On April 13, 2022 Autigen, a biotechnology portfolio company of the pharmaceutical accelerator, Ascend BioVentures, developing novel treatments for hearing loss, reported that it has signed a research collaboration and license agreement with Boehringer Ingelheim to discover, develop, and commercialize transformative therapies for patients with SNHL (Press release, Boehringer Ingelheim, APR 13, 2022, View Source [SID1234612144]).

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About 430 million people suffer from moderate to complete hearing loss worldwide, a number expected to rise to about 700 million by 2050. Hearing loss can significantly impact multiple aspects of life, including the loss of the ability to follow spoken conversations. Hearing loss contributes to increased risk of dementia and cognitive decline. Deafness in early life, if left unaddressed, is associated with poor development of spoken language and higher unemployment rates in later life. In addition, hearing loss represents a significant psychological burden such as loneliness, isolation, depression, and anxiety.

SNHL, which can eventually lead to deafness, accounts for about 90% of reported hearing loss cases and is related to a degeneration of sensory hair cells (HC) in the inner ear.

"We are excited to enter this collaboration with Boehringer-Ingelheim, an innovation-led company with a strong commitment to developing breakthrough regenerative and hearing loss therapies." said Elaine Hamm, Ph.D., President, Autigen and CEO of Ascend BioVentures. "This collaboration advances our leadership position in novel therapies for sensorineural hearing loss (SHNL), an approach that Autigen and our collaborators at the Hough Ear Institute, have pioneered. It marks the first major licensing partnership out of our pharmaceutical accelerator, Ascend BioVentures and will enable Autigen to advance its novel therapy approach to restore SHNL for patients, who currently have no approved pharmacological or biological treatment options."

Under the terms of the agreement, Autigen will receive an undisclosed upfront payment and research funding support. The company is also eligible to receive future success-based milestone payments of a total potential value of more than $100M if all milestones are achieved over the course of the partnership. Autigen is also eligible for royalties on products derived from the partnership. After a joint research collaboration phase, Boehringer Ingelheim will be responsible for further preclinical and clinical development, and commercialization.

On April 13, 2022 Microba Life Sciences (ASX:MAP), a precision microbiome science company, reported that trading on the Australian Securities Exchange (ASX) on April 4th following the completion of an initial public offering (IPO) (Press release, Biosortia Pharmaceuticals, APR 13, 2022, View Source [SID1234612143]).

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The IPO raised $30.0 million and was supported by institutional, professional and retail investors. Bell Potter Securities Limited and Canaccord Genuity (Australia) Limited were Joint Lead Managers and Underwriters to the IPO.

NYSE listed synthetic biology company Ginkgo Bioworks (NYSE: DNA) invested USD $3.5m into Microba IPO, becoming a ~4% shareholder of the Company with which Microba also signed a therapeutic development agreement to identify single-strain, live biotherapeutics product (LBP) candidates against autoimmune diseases. The collaboration aims to build on Microba’s precision approach to LBP development with an in-depth evaluation of the company’s strains using Ginkgo’s high throughput, automated screening capabilities.

The Company’s strategy is to partner or license therapeutic assets with large pharmaceutical companies early in clinical development in return for upfront, milestone and royalty payments. The Offer will enable Microba to accelerate the growth of its Services and Therapeutics business and further develop its platform technology:

• Microbiome Services – Microba is an established leader in microbiome testing with over 20,000 test reports sold to date. New major distribution partnerships including SYNLAB (EU), Genova Diagnostics (US), and G42 (Middle East) accelerate growth with multiple products scheduled for launch.

• Proprietary Databank – a large, unique, proprietary microbiome Databank comprising of over 1.2 million microbial genomes. Microba’s Databank has enabled the Company to identify novel therapeutic leads not identified by others.

• Microbiome Therapeutics – Microba leverages its growing Databank through a repeatable Therapeutics Platform to develop novel microbiome therapeutics. Microba has established multiple therapeutic programs, including for Inflammatory Bowel Disease with a Phase 1 clinical trial planned to commence in late 2022.

"We believe the human microbiome currently represents a missing piece in the treatment of major chronic diseases, and as a result a number of microbiome-based therapeutics are progressing through clinical development globally"

said Luke Reid, CEO at Microba.

"Ginkgo’s high throughput screening automation combined with our novel data-driven approach to therapeutic discovery from the microbiome can potentially accelerate development of breakthrough new drugs for autoimmune diseases."

Through this partnership, Ginkgo will provide high-throughput screening for Microba’s proprietary library of human microbiome-isolated strains, with the goal of improving treatment for autoimmune diseases such as lupus, psoriatic arthritis and certain autoimmune liver diseases. Microba plans to leverage Ginkgo’s high-throughput anaerobic culturing, multi-omics data collection and analysis, functional bioassay screening, and media and fermentation optimization capabilities to generate data sets that may help characterize potential therapeutic and non-therapeutic uses of the strains. The initial partnership combining Microba’s biobank and Ginkgo’s anaerobic development capabilities is expected to run approximately two years.

"Microba is doing truly innovative work during an exciting time for the field of microbiome science,"

said Jennifer Wipf, Senior Vice President, Commercial Cell Engineering of Ginkgo.

"We’re happy to welcome new partners like Microba as we apply our platform to more applications in the living therapeutics and microbiome space."

On April 13, 2022 Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported its Annual Report on Form 10-K and reported final audited financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Supernus, APR 13, 2022, View Source;2022.htm [SID1234612142]).

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On February 28, 2022, the Company announced preliminary unaudited results for the fourth quarter and full year ended December 31, 2021. This press release updates and supersedes the press release issued on February 28, 2022 as a result of the filing of the Company’s audited year-end financial statements.

In completing its year-end financial statements, one change relating to non-recurring acquisition-related costs associated with the acquisition of Adamas Pharmaceuticals, Inc. (Adamas) was made to the preliminary unaudited results. This change and the resulting final audited results are in the table below:

As compared to the preliminary unaudited results, combined R&D and SG&A expenses increased by approximately $15.6 million and final operating earnings was reduced by that amount, in each case, for the three and twelve months ended December 31, 2021. The final audited results do not include any other changes from the 2021 preliminary unaudited information included in the February 28, 2022 press release. Detailed final audited results for the period ended December 31, 2021, can be found at the end of this release.

Non-GAAP operating earnings adjust for non-cash items including share-based compensation expense, depreciation and amortization, and accretion of contingent consideration, and for non-recurring costs. Acquisition-related costs reflect non-recurring acquisition-related costs related to the Adamas acquisition. Other R&D reflects a non-cash expense related to the equity investment in Navitor due to the accounting impact of the March 2021 Navitor corporate restructuring. Included in the amortization of intangible assets is amortization of acquired intangible assets from the Adamas acquisition in November 2021.

Full Year 2022 Financial Guidance — GAAP to Non-GAAP Adjustments

An itemized reconciliation between projected operating earnings on a GAAP basis and projected operating earnings on a non-GAAP basis is as follows:

Non-GAAP Financial Information

This press release contains a financial measure, non-GAAP operating earnings, which does not comply with United States generally accepted accounting principles (GAAP). The non-GAAP financial measure should be considered in addition to, not as a substitute for or in isolation from, or superior to measures prepared in accordance with GAAP. Non-GAAP operating earnings adjust for non-cash share-based compensation expense, depreciation and amortization, and accretion of contingent consideration, and for factors that are unusual, non-recurring or unpredictable, and exclude those costs, expenses, and other specified items presented in the reconciliation tables in this press release. We believe the use of non-GAAP operating earnings measure is useful supplemental information to investors regarding the Company’s results of operations and assist management, analysts, and investors in evaluating the performance of the business. There are limitations associated with the use of non-GAAP financial measure. Including such measure may not be entirely comparable to similarly titled measures used by other companies, may not reflect all items of income and expense, as applicable, that affect our operations, potential differences among calculation methodologies, may differ from the non-GAAP information used by other companies, including peer companies, and therefore comparability may be limited. We mitigate these limitations by reconciling the non-GAAP financial measures to the most comparable GAAP financial measures. Investors are encouraged to review the reconciliation. The Company’s 2022 financial guidance is also being provided on both a reported and a non-GAAP basis.

On April 13, 2022 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it has scheduled its first quarter 2022 financial results conference call and webcast for 5:00 a.m. Pacific Time (8:00 a.m. Eastern Time) on May 4, 2022 (Press release, Neurocrine Biosciences, APR 13, 2022, View Source [SID1234612141]).

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The schedule for the press release and conference call / webcast is as follows:

NBIX

The webcast can also be accessed on Neurocrine Biosciences’ website under Investors at www.neurocrine.com. A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.

On April 13, 2022 Astellas Pharma Inc. (TSE:4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and Seagen Inc. (Nasdaq:SGEN) reported that the European Commission (EC) has approved PADCEV (enfortumab vedotin) as monotherapy for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a platinum-containing chemotherapy and a PD-1/L1 inhibitor (Press release, Seagen, APR 13, 2022, View Source [SID1234612139]). The EC approval is supported by data from the global phase 3 EV-301 trial that demonstrated an overall survival (OS) benefit compared with chemotherapy.

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"The approval of enfortumab vedotin in the European Union is a significant milestone for people living with advanced urothelial cancer who have had limited treatment options and poor survival rates," said Ahsan Arozullah, M.D., M.P.H., Vice President, Medical Sciences-Oncology, Astellas. "We look forward to working with health authorities to ensure people living with advanced urothelial cancer can access this new treatment option as soon as possible."

The EV-301 trial compared enfortumab vedotin to chemotherapy in adult patients (n=608) with locally advanced or metastatic urothelial cancer who were previously treated with platinum-based chemotherapy and a PD-1/L1 inhibitor. At the time of the pre-specified interim analysis, patients who received enfortumab vedotin (n=301) in the trial lived a median of 3.9 months longer than those who received chemotherapy (n=307). Median OS was 12.9 vs. 9 months, respectively [Hazard Ratio=0.70 (95% Confidence Interval [CI]: 0.56, 0.89), p=0.001]. Across clinical trials, the most common adverse reactions with enfortumab vedotin were alopecia, fatigue, decreased appetite, peripheral sensory neuropathy, diarrhea, nausea, pruritus, dysgeusia, anemia, weight decreased, rash maculo-papular, dry skin, vomiting, aspartate aminotransferase increased, hyperglycemia, dry eye, alanine aminotransferase increased and rash.

"The EV-301 study is the first randomized trial to show improved overall survival in patients with advanced urothelial cancer who received a platinum-containing chemotherapy and an immunotherapy," said Professor Ignacio Durán, M.D., Ph.D., Hospital Universitario Marqués de Valdecilla, Spain. "This approval of enfortumab vedotin from the European Commission is an important moment for these patients and their physicians."

Results from the EV-301 trial are intended to support global registrations for enfortumab vedotin. The EC marketing authorization for enfortumab vedotin is applicable in the European Union (EU) Member States, as well as Iceland, Norway and Liechtenstein.1

Urothelial cancer is the most common type of bladder cancer.2 In Europe, an estimated 204,000 people were diagnosed with urothelial cancer in 2020, and more than 67,000 died as a result of the disease.3 Enfortumab vedotin is the first antibody-drug conjugate authorized in the EU for people living with urothelial cancer.

About Urothelial Cancer

Urothelial cancer is the most common type of bladder cancer (90 percent of cases) and can also be found in the renal pelvis (where urine collects inside the kidney), ureter (tube that connects the kidneys to the bladder) and urethra.2 Globally, approximately 573,000 new cases of bladder cancer and 212,000 deaths are reported annually.3

About the EV-301 Trial

The EV-301 trial (NCT03474107) was a global, multicenter, open-label, randomized phase 3 trial designed to evaluate enfortumab vedotin versus physician’s choice of chemotherapy (docetaxel, paclitaxel or vinflunine) in 608 patients with locally advanced or metastatic urothelial cancer who were previously treated with a PD-1/L1 inhibitor and platinum-based therapies.4 The primary endpoint was overall survival and secondary endpoints included progression-free survival, overall response rate, duration of response and disease control rate, as well as assessment of safety/tolerability and quality-of-life parameters. Results were published in the New England Journal of Medicine.

About PADCEV (enfortumab vedotin)

PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.5,6 Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).5 PADCEV is co-developed by Astellas and Seagen.

Important Safety Information

The full European Summary of Product Characteristics (SmPC) for PADCEV will be available from the European Medicines Agency website at www.ema.europa.eu.