On May 4, 2022 Lyell Immunopharma, Inc., (Nasdaq: LYEL), a T-cell reprogramming company dedicated to the mastery of T cells to cure patients with solid tumors, reported that members of its senior management team will participate in the BofA Securities 2022 Healthcare Conference on Wednesday, May 11 at 5:20 pm PT (Press release, Lyell Immunopharma, MAY 4, 2022, View Source [SID1234613517]).

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A live webcast of the fireside chat can be accessed through the investor relations section of the Company’s website at www.lyell.com. Following the live presentation, a replay of the webcast will be available on the Company’s website for 90 days following the presentation date.

On May 4, 2022 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported its financial results for the first quarter ended March 31, 2022 and reiterated financial guidance for 2022 (Press release, Neurocrine Biosciences, MAY 4, 2022, View Source [SID1234613534]).

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"INGREZZA’s first quarter performance reflects the opportunity we have to help patients living with tardive dyskinesia (TD). We completed our salesforce expansion in April and we now have three dedicated teams across psychiatry, neurology, and long-term care who are focused on improving diagnosis and treatment rates for TD," said Kevin Gorman, Ph.D., Chief Executive Officer of Neurocrine Biosciences. "With a growing product in INGREZZA, several mid-to-late-stage clinical data read-outs this year and next, and a strong financial profile, Neurocrine Biosciences continues to establish our position as a leading neuroscience-focused company."

First Quarter INGREZZA Net Product Sales and Commercial Highlights:
•Net product sales were $303 million with total prescriptions (TRx) of approximately 57,600
•Net product sales and TRx grew 32% and 33%, respectively, vs. first quarter of 2021
•Improved levels of persistence and compliance for existing patients when compared to prior first quarters driven by strong commercial execution
•Record number of new patients
•In April 2022, salesforce expansion was completed establishing separate psychiatry, neurology, and long-term care teams
Financial Highlights:
•First quarter 2022 GAAP net income and diluted earnings per share of $14 million and $0.14, respectively, compared with $32 million and $0.33, respectively, for first quarter 2021
•First quarter 2022 non-GAAP net income and diluted earnings per share of $30 million and $0.30, respectively, compared with $49 million and $0.50, respectively, for first quarter 2021
•Differences in first quarter 2022 GAAP and non-GAAP financial results compared with first quarter 2021 driven by:
◦Increased R&D expense in support of an expanded and advancing clinical portfolio
◦Increased SG&A expense primarily due to ongoing commercial initiatives, including the TD Spotlight-branded direct-to-consumer INGREZZA advertising campaign which launched in May 2021 and deployment of the expanded salesforce in March 2022
•At March 31, 2022, the Company had cash, cash equivalents and marketable securities of $1.2 billion
A reconciliation of GAAP to non-GAAP financial results can be found in Table 3 and Table 4 at the end of this earnings release.
Recent Events:
•In March 2022, Mitsubishi Tanabe Pharma Corporation (MTPC) received Japanese Ministry of Health, Labour and Welfare approval for DYSVAL (valbenazine) for the treatment of tardive dyskinesia in Japan. Under the terms of the license agreement, the Company is entitled to receive a milestone payment of $20 million upon MTPC’s first commercial sale of DYSVAL in Japan, which is expected to occur in the second quarter of 2022.
•In April 2022, Neurocrine Biosciences presented Phase 3 data for the KINECT-HD study evaluating valbenazine for chorea associated with Huntington disease. In the study, valbenazine met the primary endpoint of significant (p<0.0001) improvement in chorea severity versus placebo as measured by the Unified Huntington’s Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score, with improvements beginning in week 2. Clinically meaningful results, demonstrated by greater response rates, were seen by clinicians (CGI-C) and patients (PGI-C) for valbenazine versus placebo. In addition, the safety profile was consistent with the known safety profile of valbenazine. The Company plans to submit a supplemental New Drug Application to the U.S. Food and Drug Administration in the second half of 2022.
1.INGREZZA sales guidance for fiscal 2022 reflects approximately 20% year-over-year growth, at the mid-point of the range, and is based on recent trends, an anticipated improving COVID-19 related environment throughout the year, and benefit from our recently completed salesforce expansion during the second half of 2022. If new COVID-19 related disruptions emerge, the Company’s ability to meet these expectations could be negatively impacted.
2.GAAP R&D guidance includes amounts for milestones that are probable of achievement or have been achieved and (ii) amounts for in-process research and development once significant collaboration and licensing arrangements have been completed. GAAP R&D Guidance includes approximately $7 million of milestone expense for the Xenon collaboration which was achieved in January 2022.
3.Non-GAAP guidance adjusted to exclude estimated non-cash stock-based compensation expense of $60 million in R&D and $110 million in SG&A.
Based upon Federal NOL’s and tax credits, the Company expects to begin making cash payments for Federal income tax beginning in the fourth quarter of 2022.Conference Call and Webcast Today at 8:00 AM Eastern Time
Neurocrine Biosciences will hold a live conference call and webcast today at 8:00 a.m. Eastern Time (5:00 a.m. Pacific Time). Participants can access the live conference call by dialing 800-895-3361 (US) or 785-424-1062 (International) using the conference ID: 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 May 4, 2022 Synthetic Biologics, Inc. (NYSE American: SYN), a diversified clinical-stage company developing therapeutics designed to treat diseases in areas of high unmet need, reported an upcoming oral presentation on novel oncolytic adenovirus VCN-11 at the 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), to be held virtually and in-person from May 16-19, 2022 in Washington, D.C (Press release, Synthetic Biologics, MAY 4, 2022, View Source [SID1234613552]).

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The oral presentation will feature Ramon Alemany, Ph.D., Head of the Immunotherapy and Virotherapy Group at the Translational Research Laboratory of the Institut Catala d’Oncologia (ICO) and Institut de Investigacio Biomedica de Bellvitage (IDIBELL).

"We are excited to present new data on VCN-11, which should further position us at the forefront of oncolytic virus development," said Steven A. Shallcross, Chief Executive Officer of Synthetic Biologics. "VCN-11 is a next-generation adenovirus that is genetically modified to express hyaluronidase and degrade the protective tumor stroma barrier. It is also engineered to incorporate a proprietary albumin binding domain in the virus’ outer shell, which is intended to improve systemic delivery by enabling the virus to coat itself in albumin and thereby evade neutralizing antibodies (NAbs). The presentation at ASGCT (Free ASGCT Whitepaper) will include preclinical results showcasing the potential of VCN-11 to balance safety, with no major toxicities observed, and effectively target tumors after intravenous re-administration, even in the presence of high level NAbs. We look forward to building upon our foundation of compelling proof-of-mechanism data and continuing to advance our oncolytic adenovirus (OV) program through clinical development."

The full abstract (#98) is accessible on the ASGCT (Free ASGCT Whitepaper) conference portal and details for the oral presentation are included below.

Title: Oncolytic Adenovirus with Hyaluronidase Activity That Evades Neutralizing Antibodies and Allows Re-Administration: VCN-11
Session Title: Cancer-Oncolytic Viruses
Presenter: Ramon Alemany, Ph.D.
Presentation Date and Time: Monday, May 16, 2022, 5:15 – 5:30 p.m. ET

On May 4, 2022 Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, reported financial results for the first quarter ended March 31, 2022 (Press release, Zymeworks, MAY 4, 2022, View Source [SID1234613570]).

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"We are incredibly excited to celebrate the milestone of the last patient enrolled for our pivotal study in second-line biliary tract cancers, as well as the continued progression of multiple zanidatamab clinical studies that we will be presenting over the course of the year starting at ASCO (Free ASCO Whitepaper) in June," said Kenneth Galbraith, Chair & CEO. "The completion of enrollment in HERIZON-BTC-01 is an important step forward in our efforts to provide patients with a new HER2-targeted therapy for BTC with the potential to improve on the current standard of care. We completed this clinical milestone ahead of our previously guided timeline set out in January, and hope to continue delivering on our corporate goals and exceeding expectations as we move forward."

First Quarter 2022 Business Highlights and Recent Developments

Completed Enrollment in HERIZON-BTC-01 Pivotal Trial in 2L Biliary Tract Cancers (BTC)
Enrollment was completed ahead of schedule for the global, pivotal trial
(HERIZON-BTC-01) evaluating zanidatamab monotherapy in patients with previously-treated advanced HER2-amplified BTC. The primary endpoint of this pivotal trial is confirmed objective response rate as determined by independent central review and we expect to finalize and present top-line data by early 2023. We expect that full details of the study will be presented at a major medical meeting in 2023.
Unveiled Next-Generation TOPO1i ADC Platform Presentation
The presentation shown in March at World ADC London highlighted the development of our next-generation TOPO1i ADC payload technology. We anticipate sharing more information on potential therapeutic candidates leveraging this TOPO1i platform at our R&D day in the fourth quarter of this year as we advance towards our goal of submitting two new Investigational New Drug applications by the end of 2024.
Completed Licensing Agreement with Atreca Utilizing Auristatin-Based ADC Platform
The technology licensing agreement with Atreca provides further validation of our auristatin-based ADC and technology platforms and showcases our ability to generate continued non-dilutive funding opportunities.
ZW49 Enrollment Continues to Advance Toward Anticipated Data Readout in 2H22
Zymeworks’ second clinical-stage asset and first biparatopic HER2-targeting antibody-drug conjugate, ZW49, has completed enrollment of 30 patients in the expansion cohorts targeting 2.5 mg/kg every three weeks. Additionally, the weekly dosing regimen recently expanded enrollment in the 1.5 mg/kg cohort and, in parallel, is now enrolling patients in the 1.75 mg/kg escalation cohort. Enrollment continues to progress well and we remain on target to submit data for presentation at a major medical meeting expected to occur in the second half of this year.
Zanidatamab Update at 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting

Zymeworks’ Asia-Pacific partner BeiGene will present data at the upcoming ASCO (Free ASCO Whitepaper) meeting in June on the first-line treatment of patients with HER2+ metastatic breast cancer using zanidatamab plus chemotherapy and on the first-line treatment of patients with HER2+ metastatic GEA using zanidatamab in combination with chemotherapy and tislelizumab. The outcomes of these treatments will be released in abstract form on May 26th, and more detailed information will be discussed at poster presentations on June 4th for first-line GEA and on June 6th for first-line breast cancer. Both presentations will provide further validation of zanidatamab’s clinical efficacy and stand to strengthen its position as a leading biparatopic HER2-targeting antibody. We plan to host a conference call to present the clinical results and clinical development strategy for zanidatamab after the ASCO (Free ASCO Whitepaper) Annual Meeting.

"We look forward to discussing these two important datasets at the ASCO (Free ASCO Whitepaper) meeting, and how the results will help shape our future development plans for zanidatamab," said Neil Josephson, M.D., Chief Medical Officer. "This will be the first presentation of clinical data with zanidatamab in the first-line treatment of advanced or metastatic HER2-positive breast cancer. The GEA presentation will contain new data from patients treated with standard of care first-line chemotherapy combined with zanidatamab, and the PD-1 inhibitor tislelizumab; a regimen that is being evaluated in the ongoing phase 3 HERIZON-GEA-01 study. We also look forward to additional opportunities available throughout 2022 to provide progress updates and present additional clinical data to support our global development program for zanidatamab."

Financial Results for the Quarter Ended March 31, 2022

Zymeworks’ revenue relates primarily to non-recurring upfront fees, expansion payments or milestone payments from collaboration and license agreements, which can vary in timing and amount from period to period, as well as payments for research and development support. Revenue for the three months ended March 31, 2022 was $1.9 million compared to $0.6 million for the same period of 2021. Revenue for 2022 related to research support and other payments from our partners including cost sharing arrangements. Revenue for the same period in 2021 was also related to research support and other payments from our partners.

Research and development expense increased by $18.2 million in the three months ended March 31, 2022 compared to the same period in 2021. Research and development expense in 2022 included non-cash stock-based compensation recovery of $3.2 million, comprised of a $2.7 million recovery from equity classified awards and a $0.5 million recovery related to the non-cash mark-to-market revaluation of certain historical liability classified awards, as well as $5.5 million from restructuring expenses. Excluding stock-based compensation expense and restructuring expenses, research and development expense increased on a Non-GAAP basis by $17.7 million in 2022 compared to 2021. The increase related primarily to higher clinical trial expenses for zanidatamab, increased drug manufacturing expenses, severance and other expenses incurred due to the Company’s restructuring program, partly offset by lower clinical trial expense for ZW49.

We expect research and development expenditures to fluctuate over time in line with the advancement, expansion and completion of the clinical development of our product candidates, as well as our ongoing preclinical research activities.

Excluding the impact of stock-based compensation and restructuring expenses, general and administrative expense increased on a Non-GAAP basis by $3.2 million in 2022 compared to 2021. This increase was primarily due to severance and other expenses incurred due to the Company’s restructuring program in 2022 as well as a non-recurring sales tax refund recognized in 2021, which offset expenses in the prior year.

Net loss for the three months ended March 31, 2022 was $72.6 million compared to $44.6 million for the same period of 2021. This was primarily due to increase in research and development expenses and general and administrative expenses referred to above.

"We continue to make progress upon our goal of improving our financial position and have successfully completed the first steps with the reduction in anticipated spending through prioritization of R&D programs and the previously announced new equity issuance closed in January," said Chris Astle, Ph.D., SVP and Chief Financial Officer. "We remain committed to further improving our cash position through non-dilutive capital and executing on the framework laid out in January, and we look forward to reporting on these initiatives in the coming months."

As of March 31, 2022, Zymeworks had $300.5 million in cash resources consisting of cash, cash equivalents and short-term investments. Based on our current operating plan, we believe that our current cash resources, and proceeds from certain existing collaboration payments we anticipate receiving, will enable us to fund our planned operations into the second half of 2023 and potentially beyond. Further, we continue to make good progress towards our previously announced goal of executing on new partnerships and collaborations in order to provide additional non-dilutive sources of funding for our operations beyond 2023.

On May 4, 2022 Novartis reported that the European Commission (EC) has approved Kymriah (tisagenlecleucel), a CAR-T cell therapy, for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy (Press release, Novartis, MAY 4, 2022, View Source [SID1234614190]). The approval follows a positive opinion in March by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and is applicable to all 27 European Union member states plus Iceland, Norway and Liechtenstein. This approval marks the third indication for Kymriah and makes it the first CAR-T cell therapy approved in the EU for these patients, which include those with r/r FL grade 1, 2 and 3A1.

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"When follicular lymphoma fails to respond to treatment or comes back, it is typically more aggressive and difficult to treat; patients often end up cycling through multiple lines of therapy with decreasing benefit," said Catherine Thieblemont, MD, PhD, Professor of Hematology in the Paris VII- University, France and Head of the Hemato-Oncology Unit of St-Louis Hospital in Paris. "The approval of Kymriah in Europe brings patients closer to a potentially definitive therapy, providing us hope for improved outcomes."

The approval is based on the global Phase II ELARA trial showing that 86% of patients who were treated with Kymriah had a response, including 69% who had a complete response (CR)1. Prolonged durable response to treatment was demonstrated with an estimated 87% of patients who achieved a CR still in response at or more than nine months after initial response1. In the trial, 94 infused patients were evaluated for efficacy with a median follow-up of approximately 21 months1.

Among 97 patients evaluable for safety, the safety profile of Kymriah was remarkable1. Cytokine release syndrome (CRS) was reported in 50% of patients after Kymriah infusion, and no Grade 3 or 4 events were reported, as defined by the Lee scale1. Neurological adverse reactions occurred in 9% of patients (1% were Grade 3 or 4) within eight weeks after Kymriah infusion1. Severe infections (Grade 3 or 4) occurred in 16% of patients1.

"With this approval, we are pleased to be able to offer this transformative therapy to more people across the globe living with this advanced blood cancer," said Marie-France Tschudin, President, Innovative Medicines International & Chief Commercial Officer, Novartis. "With long-lasting responses and a safety profile that allows for flexible administration, we are striving to rewrite cancer survival and alleviate the burden of this disease for patients and the healthcare system."

In addition to r/r FL, Kymriah is approved for the treatment of pediatric and young adult patients up to and including 25 years of age with B cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse, and adult patients with r/r diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy1.

About Novartis commitment to Oncology Cell Therapy
As part of the unique Novartis strategy to pursue four cancer treatment platforms – radioligand therapy, targeted therapy, immunotherapy and cell and gene therapy – we strive for cures through cell therapies in order to enable more patients to live cancer-free. We will continue to pioneer the science and invest in our manufacturing and supply chain process to further advance transformative innovation.

Novartis was the first pharmaceutical company to significantly invest in pioneering CAR-T research and initiate global CAR-T trials. Kymriah, the first approved CAR-T cell therapy, developed in collaboration with the Perelman School of Medicine at the University of Pennsylvania, is the foundation of the Novartis commitment to CAR-T cell therapy.

We have made strong progress in broadening our delivery of Kymriah, which is currently available for use in at least one indication in 30 countries and at more than 370 certified treatment centers, with clinical and real-world experience from administration to more than 6,900 patients. We continue to pioneer in cell therapy, leveraging our vast experience to develop next-generation CAR-T cell therapies. These therapies will utilize our new T-Charge platform being evaluated to expand across hematological malignancies and bring hope for a cure to patients with other cancer types.