On May 7, 2024 Theragent Inc., a comprehensive CDMO focused on advancing next-generation cell- and tissue-based therapies, reported a partnership with Pluristyx, Inc., an early stage, privately held biotechnology company specializing in induced Pluripotent Stem Cell (iPSC) products (Press release, Theragent, MAY 7, 2024, https://theragent.com/theragent-and-pluristyx-enter-partnership-to-streamline-and-advance-ipsc-derived-therapy-development/ [SID1234642800]). Combined, this partnership will allow the companies to provide a seamless, end-to-end pathway to therapeutics developers from iPSC derivation to commercial production of iPSC-derived drug products.

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Under the terms of the collaboration, Pluristyx’s iPSC platform, including licenses for iPSC derivation, consent for use, and novel mechanisms such as the FailSafe functional suicide switch and iACT Stealth Cells immune evasion technology, will be utilized for early-stage programs and then synchronously transferred to Theragent for GMP processing including scale-up and clinical- to commercial-scale production at their state-of-the-art manufacturing facility in Arcadia, CA.

"Pluristyx is eager to grow our relationship with Theragent to create a synchronized platform that combines our clinical-grade iPSC lines and their CGMP manufacturing capabilities," said Dr. Benjamin Fryer, CEO and Co-founder of Pluristyx. "Together, we’re able to create a streamlined and transparent path from the bench to commercialization for the next-generation of iPSC-derived treatments."

"iPSC’s show untapped therapeutic potential, making it natural to enter a preferred partnership with an iPSC technical authority like Pluristyx," said Dr. Yu Yen, MD, PhD, and CEO of Theragent. "The tie-up leverages our stem cell expertise and our modern Pharma 4.0-driven manufacturing facility, resulting in ‘right first time, on time’ tech transfer and production – saving clients time and money while de-risking their clinical program."

On May 7, 2024 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported that it will present at the following investor conferences in May (Press release, UroGen Pharma, MAY 7, 2024, View Source [SID1234642801]):

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Citizens JMP Life Sciences Conference – May 14, 2024
Fireside Chat: May 14, 2024 at 10:00am ET
Location: New York, NY
Webcast Registration Link: Here

H.C. Wainwright BioConnect Conference – May 20, 2024
Fireside Chat: May 20, 2024 at 9:00am ET
Location: New York, NY
Webcast Registration Link: Here

On May 7, 2024 Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of Antibody Radiation Conjugates (ARCs) and other targeted radiotherapies, reported it will host a KOL call on Monday, May 20, 2024, at 8:00 AM ET to provide updates and highlight its recently announced clinical trial to study Iomab-ACT with a leading FDA approved CAR-T cell therapy at the University of Texas Southwestern (UTSW) (Press release, Actinium Pharmaceuticals, MAY 7, 2024, View Source [SID1234642807]). Iomab-ACT is an ARC that targets CD45, a marker expressed on blood cancer cells and immune cells that is intended to enable conditioning prior to cell and gene therapies such as CAR T-cell therapy and replace the non-targeted chemotherapy that is currently used for conditioning. There are six CAR-T cell therapies approved to treat patients with leukemias, lymphomas and multiple myeloma that collectively reached sales in 2023 exceeding $3.5 billion.

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Dr. Farrukh Awan specializes in the treatment of patients with leukemia and lymphoma including CAR-T therapy and bone marrow transplantation. Dr. Awan will serve as principal investigator for this study that will be led by UTSW.

To date, Iomab-ACT has been studied in a clinical trial with an investigational CD19 targeting CAR-T cell therapy developed by Memorial Sloan Kettering Cancer Center (MSKCC) under a National Institutes of Health funded grant. The trial at UTSW will be the first trial to study Iomab-ACT or any targeted radiotherapy conditioning agent with an FDA approved CAR-T therapy.

Registration for the KOL webinar can be found here or on the investor relations page of Actinium’s website here.

Iomab-ACT Phase 1 CAR-T Conditioning Results

Actinium presented results from its ongoing phase 1 trial using Iomab-ACT as conditioning prior to CD19 CAR-T therapy for patients with relapsed or refractory B-cell Acute Lymphoblastic Leukemia (B-ALL) or Diffuse Large B-cell Lymphoma (DLBCL) at the Tandem Meetings I Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR the combined annual meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for International Blood & Marrow Transplant Research (CIBMTR) in February 2024. Importantly, no patients (0/4) developed immune effector cell-associated neurotoxicity syndrome (ICANS) of any grade, a major safety measure of the study and minimal Cytokine Release Syndrome (CRS). ICANS is observed in 25% or more of pts w/ R/R B-ALL and DLBCL treated with various currently approved CAR T-cell products. Iomab-ACT demonstrated transient depletion of peripheral blood lymphocytes and monocytes. Persistence of CAR T-cells up to 8 weeks and minimal non-hematologic toxicities have been observed to date.

Targeted Radiotherapy Conditioning Opportunity

The opportunity exists for better conditioning in other areas of cellular therapy, such as CAR-T as well as gene therapies. The pipeline of CAR-T and gene therapies has rapidly expanded, with the addressable patient population expected to nearly double and reach approximately 93,000 patients in the U.S. by 2030 based on the current pipeline of cellular therapies. The CAR-T market size in terms of revenue is estimated to grow at a CAGR of approximately 11% over the next 5 plus years. Currently, there are six CAR T-cell therapies approved by the FDA that are used to treat patients with lymphomas, leukemia, and multiple myeloma, which collectively had total sales of over $3.5 billion in 2023. The addressable market for Iomab-ACT is in line with the patient population for cell and gene therapies as all patients must receive some type of conditioning prior to these treatments. We will continue to develop Iomab-ACT, our next-generation conditioning program for rapidly growing cell and gene therapies based on early promising results, ultimately with the value proposition of improving overall access and outcomes for patients who need cellular or gene therapies. A potential blockbuster revenue opportunity exists for Iomab-ACT assuming it can provide one or more clinical benefits related to lower CRS, less ICANS, longer duration of response with selective lymphodepletion or a higher overall success rate of cellular therapy due to benefits of targeted conditioning.

On May 7, 2024 Veracyte, Inc. (Nasdaq: VCYT) reported financial results for the first quarter ended March 31, 2024 (Press release, Veracyte, MAY 7, 2024, View Source [SID1234642802]).

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"We had a strong start to 2024, driven by robust growth from our market-leading Decipher Prostate and Afirma tests. We executed upon our strategies to further penetrate these markets by expanding our clinical evidence, and, for Decipher, securing enhanced status in guidelines while also achieving a key commercial reimbursement milestone," said Marc Stapley, Veracyte’s chief executive officer. "We also expanded our capabilities into minimal residual disease (MRD) testing by completing our acquisition of C2i Genomics and initiating development of our first test using their novel, whole-genome approach."

Key Business Highlights

Increased first quarter total revenue by 17% to $96.8 million, compared to the first quarter of 2023.
Grew total test volume to 33,424, an increase of 16% compared to the first quarter of 2023.
Received the highest-level rating among gene expression tests for the Decipher Prostate Genomic Classifier in updated NCCN* prostate cancer guidelines.
Expanded clinical evidence for the Decipher Prostate test with a study published in JCO Precision Oncology showing that it is prognostic for progression in prostate cancer patients undergoing Active Surveillance.
Demonstrated the power of the Veracyte Diagnostic Platform to expand evidence with the presentation of 14 abstracts at the American Urological Association’s 2024 Annual Meeting, including podium presentations for our research-use-only Decipher GRID offerings in prostate cancer and bladder cancer.
Contracted with one of the largest commercial payers in the U.S., making the Decipher Prostate test an in-network offering for its close to 30 million members.
Received Medicare coverage for our Afirma TERT promoter gene mutation test, following publication of analytical validity data for the test in the Journal of Endocrinology & Metabolism.
Completed the acquisition of C2i Genomics and initiated development of an MRD test for muscle-invasive bladder cancer.
Ended the first quarter with $209.2 million of cash and cash equivalents.
* National Comprehensive Cancer Network. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.

First Quarter 2024 Financial Results

Total revenue for the first quarter of 2024 was $96.8 million, an increase of 17% compared to $82.4 million reported in the first quarter of 2023. Testing revenue was $90.3 million, an increase of 25% compared to $72.4 million in the first quarter of 2023, driven primarily by the strong performance of our Decipher Prostate and Afirma tests. Product revenue was $3.5 million, a decrease of 9% compared to $3.9 million in the first quarter of 2023. Biopharmaceutical and other revenue was $3.0 million, a decrease of 51% compared to $6.1 million in the first quarter of 2023.

Total gross margin for the first quarter of 2024, including the amortization of acquired intangible assets, was 65%, compared to 62% in the first quarter of 2023. Non-GAAP gross margin, excluding the amortization of acquired intangible assets and other acquisition related expenses was 68%, compared to 68% in the first quarter of 2023.

Operating expenses, excluding cost of revenue, were $67.1 million. Non-GAAP operating expenses, excluding cost of revenue, amortization of acquired intangible assets, other acquisition related expenses and other restructuring costs, grew 6% to $61.6 million compared to $58.1 million in the first quarter of 2023.

Net loss for the first quarter of 2024 was $1.9 million, an improvement of 77% compared to the first quarter of 2023. Basic and diluted net loss per common share was $0.02, an improvement of $0.09 compared to the first quarter of 2023. Net cash used in operating activities in the first three months of 2024 was $9.0 million inclusive of the typical annual bonus payments and payroll tax reset in the first quarter, as well as C2i acquisition-related expenses, an increase of $6.8 million compared to the same period in 2023.

A reconciliation of GAAP to non-GAAP financial measures has been provided in the tables included in this press release. An explanation of these measures is also included below under the heading "Note Regarding Use of Non-GAAP Financial Measures."

2024 Financial Outlook

The company is raising full-year 2024 total revenue guidance to $402 million to $410 million, representing year-over-year growth of 11% to 14% and testing growth of 15% to 18%. This guidance range represents an increase compared to prior guidance of $394 million to $402 million. In addition, the company now expects cash, cash equivalents and short-term investments at the end of the year to be $236 million to $240 million compared to prior guidance of $230 million to $234 million.

Conference Call and Webcast Details

Veracyte will host a conference call and webcast today at 4:30 p.m. Eastern Time to discuss the company’s financial results and provide a general business update. The conference call will be webcast live from the company’s website and will be available via the following link: View Source The webcast should be accessed 10 minutes prior to the conference call start time. A replay of the webcast will be available for one year following the conclusion of the live broadcast and will be accessible on the company’s website at View Source

The conference call dial-in can be accessed by registering at the following link: https://register.vevent.com/register/BI5ccb6c3c55e34500b5c9256eb8e1a7f4

On May 7, 2024 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel radioimmunotherapy and, antibody-based therapeutic products for the treatment of cancer, reported financial results for the first quarter ended March 31, 2024 (Press release, Y-mAbs Therapeutics, MAY 7, 2024, View Source [SID1234642803]).

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"We continued to make meaningful progress across our commercial and clinical development initiatives during the first quarter of this year," said Mike Rossi, President and Chief Executive Officer. "On the commercial front, the first quarter of 2024 marked the highest quarter of U.S. demand for DANYELZA (naxitamab-gqgk) since its initial launch. Our recorded net product revenues in the first quarter were primarily driven by heightened demand across both new and existing U.S. accounts. DANYELZA remains a leading anti-GD2 therapy, and we continue to believe in its potential to serve patients beyond the high-risk relapse/refractory neuroblastoma market. In addition, we are highly encouraged by the clinical advancement of our Self-Assembly DisAssembly ("SADA") Pretargeted Radioimmunotherapy ("PRIT") Technology platform. With the potential to deliver optimal therapy with minimal toxicity, increase physician participation in the patient treatment journey, and leverage existing infrastructure as a potentially isotope-agnostic platform, we believe SADA PRIT has the potential to make a positive and lasting impact on patient care."

First Quarter 2024 and Recent Corporate Developments

● In April 2024, Y-mAbs’ distribution partner in Latin America, Adium, initiated the commercial launch of DANYELZA in Brazil and Mexico.
● On April 25, 2024, Y-mAbs announced several abstracts to be presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting taking place May 31 through June 4, 2024, in Chicago, IL. The Company will be available to comment at booth #35151 on the Exhibition Floor of McCormick Place.
● On March 8, 2024, Bo Kruse, Executive Vice President, Chief Financial Officer, Secretary and Treasurer of Y-mAbs informed the Company of his intention to resign from such offices effective as of the date the Company appoints his successor and such successor commences employment with the Company and to resign from employment with the Company effective July 31, 2024. A search firm has been retained to assist in the recruitment of a new Chief Financial Officer.
● On February 29, 2024, the Board of Directors of Y-mAbs increased the size of the Board from eight to nine directors and elected Mary A. Tagliaferri, M.D., to serve as a Class I director of the Company. Dr. Tagliaferri’s term as a Class I director continues until the Company’s 2025 annual meeting of stockholders.

Graphic

Financial Results

Revenues

DANYELZA total net product revenues were $19.4 million for the quarter ended March 31, 2024, which represented a decrease of 4% over $20.3 million in the comparable period of 2023. Y-mAbs’ U.S. DANYELZA net product revenues were $18.6 million for the quarter ended March 31, 2024, an increase of 11% over $16.8 million in the comparable period of 2023. The Company had international DANYELZA net product revenues of $0.8 million and $3.4 million in the quarters ended March 31, 2024 and 2023, respectively. The decrease in the quarter ended March 31, 2024, compared to the quarter ended March 31, 2023, was a result of a 2023 initial product stocking shipment of $2.5 million to Y-mAbs’ distribution partner, WEP, in connection with an early access program for DANYELZA in Europe. The Company did not have a shipment to WEP in the quarter ended March 31, 2024. Volumes for international shipments can vary from quarter to quarter, and the Company expects a higher volume to resume in future quarters in 2024.

U.S. DANYELZA net product revenues increased 3% compared to the quarter ended December 31, 2023, when excluding the $0.3 million and $1.3 million impact of a Medicaid accrual change in estimate recognized as increases in net product revenues in the quarters ended March 31, 2024 and December 31, 2023, respectively. DANYELZA total net product revenues of $19.4 million in the first quarter of 2024, represented a 17% decrease compared to the fourth quarter of 2023, primarily driven by decreased international revenues.

As of March 31, 2024, Y-mAbs has delivered DANYELZA to 63 centers across the U.S. since initial launch, with five new accounts added in the first quarter of 2024. During the quarter ended March 31, 2024, approximately 60% of the vials sold in the U.S. were sold outside of Memorial Sloan Kettering Cancer Center ("MSK"), compared to 55% in the fourth quarter ended December 31, 2023.

The Company had license revenues of $0.5 million for the quarter ended March 31, 2024. License revenue for the quarter ended March 31, 2024 arose from the January 2024 acceptance of the Brazilian Medicines Market Regulation Chamber ("CMED") price for DANYELZA. The Company did not have license revenue for the quarter ended March 31, 2023.

Operating Costs and Expenses

Cost of Goods Sold

Cost of goods sold was $2.1 million for the quarters ended March 31, 2024 and 2023, respectively.

The Company’s gross margin in the quarters ended March 31, 2024 and 2023 remained approximately constant at 89% and 90%, respectively. The Company defines gross margin as net product revenues less cost of goods sold divided by net product revenues.

Research and Development

Research and development expenses were $13.3 million for the quarter ended March 31, 2024, a decrease of 1% compared to $13.4 million for the quarter ended March 31, 2023. The $0.1 million decrease was mainly due to decreased personnel related costs of $2.6 million, inclusive of the impact of the Company’s restructuring charge recorded in the quarter ended March 31, 2023, partially offset by a $2.5 million increase in clinical trial expenses related to the Company’s investments in SADA PRIT Technology in 2024.

Selling, General, and Administration

Selling, general, and administrative expenses were $11.4 million for the quarter ended March 31, 2024, which was a $0.8 million decrease compared to $12.2 million for the quarter ended March 31, 2023. The $0.8 million decrease in selling, general, and administrative expenses was primarily attributable to a decrease in personnel related costs related to the Company’s $1.1 million restructuring charge recorded in the quarter ended March 31, 2023.

Graphic

Interest and Other Income

Interest and other income was $0.4 million for the quarter ended March 31, 2024, as compared to $1.1 million for the quarter ended March 31, 2023. The decrease of $0.7 million was primarily due to a $0.6 million decrease in foreign currency transaction income.

Net Loss

Y-mAbs reported a net loss for the quarter ended March 31, 2024, of $(6.6) million, or $(0.15) per basic and diluted share, which was relatively flat compared to net loss of $(6.4) million, or $(0.15) per basic and diluted share, for the quarter ended March 31, 2023.

Cash and Cash Equivalents

As of March 31, 2024, Y-mAbs had approximately $75.7 million in cash and cash equivalents which, together with anticipated DANYELZA product revenues, is expected to support operations as currently planned into 2027. This estimate reflects the Company’s current business plan that is supported by assumptions that may prove to be inaccurate, such that Y-mAbs could use its available capital resources sooner than it currently expects. The cash burn for the first quarter of 2024 was $2.9 million.

2024 Financial Guidance

Management reiterates its full year 2024 guidance:

● Anticipated total DANYELZA net product revenues of between $95 million and $100 million;
● Anticipated operating expenses of between $115 million and $120 million;
● Anticipated total annual cash burn of between $15 million and $20 million; and
● Cash and cash equivalents anticipated to continue to support operations as currently planned into 2027.