Anticipated Key Milestones

On February 9, 2023 Erasca presented its key milestones presentation (Presentation, Erasca, FEB 9, 2023, View Source [SID1234626990]).

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Decibel Therapeutics to Present at the 46th Annual Association for Research in Otolaryngology (ARO) MidWinter Meeting

On February 9, 2023 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported that it will present findings from its drug discovery and development programs at the 46th Annual MidWinter Meeting of the Association for Research in Otolaryngology (ARO), being held in Orlando, Florida February 11–15, 2023 (Press release, Decibel Therapeutics, FEB 9, 2023, View Source [SID1234626989]).

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The Company will present new data on its lead investigational gene therapy product candidate, DB-OTO, as well as new data across its gene therapy pipeline. Decibel will also present additional data from the interim analysis of the Phase 1b clinical trial of DB-020 in patients receiving cisplatin chemotherapy.

Podium Presentations

Podium 1 | Precise Targeting of GJB2 Cells Results in Safe and Efficacious Gene Therapy in a Rodent Model of Hearing Loss Due to GJB2 Deficiency
Presenter: Kathryn Ellis, Ph.D.
Session Title: Gene Therapy
Date & Time: Saturday, February 11, 2:00 – 4:00 p.m. ET

Podium 26 | Development of DB-020, a Locally Administered Product for Protection Against Cisplatin-Induced Ototoxicity
Presenter: John Lee
Session Title: Cisplatin Ototoxicity
Date & Time: Tuesday, February 14, 10:30 a.m. – 12:30 p.m. ET

Poster Presentations

SU83 | Development of a Platform for Parallel Functional Evaluation of Cell Type Specific Synthetic Promoters for Gene Therapy

SU84 | Dual Vector Gene Therapy Approaches for STRC-Related Hearing Loss

MO83 | Nonclinical Pharmacology, Biodistribution, and Safety Studies Supporting the Clinical Development of DB-OTO (AAV1-Myo15-hOTOFv5) for Hearing Loss Due to Genetic Otoferlin Protein Deficiency

TU83 | Development of a Utricle Culture System for Exploring Dual AAV-Mediated Expression Kinetics of Human Otoferlin

TU232 | Advancing Bioinformatically Informed Targets to Achieve In Vivo Vestibular Regeneration

Artios Announces Initiation of Phase 2 Randomized Trial for ATR Inhibitor ART0380 plus Gemcitabine in Patients with Platinum Resistant Ovarian Cancer

On February 9, 2023 Artios Pharma Limited (Artios), a clinical-stage biotech company pioneering the development of novel small molecule therapeutics that target the DNA damage response ("DDR") process in order to treat patients with a broad range of cancers, reported the initiation of a randomized, Phase 2 expansion cohort in the ongoing Phase 1/2 trial evaluating its ataxia telangiectasia and Rad3-related ("ATR") Inhibitor, ART0380, in combination with gemcitabine for the treatment of platinum resistant ovarian cancer (Press release, Artios Pharma, FEB 9, 2023, View Source [SID1234626988]).

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The initiation of the Phase 2 trial follows the successful Phase 1 dose escalation demonstrating a favorable safety and tolerability profile, clinical activity, and preferred pharmacokinetics with ART0380 in advanced solid tumors. The recommended Phase 2 dose of ART0380 monotherapy has been defined for both intermittent and continuous daily dosing schedules together with the regimen of ART0380 in combination with gemcitabine. Clinically active RECIST confirmed responses occurred across doses and in tumors harboring DDR deficiencies as predicted through Artios’ DcoDeR platform.

Dr. Niall Martin, Chief Executive Officer at Artios, said: "This is the first of three proof of concept Phase 2 studies planned to evaluate our potentially best-in-class ATR inhibitor, ART0380, that has demonstrated a favorable safety and tolerability profile and clinical activity in the Phase 1 dose escalation. We are delighted to be collaborating with ovarian cancer experts at GEICO in Spain on this important study that will help bolster our understanding of the therapeutic potential of ART0380 and further inform our clinical development strategy. We look forward to initial Phase 2 data expected in the first half of 2025."

Antonio Gonzalez-Martin, MD, PhD, President and Founding member of the Spanish Ovarian Cancer Research Group (GEICO), Principal Investigator for the trial, said: "Ovarian cancer continues to be a difficult to treat cancer that ultimately leads to relapse. We are excited to collaborate with Artios to help bring an ATR targeted therapy with the potential to be highly effective in patients with platinum-resistant disease who are in much need of innovative treatment options. The favorable pharmacokinetic profile enabling combination with DNA damaging agents offers the potential of a new treatment option for patients with platinum resistant ovarian cancer. We at GEICO are looking forward to offering this novel treatment approach to our patients in the trial."

The ongoing global, open-label, multi-center, Phase 1/2 study is investigating the safety, tolerability, pharmacokinetic profile, and preliminary efficacy of ART0380 (GEICO-114-O trial) as a monotherapy or in combination with gemcitabine or irinotecan. The randomized, Phase 2 portion is designed to evaluate intermittent dosing of ART0380 in combination with gemcitabine in 21-day cycles in patients with high grade serous ovarian, primary peritoneal or fallopian tube carcinoma. The Phase 2 expansion in combination with gemcitabine is expected to enroll up to 60 patients and will be conducted at multiple oncology centers across the United States and Spain.

ART0380 is a potent, selective, oral inhibitor of ATR, a master regulator of DNA replication stress response. ART0380 is being developed as an oral anti-cancer agent for the treatment of cancers that harbor defects in DNA repair and in combination with agents including those that cause DNA damage. ART0380 was originally in-licensed by Artios from The University of Texas MD Anderson Cancer Center and ShangPharma Innovation in 2019. The molecule was jointly developed as part of a collaboration between ShangPharma and MD Anderson’s Therapeutics Discovery Division.

AbbVie Reports Full-Year and Fourth-Quarter 2022 Financial Results

On February 9, 2023 AbbVie (NYSE:ABBV) reported financial results for the fourth quarter and full year ended December 31, 2022 (Press release, AbbVie, FEB 9, 2023, View Source [SID1234626987]).

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"2022 was another highly productive year capping a decade of outstanding performance. Since our inception, we have built a diverse portfolio of growth products with significant leadership positions, developed a robust pipeline of innovative assets and created a culture of strong execution," said Richard A. Gonzalez, chairman and chief executive officer, AbbVie. "Looking forward, we have a solid foundation which will allow us to absorb the U.S. Humira loss of exclusivity, return to strong top-line growth in 2025 and drive top-tier financial performance over the long term."

Note: "Operational" comparisons are presented at constant currency rates that reflect comparative local currency net revenues at the prior year’s foreign exchange rates.

1Beginning in the first quarter 2022, AbbVie includes the impact of upfront and milestone payments related to collaborations, licensing agreements and other asset acquisitions in its reported non-GAAP financial measures.

Fourth-Quarter Results

Worldwide net revenues were $15.121 billion, an increase of 1.6 percent on a reported basis, or 3.8 percent on an operational basis.
Global net revenues from the immunology portfolio were $7.925 billion, an increase of 17.5 percent on a reported basis, or 19.5 percent on an operational basis.
Global Humira net revenues of $5.579 billion increased 4.6 percent on a reported, or 6.0 percent on an operational basis. U.S. Humira net revenues were $5.006 billion, an increase of 9.9 percent. Internationally, Humira net revenues were $573 million, a decrease of 26.5 percent on a reported basis, or 16.9 percent on an operational basis.
Global Skyrizi net revenues were $1.576 billion, an increase of 76.1 percent on a reported basis, or 78.9 percent on an operational basis.
Global Rinvoq net revenues were $770 million, an increase of 49.0 percent on a reported basis, or 55.4 percent on an operational basis.
Global net revenues from the hematologic oncology portfolio were $1.631 billion, a decrease of 12.9 percent on a reported basis, or 11.2 percent on an operational basis.
Global Imbruvica net revenues were $1.115 billion, a decrease of 19.5 percent, with U.S. net revenues of $841 million and international profit sharing of $274 million.
Global Venclexta net revenues were $516 million, an increase of 5.7 percent on a reported basis, or 12.2 percent on an operational basis.
Global net revenues from the neuroscience portfolio were $1.710 billion, an increase of 3.4 percent on a reported basis, or 5.1 percent on an operational basis.
Global Botox Therapeutic net revenues were $728 million, an increase of 8.5 percent on a reported basis, or 10.7 percent on an operational basis.
Global Vraylar net revenues were $565 million, an increase of 15.5 percent.
Global Ubrelvy net revenues were $197 million, an increase of 7.7 percent.
Global net revenues from the aesthetics portfolio were $1.287 billion, a decrease of 8.5 percent on a reported basis, or 4.2 percent on an operational basis.
Global Botox Cosmetic net revenues were $642 million, an increase of 2.6 percent on a reported basis, or 7.1 percent on an operational basis.
Global Juvederm net revenues were $322 million, a decrease of 25.4 percent on a reported basis, or 19.0 percent on an operational basis.
On a GAAP basis, the gross margin ratio in the fourth quarter was 72.4 percent. The adjusted gross margin ratio was 86.0 percent.
On a GAAP basis, selling, general and administrative (SG&A) expense was 22.6 percent of net revenues. The adjusted SG&A expense was 20.8 percent of net revenues.
On a GAAP basis, research and development (R&D) expense was 11.8 percent of net revenues. The adjusted R&D expense was 11.5 percent of net revenues, reflecting funding actions supporting all stages of our pipeline.
Acquired IPR&D and milestones expense was 1.6 percent of net revenues.
On a GAAP basis, the operating margin in the fourth quarter was 36.4 percent. The adjusted operating margin was 52.1 percent.
Net interest expense was $476 million.
On a GAAP basis, the tax rate in the quarter was 16.6 percent. The adjusted tax rate was 13.4 percent.
Diluted EPS in the fourth quarter was $1.38 on a GAAP basis. Adjusted diluted EPS, excluding specified items, was $3.60. These results include an unfavorable impact of $0.13 per share related to acquired IPR&D and milestones expense.

Note: "Operational" comparisons are presented at constant currency rates that reflect comparative local currency net revenues at the prior year’s foreign exchange rates.

Recent Events

AbbVie announced the European Commission (EC) approved Skyrizi (risankizumab, 600 mg intravenous induction and 360 mg subcutaneous maintenance therapy) as the first specific interleukin-23 (IL-23) inhibitor for the treatment of adults with moderately to severely active Crohn’s disease (CD) who have had inadequate response, lost response or were intolerant to conventional or biologic therapy. The approval is supported by results from three Phase 3 studies in which Skyrizi demonstrated significant improvement in clinical remission and endoscopic response, compared to placebo, as both induction and maintenance therapy. This approval marks the third indication for Skyrizi in the European Union (EU). Skyrizi is part of a collaboration between Boehringer Ingelheim and AbbVie, with AbbVie leading development and commercialization globally.

At the American College of Rheumatology’s (ACR) annual meeting, AbbVie shared 36 abstracts that underscore AbbVie’s commitment to advancing research to help more people living with rheumatic diseases. Analyses presented showcased data from the clinical trial programs evaluating Rinvoq (upadacitinib) for the treatment of rheumatoid arthritis (RA), psoriatic arthritis (PsA) and axial spondyloarthritis (AS) as well as data evaluating Skyrizi for the treatment of psoriasis (PsO) and PsA.

AbbVie announced that the U.S. Food and Drug Administration (FDA) accepted, for priority review, the Biologics License Application (BLA) for epcoritamab, an investigational subcutaneous bispecific antibody for the treatment of adult patients with relapsed/refractory (r/r) large B-cell lymphoma (LBCL). Additionally, AbbVie announced the European Medicines Agency (EMA) validated AbbVie’s Marketing Authorization Application (MAA) for epcoritamab for the treatment of adult patients with r/r diffuse LBCL. Both applications are supported by data from the EPCORE NHL-1 Phase 2 trial evaluating the safety and preliminary efficacy of subcutaneous epcoritamab in adult patients with relapsed, progressive or refractory CD20+ mature B-cell non-Hodgkin’s lymphoma (NHL). Epcoritamab is being co-developed by Genmab and AbbVie.

At the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, AbbVie presented results from nearly 65 abstracts, including 15 oral presentations, across eight types of cancer. Highlights included four oral presentations evaluating investigational epcoritamab for the treatment of r/r follicular lymphoma (FL), previously untreated FL, r/r diffuse LBCL as well as Richter’s syndrome; data from cohort three of the Phase 2 REFINE study evaluating navitoclax in combination with ruxolitinib in JAK inhibitor-naïve patients with myelofibrosis (MF); data from the Phase 2 CAPTIVATE and Phase 3 GLOW studies evaluating fixed-duration treatment in patients with chronic lymphocytic leukemia (CLL)/small lymphocytic leukemia (SLL) who received the Imbruvica (ibrutinib) + Venclexta (venetoclax) combination regimen. AbbVie also presented multiple abstracts evaluating Venclexta in approved CLL and acute myeloid leukemia (AML) indications and the investigational multiple myeloma (MM) indication. Imbruvica is jointly developed and commercialized with Janssen Biotech, Inc. Venetoclax is being developed by AbbVie and Roche and is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S.

AbbVie announced that the FDA approved Vraylar (cariprazine) as an adjunctive therapy to antidepressants for the treatment of major depressive disorder (MDD) in adults. The approval marks the fourth indication for Vraylar, which is now the first and only dopamine and serotonin partial agonist FDA-approved for the most common forms of depression – as an adjunctive treatment for MDD and the treatment of depressive episodes associated with bipolar I disorder. The approval is supported by clinical data demonstrating the efficacy and well-established tolerability of Vraylar as an adjunctive treatment for MDD with an antidepressant therapy. Vraylar is being co-developed by AbbVie and Gedeon Richter Plc.

At the European Headache Federation Congress (EHC) 2022, AbbVie shared 15 abstracts which underscore its commitment to people living with migraine. Presentations highlighted late-breaking data from the Chronic Migraine Epidemiology and Outcomes – International (CaMEO-I) study, evaluating the frequency and burden of neck pain with headache among people with and without migraine, as well as results from the Phase 3 PROGRESS trial evaluating Qulipta (atogepant) for the preventive treatment of chronic migraine (CM) in patients living in Europe.
AbbVie announced new results from its exploratory NOVA phase 2 dose-ranging study evaluating the efficacy and safety of AGN-151607, a novel investigational neurotoxin for the prevention of postoperative atrial fibrillation (POAF) in cardiac surgery patients, at the American Heart Association Scientific Sessions meeting. The study’s primary endpoint was not met for the modified intent-to-treat (mITT) population; however, the data showed relative risk reduction in specific study populations such as coronary artery bypass graft (CABG) patients and patients aged 65 years and older, as well as overall lower rates of rehospitalization within 30 days compared to placebo. Adverse Events (AEs) were numerically similar across all treatment groups.

AbbVie and HotSpot Therapeutics, Inc., a biotechnology company pioneering the discovery and development of small molecule allosteric therapies for the treatment of cancer and autoimmune diseases, announced an exclusive worldwide collaboration and option to license agreement for HotSpot’s discovery-stage interferon regulatory factor 5 (IRF5) inhibitor program for the potential treatment of autoimmune diseases.

AbbVie and Immunome, a clinical-stage biopharmaceutical company that utilizes its human memory B cell platform to discover and develop first-in-class antibody therapeutics, announced a worldwide collaboration and option agreement directed to the discovery of up to 10 novel antibody-target pairs arising from three specified tumor types using Immunome’s Discovery Engine.

AbbVie and Anima Biotech, announced a collaboration to discover and develop mRNA biology modulators for three targets across Immunology and Oncology. Anima will use its mRNA Lightning platform to discover novel mRNA biology modulators against the collaboration targets providing AbbVie exclusive rights to license and further develop and commercialize the programs.
Full-Year 2023 Outlook

AbbVie is issuing its adjusted diluted EPS guidance for the full-year 2023 of $10.70 to $11.10. The company’s 2023 adjusted diluted EPS guidance excludes any impact from acquired IPR&D and milestones that may be incurred during 2023, as both cannot be reliably forecasted.

Lirum Therapeutics Announces Closing of $7 Million Private Placement of Common Stock

On February 8, 2023 Lirum Therapeutics, Inc. (the "Company" or "Lirum"), an innovative clinical-stage biopharmaceutical company focused on the treatment of debilitating diseases, reported that it has closed a private placement, which resulted in gross proceeds to Lirum of over $7 million, before deducting offering expenses (Press release, Lirum Therapeutics, FEB 8, 2023, View Source [SID1234651601]). Net proceeds will be used by Lirum primarily to advance its clinical and preclinical studies, for working capital, and to potentially expand its product candidate pipeline.

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Lirum’s lead product candidate, LX-101, is a novel, clinical-stage, targeted therapy directed to the IGF-1 receptor (IGF-1R). The IGF-1/IGF-1R pathway is active in multiple cancers and autoimmune diseases, including thyroid eye disease (TED). LX-101 consists of a proprietary, precision-engineered IGF-1 variant coupled to a rational therapeutic payload, bringing a novel and differentiated mechanism of action versus current and past IGF-1R targeted approaches.

LX-101 has been evaluated in Phase 1 trials of patients with advanced, pretreated cancer, where it was found to be well-tolerated and demonstrated single agent activity. No dose limiting toxicity or maximum tolerated dose have been reached, and Lirum intends to continue to dose escalate and optimize the administration schedule.

Lirum plans to focus development of LX-101 toward specific adult and pediatric oncology and autoimmune indications which carry a strong scientific, medical, and business rationale. These include certain cancers harboring genetic alterations that affect the IGF-1/IGF-1R pathway and/or where IGF-1R overexpression correlates with areas of unmet medical need, as well as in certain autoimmune diseases, including TED where IGF-1R has been commercially validated.

ThinkEquity acted as sole placement agent for the offering.

The securities offered and sold by Lirum in the private placement have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or state securities laws and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission (the "SEC") or an applicable exemption from such registration requirements.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy, 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. The shares of common stock will not be registered under the Securities Act or any state securities laws, and unless so registered, may not be offered or sold in the United States except pursuant to an exemption from the registration requirements of the Securities Act and applicable state laws.