OS in high-risk patients is only 6 months with the current standard of care.20% of newly diagnosed patients have metastatic disease.Targeted release of IFN-α (Interferon alpha), which is the anti-tumor payload of our platform, we believe the use of a cell-based delivery mechanism is an innovative and clinically relevant approach to modulating the immune microenvironment in patients with immunogenic malignancies, such as metastatic RCC or Urogenital Cancer (UC).IFN efficacy has been established in UC and immunotherapy is also now well established in this type of cancer, offering the potential opportunity of using Temferon in combination with other I/O products such as checkpoint inhibitors. “We believe the clinical data we are observing in the GBM trial on safety, tolerability, and biological activity allow us to progress the expansion of our platform,” said Pierluigi Paracchi, Chief Executive Officer of Genenta. "The use of Temferon for the treatment of Urogenital Cancer patients is an important step for validating in humans the agnostic nature of our product that has already been demonstrated in several tumor animal models”, continued Pierluigi Paracchi. Temferon is Genenta's product at the most advanced stage of development and consists of the patient's own stem progenitor cells modified with Genenta's platform to express Interferon Alpha (IFN-α) within solid tumors. IFN-α is a well-known immunomodulatory protein that has been used in the clinic for decades for the treatment of a variety of cancers, but with limited current use because of the systemic toxicity. Genenta's platform is designed to avoid systemic toxicity and selectively deliver therapeutic activity within the solid tumor. From pre-clinical experiments, it has been observed that Temferon breaks tumor-induced tolerance, thus allowing the immune system to recognize the tumor and mount a durable immune response. About Genenta and TemferonGenenta (www.genenta.com) is a clinical-stage biotechnology company engaged in the development of a proprietary hematopoietic stem cell therapy for the treatment of a variety of solid tumor cancers. Temferon™ is based on ex-vivo gene transfer into autologous Tie2+ hematopoietic stem/progenitor cells (HSPCs) to deliver immunomodulatory molecules directly via tumor-infiltrating monocytes/macrophages (Tie2 Expressing Monocytes - TEMs). Temferon, which is under investigation in a phase 1/2a clinical trial in newly diagnosed Glioblastoma Multiforme patients who have an unmethylated MGMT gene promoter (uMGMT-GBM), is designed to reach solid tumors, induce a durable immune response not restricted to pre-selected tumor antigens nor type, and avoid systemic toxicity, which are some of the main unresolved challenges in immuno-oncology. Forward-Looking StatementsStatements in this press release contain “forward-looking statements,” within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, that are subject to substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as “anticipate,” “believe,” “contemplate,” “could,” “estimate,” “expect,” “intend,” “seek,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “suggest,” “target,” “aim,” “should,” "will,” “would,” or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on Genenta’s current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict, including risks related to the completion and timing of the phase 1/2a clinical trial or any studies relating to the treatment of glioblastoma multiforme patients who have an unmethylated MGMT gene promoter (uMGMT-GBM). Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. These and other risks and uncertainties are described more fully in the section titled “Risk Factors” in Genenta's Annual Report on Form 20-F for the year ended December 31, 2022 filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of the date of this announcement, and Genenta undertakes no duty to update such information except as required under applicable law. Genenta MediaTiziana Pollio+39 348 23 15 143tiziana.pollio@genenta.com 1 Tie2 Expressing Monocytes