On May 5, 2022 CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, reported that it will host a conference call and live audio webcast on Thursday, May 12, 2022 at 4:30 p.m. Eastern Time to discuss financial results for the first quarter ended March 31, 2022 and to provide a business update (Press release, CymaBay Therapeutics, MAY 5, 2022, View Source [SID1234613750]).

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Conference Call Details
To access the live conference call, please dial 877-407-0784 from the U.S. and Canada, or 201-689-8560 internationally, Conference ID#13728967. To access the live and subsequently archived webcast of the conference call, go to the Investors section of the company’s website at View Source

On May 5, 2022 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies (tumor infiltrating lymphocyte, TIL, and peripheral-blood lymphocyte, PBL), reported first quarter 2022 financial results and corporate updates (Press release, Iovance Biotherapeutics, MAY 5, 2022, View Source [SID1234613749]).

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Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, "Iovance had a positive start to the year across our lead program lifileucel in metastatic melanoma as well as our growing TIL pipeline. We recently reported favorable feedback from the FDA on our potency assays and assay matrix, which brings us a step closer to our planned BLA submission for lifileucel in metastatic melanoma. We continue to enroll patients in clinical studies to investigate our TIL therapies in multiple solid tumors, with plans to initiate a Phase 3 clinical trial of lifileucel in combination with pembrolizumab in frontline melanoma. In addition, the FDA has allowed an IND to proceed with a clinical trial of our PD-1 inactivated, gene-edited TIL therapy, IOV-4001. Our TIL platform, clinical data, and people are a solid foundation to establish TIL as the next class of paradigm-shifting therapy for cancer patients with significant unmet need."

First Quarter 2022 Highlights and Recent Corporate Updates

Regulatory

Iovance TIL therapy (lifileucel) in metastatic melanoma (post-anti-PD-1): Iovance received positive feedback from the U.S. Food and Drug Administration (FDA) on both its potency assay matrix and its proprietary cell co-culture assay included in the potency assay matrix. Iovance expects to request a pre-BLA meeting in July 2022 and to complete a BLA submission for lifileucel by August 2022.

IOV-4001 (PD-1 inactivated TIL therapy) Investigational New Drug (IND) Application: The FDA allowed an IND to proceed for Iovance’s first genetically modified TIL therapy, IOV-4001, for the treatment of previously treated advanced melanoma or metastatic non-small cell lung cancer (mNSCLC). IOV-4001 leverages the gene editing TALEN technology licensed from Cellectis to inactivate PD-1 expression. A clinical trial of IOV-4001 is expected to begin in 2022.

Clinical

Iovance TIL therapy (lifileucel) in frontline (anti-PD-1 naïve) metastatic melanoma:
Updated clinical data (Cohort 1A in the IOV-COM-202 trial, n=12): Updated clinical data announced in April 2022 demonstrated an overall response rate (ORR) of 67% for lifileucel in combination with pembrolizumab. Eight out of 12 patients had a confirmed objective response, including three complete responses and five partial responses.
Frontline melanoma strategy: Iovance plans to open a Phase 3 trial of lifileucel in combination with pembrolizumab in frontline metastatic melanoma in late 2022. The FDA previously granted Fast Track Designation for lifileucel in combination with pembrolizumab for the treatment of immune checkpoint inhibitor naïve metastatic melanoma.

Iovance TIL therapy (LN-145) in second-line mNSCLC:
Enrollment is ongoing at more than 30 active clinical sites in the U.S., Canada and Europe for the IOV-LUN-202 trial of LN-145 in patients with mNSCLC. A Trial in Progress (TIP) poster on IOV-LUN-202 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting featured updated eligibility criteria to broaden enrollment in reflection of the unmet need in mNSCLC.
Iovance is engaged in discussions with the FDA about the potential for IOV-LUN-202 to serve as a registrational trial for LN-145 in mNSCLC and intends to execute an updated regulatory strategy based on this dialogue and feedback.

Lifileucel in cervical cancer: Iovance is engaged in regulatory discussions about a potential BLA for lifileucel in cervical cancer and intends to execute an updated registrational strategy based on FDA dialogue and feedback.

Next-Generation Research Programs

Data presentations:
AACR 2022 Annual Meeting: A poster highlighting preclinical data for IOV-4001 demonstrated that anti-tumor activity of IOV-4001 was superior to non-edited TIL product whether alone or in combination with an anti-PD-1 antibody in a murine model of melanoma.
2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings: Research posters described TIL products manufactured from cryopreserved tumor samples shipped from Australia and a potential approach to optimize TIL memory-like phenotype and increase functionality during the manufacturing process.

Additional updates:
Several additional targets for genetic modification using the TALEN technology, including double genetic knock-out programs, are advancing in preclinical development.
Additional research and preclinical studies of next generation TIL therapies and related technologies include approaches to increase TIL potency using CD39/69 double negative TILs and gene knock-in targets as well as development of a novel interleukin-2 (IL-2) analog (IOV-3001).

Manufacturing

The Iovance Cell Therapy Center (iCTC) was awarded an Honorable Mention by the International Society for Pharmaceutical Engineering (ISPE) in the 2022 Facility of the Year Awards.
Corporate

Cash position of $516.0 million at March 31, 2022 is expected to be sufficient into 2024.

Iovance currently owns more than 40 granted or allowed U.S. and international patents for TIL compositions and methods of treatment and manufacturing in a broad range of cancers, with Gen 2 patent rights expected to provide exclusivity into 2038. More information on Iovance’s patent portfolio can be found on the Intellectual Property page on www.iovance.com.
First Quarter 2022 Financial Results

Iovance had $516.0 million in cash, cash equivalents, investments and restricted cash at March 31, 2022, compared to $602.1 million at December 31, 2021. The cash position is expected to be sufficient to fund current and planned operations into 2024.

Jean-Marc Bellemin, Chief Financial Officer of Iovance, said, "With late-stage clinical assets in our pipeline, as well as a strong balance sheet and investments focused on launch preparations, we are well positioned to execute our mission to innovate, develop and deliver TIL therapy for patients with cancer while enhancing shareholder value."

Net loss for the first quarter ended March 31, 2022, was $91.6 million, or $0.58 per share, compared to a net loss of $75.4 million, or $0.51 per share, for the first quarter ended March 31, 2021.

Research and development expenses were $68.3 million for the first quarter ended March 31, 2022, an increase of $12.4 million compared to $55.9 million for the first quarter ended March 31, 2021. The increase in research and development expenses in the first quarter 2022 over the prior year period was primarily attributable to growth of the internal research and development team, including stock-based compensation expense, as well as facility-related costs.

General and administrative expenses were $23.4 million for the first quarter ended March 31, 2022, an increase of $3.8 million compared to $19.6 million for the first quarter ended March 31, 2021. The increase in general and administrative expenses in the first quarter 2022 compared to the prior year period was primarily attributable to growth of the internal general and administrative and commercial teams, including stock-based compensation expense, facility-related costs associated with the build out of the new corporate headquarters, increases in intellectual property filing and legal expenses and enhancements to the information technology infrastructure.

For additional information, please see the Company’s Selected Condensed Consolidated Balance Sheet and Statement of Operations below.

Webcast and Conference Call

Iovance will host a conference call today at 4:30 p.m. ET to discuss first quarter 2022 financial results and corporate updates. The conference call dial-in numbers are 1 (844) 646-4465 (domestic) or 1 (615) 247-0257 (international), the conference ID is #4655146. The live webcast can be accessed in the Investors section of the company’s website at View Source The archived webcast will be available for a year in the Investors section at www.iovance.com.

On May 5, 2022 Cerus Corporation (Nasdaq: CERS) reported financial results for the first quarter ended March 31, 2022 (Press release, Cerus, MAY 5, 2022, View Source [SID1234613748]).

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Recent developments and highlights include:

First quarter 2022 total revenue of $43.0 million, reflecting a 46% increase over the prior year period. Total revenue was composed of (in thousands, except %):

First quarter 2022 net loss attributable to Cerus Corporation of $12.3 million, or $0.07 per basic and diluted share, reflecting an improvement of $5.2 million over the prior year period of $17.5 million, or $0.10 per basic and diluted share, as a result of higher sales and continued operating discipline.
As of the date of this release, the Company is increasing its 2022 annual product revenue guidance range from $157-164 million to $160 million to $165 million, representing a 22% to 26% increase over full-year 2021 reported product revenue.
The Company announced that it has signed a multi-year contract for the INTERCEPT Blood System for Platelets with the American Red Cross, which is the largest producer of INTERCEPT-treated blood components in the world.
The Company and Fresenius Kabi announced a ten-year contract extension for the production of kits for the INTERCEPT Blood System. Under the new agreement, the companies will work together to expand scale at multiple Fresenius Kabi facilities, enabling manufacturing at additional locations and unlocking further economies of scale to improve the cost profile of INTERCEPT kits.
Community Blood Center of Appleton became the Company’s third blood center production partner to obtain BLA approval for the INTERCEPT Blood System for Cryoprecipitation, which is approved for the production INTERCEPT Fibrinogen Complex.
Introduced non-GAAP Adjusted EBITDA, which for the first quarter of 2022 was negative $3.7 million, compared to negative $11.5 million during the prior year period. Cerus’ non-GAAP Adjusted EBITDA is a measure the Company has disclosed to highlight the performance of its business and its progress toward achieving cashflow breakeven. For additional information, please see definitions and the reconciliation of this non-GAAP measure accompanying this release.
Cash, cash equivalents, and short-term investments were $108.6 million at March 31, 2022.
"Cerus continued to build on its leadership position in establishing pathogen reduction as a new standard of care for blood safety during the first quarter of 2022. I am pleased to report growth from all of our major geographic regions during the period, despite the significant geopolitical turmoil and economic uncertainty that has accompanied the start to this year," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer. "As we anticipated, demand for INTERCEPT platelet kits continues to be strong in the U.S., where hospitals are choosing pathogen reduction to comply with the FDA bacterial safety guidance."

"In collaboration with our manufacturing partners, we continue to make solid progress scaling up our operations to unlock additional capacity in support of anticipated strong growth well into the future," Greenman continued. "With the visibility we have for another year of robust demand for INTERCEPT products, we have opted to raise our product revenue guidance range for 2022."

Revenue

Product revenue during the first quarter of 2022 was $37.4 million, compared to $23.4 million during the prior year period. The year-over-year growth in product revenue during the quarter came from each of the Company’s major geographic regions and was primarily driven by increased sales of INTERCEPT platelet kits in the U.S. market.

First quarter 2022 government contract revenue was $5.6 million, compared to $6.2 million during the prior year period. Reported government contract revenue is comprised of funding associated with research and development (R&D) activities related to the INTERCEPT Blood System for Red Blood Cells and sponsored efforts related to the development of next-generation pathogen reduction technology for whole blood.

Product Gross Profit & Margin

Product gross profit for the first quarter of 2022 was $19.4 million, increasing by $7.1 million over the prior year period. Product gross margin for the first quarter of 2022 was 51.7% compared to 52.5% for the first quarter of 2021, and represents an increase of approximately 60 basis points compared to the fourth quarter of 2021.

Operating Expenses

Total operating expenses for the first quarter of 2022 were $34.8 million compared to $34.9 million for the same period of the prior year. Despite having to navigate inflationary pressures, the Company was able to demonstrate financial discipline, resulting in operating leverage that is strengthening the Company’s overall financial position.

Selling, general, and administrative (SG&A) expenses for the first quarter of 2022 totaled $20.7 million, compared to $19.2 million for the first quarter of 2021. The year-over-year increase in SG&A expenses for the first quarter was tied to increased hiring and stock-based compensation.

R&D expenses for the first quarter of 2022 were $14.1 million, compared to $15.7 million for the first quarter of 2021. In the first quarter, the Company’s R&D expenses declined on a year-over-year basis as a result of various research projects reaching completion.

Net Loss Attributable to Cerus Corporation

Net loss attributable to Cerus Corporation for the first quarter of 2022 was $12.3 million, or $0.07 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $17.5 million, or $0.10 per basic and diluted share, for the first quarter of 2021.

Non-GAAP Adjusted EBITDA

Non-GAAP Adjusted EBITDA for the first quarter of 2022 was negative $3.7 million, compared to non-GAAP Adjusted EBITDA of negative $11.5 million, for the first quarter of 2021. For additional information, please see definitions and the reconciliation of this non-GAAP measure accompanying this release.

Balance Sheet & Cash Use

At March 31, 2022, the Company had cash, cash equivalents and short-term investments of $108.6 million, compared to $129.4 million at December 31, 2021.

As of March 31, 2022, the Company carried $55.0 million of notes due and a balance on its revolving line of credit of $15.0 million. The Company continues to have access to $5 million under its revolving line of credit.

For the first quarter of 2022, net cash used in operating activities totaled $21.5 million as compared to $17.5 million during the prior year period, due to changes in working capital.

Increasing 2022 Product Revenue Guidance

The Company expects full-year 2022 product revenue will be in the range of $160-165 million, representing strong growth of approximately 22%-26% compared to full-year 2021 product revenue of $130.9 million. Previously, the Company’s 2022 product revenue guidance range was $157-164 million.

Quarterly Conference Call

The Company will host a conference call at 4:30 P.M. EDT this afternoon, during which management will discuss the Company’s financial results and provide a general business overview and outlook. To listen to the live webcast, please visit the Investor Relations page of the Cerus website at View Source Alternatively, you may access the live conference call by dialing (866) 235-9006 (U.S.) or (631) 291-4549 (international).

A replay will be available on Cerus’ website, or by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and entering conference ID number 8280547. The replay will be available approximately three hours after the call through May 19, 2022.

On May 5, 2022 Massive Bio, Inc., a leader in precision medicine and artificial intelligence (AI)-enabled patient-centric clinical trial enrollment for oncology, and Amber Specialty Pharmacy, a pioneer and leader in the specialty pharmacy industry with true best-in-class programs and locations strategically placed across the United States, reported its partnership to provide advanced data-driven technology solutions for patient recruitment services to the oncology research ecosystem, and thereby offer better access for cancer patients to leading-edge clinical trials (Press release, Massive Bio, MAY 5, 2022, View Source [SID1234613747]). Through this partnership, Amber Specialty Pharmacy will seamlessly connect patients in need for clinical trials with Massive Bio’s matching and concierge enrollment services and support cancer patients and providers throughout the country; the combined capabilities include Amber’s 26 million payer lives, over 50 US Limited Distribution Networks and Massive Bio’s 60,000 unique cancer patients, 12 countries global presence – making this the first and largest partnership of its kind.

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"Amber Specialty Pharmacy is dedicated to meeting the clinical, emotional, and financial needs of cancer patients and their caregivers. Our Oncology Center of Excellence (COE) was designed to provide cancer patients with an enhanced level of care during their treatment journey." said Kristin Williams, president of Amber Specialty Pharmacy. "When faced with a cancer diagnosis, time is of the essence. It is critical that advanced cancer patients have real-time access resources readily available to them, including clinical trial options and a pathway to enrollment across lines of therapy. Massive Bio’s AI technology platform and expertise in cancer clinical trials are fully aligned with these goals, resulting in improved health outcomes and reduced costs for the industry," Williams added.

Amber Specialty Pharmacy’s patient-centered model of care and oncology COE team provides patients and their caregivers individualized care with ongoing education and support to ensure patients have what they need to feel supported throughout their treatment journey. They also utilize a proprietary artificial intelligence (AI) model to identify patients at risk for lower adherence early in treatment, which allows real time interventions, offering guidance and support to patients, leading to positive treatment outcomes. The model is also built to remove barriers to coverage and building bridges toward seamless collaboration between providers and pharmacy.

"Amber Specialty Pharmacy’s best-in-class service model has earned the trust of the nation’s largest stakeholders in healthcare, as well as many pharmaceutical commercial partners in the oncology space", said Selin Kurnaz, Massive Bio’s CEO and Co-Founder. "Massive Bio’s patient centered and AI-augmented approach to trial recruitment levering our command center and SYNERGY-AI engine, combined with Amber’s advanced analytics and capabilities using a technology platform with real-time patient insights, enables all the oncology research stakeholders to optimize patient enrollment and activation at an exponential scale nationwide. We are the only company to use an AI-integrated app and mobile technology to find your eligibility for clinical trials, as well as the best sites closest to you, and it is a natural extension to integrate and partner with Amber’s outstanding Oncology COE model and platform."

Massive Bio had recently announced the launch of its NASA-style Oncology Clinical Trial Command Center (OCTCC) to disrupt and accelerate trial enrollment, and also the launch of its 100K Cancer Clinical Trial Singularity Program aimed at matching 100,000 cancer patients in real-time to cutting-edge clinical trials using its Massive Bio’s AI-based technology, website and apps across iOS and Android platforms, expanding their presence as a global company with country-level success in 12 markets.

"With over 13,000 active cancer clinical trials active in the US at any given time, the oncology trial ecosystem and its providers look to industry leaders and digital health solutions for better ways to activate real-time data insights and succeed in this complex research environment, which is ever-focused in oral oncolytic and individualized precision medicine approaches", mentioned Arturo Loaiza-Bonilla, M.D., Co-Founder of Massive Bio. "We are excited to leverage our personalized concierge patient-centric models and Amber’s data-driven approach to generate patient insights in real time, which will augment and optimize our already successful AI-enabled trial recruitment and enrollment movement, improve patient retention, and expand access to precision cancer care and cost-saving strategies at a larger and technology-enabled scale", added Loaiza-Bonilla.

This partnership envisions to anticipate and satisfy the needs of pharma and CRO partners, research sites, clinical teams, and eligible patients in real time, using digital tools to accelerate cancer research, providing additional patient-centered services in oncology, and catalyzing the expansion of specialty pharmacy and Massive Bio’s capabilities as a new patient-centered AI-enabled standard model in oncology research.

On May 5, 2022 Dizal Pharmaceutical Co., Ltd ("Dizal", SHEX: 688192) reported the publication of the pre-clinical and early clinical data of sunvozertinib (DZD9008) for the treatment of non-small cell lung cancer with EGFR exon 20 insertion mutations in Cancer Discovery (Press release, Dizal Pharma, MAY 5, 2022, View Source [SID1234613735]).

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Due to the unique structural features of EGFR exon20ins proteins, most approved EGFR TKIs are not active against them. Sunvozertinib (DZD9008) was designed with the goal to address the limitations of existing therapies. It is an irreversible inhibitor targeting EGFR exon20ins as well as EGFR sensitizing, T790M and uncommon mutations while maintaining selectivity against wild-type EGFR. Sunvozertinib shows potent antitumor activity against EGFR exon20ins mutations at the enzymatic and cellular levels, and in patient-derived xenograft and transgenic animal models. These findings support the ongoing clinical development of sunvozertinib for the treatment of EGFR exon20ins NSCLC. Commenting on the findings, Prof. Pasi Jänne of Dana-Farber Cancer Institute and Harvard Medical School, a lead investigator of the study and the corresponding author of the paper, said "sunvozertinib was well tolerated in the study. Antitumor efficacy was observed at the doses of 100 mg and above in patients with EGFR exon20ins NSCLC across different subtypes, with prior amivantamab treatment as well as with baseline brain metastasis. Its best ORR was 48.4%." Based on the data, sunvozertinib has been granted Breakthrough Therapy Designation by US FDA and China CDE.

"This is yet another milestone achievement for the team. Like Breakthrough Therapy Designation from US and China, it reflects the quality of Dizal’s science. The data published further demonstrated sunvozertinib’s enormous potential for bringing meaningful benefits to lung cancer patients." said Dr. Xiaolin Zhang, CEO of Dizal. "At Dizal, we strive to bring differentiated therapies to treat devastating diseases. We have now established an internationally competitive portfolio with two leading assets in the global pivotal studies."

Paper Details:

Journal: Cancer Discovery
Title: Sunvozertinib, a selective EGFR inhibitor for previously treated non-small cell lung cancer with EGFR exon 20 insertion mutations
Corresponding Author: Prof. Pasi A. Jänne, Dana-Farber Cancer Institute of Harvard Medical School.
Joint Primary Authors: Prof. Mengzhao Wang, Chinese Academy of Medical Sciences & Peking Union Medical College and Prof. James Chih-Hsin Yang, Graduate Institute of Oncology of National Taiwan University
About Cancer Discovery

Cancer Discovery, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), publishes high-impact, peer-reviewed articles describing major advances in research and clinical trials [1]. As the premier cancer information resource, the Journal presents articles from the world-class universities and top-notch oncology research institutions.

About EGFR Exon20ins NSCLC

Lung cancer is the leading cause of cancer death in the world. It is classified broadly as non-small cell lung cancer (NSCLC), accounting for 85% lung cancer cases, and small cell lung cancer (SCLC). EGFR mutation is common in NSCLC. About 4–12% of all EGFR mutations are insertions at exon 20 (EGFR exon20ins) [2]. Patients with EGFR exon20ins generally don’t respond to the currently available EGFR TKIs.