On May 6, 2025 Formosa Pharmaceuticals, Inc. ("Formosa"; 6838.TW ticker-symbol on the Taiwan Stock Exchange) and Almac Discovery reported a global licensing agreement for development and commercialisation of ALM-401, a first-in-class engineered bispecific Antibody-Drug Conjugate (ADC), addressing the high unmet needs of cancer patients worldwide suffering with intractable and aggressive solid tumours (Press release, Almac, MAY 6, 2025, View Source [SID1234652564]).

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The agreement facilitates the next phases of CMC and drug development by Formosa, including IND submission and early clinical proof-of-concept in international clinical trials.

ALM-401 has been the culmination of a multi-year R&D programme at Almac Discovery deploying their proprietary OmniaScape informatics platform, protein engineering and medicinal chemistry capabilities.

The design of ALM-401 has built upon the recent clinical successes within the ADC field, including selection of a linker-payload matched to potential clinical cancer indications, and the molecule also benefits from being approximately half the size of conventional ADCs, thereby facilitating enhanced solid tumour penetration. The main features of ALM-401 include:

Innovative bi-specific target-pairing based on co-expression and functional analysis of specific, aggressive solid tumours
Sustained high-efficacy in vivo in PDX models; as presented at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) (AACR; Chicago) annual meeting
Half-the-size of conventional ADCs for enhanced solid tumour penetration and optimised manufacturing.
Formosa Pharmaceuticals, Inc. (6838.TW) is a clinical stage biotechnology company with primary focus in the areas of ophthalmology and oncology, with particular CMC and manufacturing expertise for specialist ADC therapeutics. "Formosa Pharma is pleased to have this opportunity to bring ALM-401 into our development pipeline. This novel, next-generation ADC complements our corporate strategy and resources and promises to deliver a differentiated therapy to cancer patients worldwide. We look forward to collaborating closely with Almac Discovery in advancing this exciting program through clinical trials, commercialization, and beyond." said Dr. Erick Co, President and CEO of Formosa Pharmaceuticals.

"We are pleased to have met Formosa’s exacting selection requirements for Next Generation ADC candidates," said Dr Stephen Barr, President and Managing Director of Almac Discovery. "This agreement allows the seamless progression of the molecule into clinical evaluation, driven by Formosa."

"In addition to an excellent preclinical efficacy profile, high-quality, robust and scalable CMC and manufacturing is a key competitive advantage for ADCs in the fast-moving race for effective cancer therapeutics" commented Dr Graham Cotton, Vice-President of Protein Therapeutics, Almac Discovery.

On May 6, 2025 Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in molecular diagnostic testing and precision medicine, reported financial results for its first quarter ended March 31, 2025 and updated its previously issued financial guidance on business performance for the full-year 2025 (Press release, Myriad Genetics, MAY 6, 2025, View Source [SID1234652583]).

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"We had a challenging first quarter of 2025 with strength in our prenatal and oncology MyRisk tests offset by softness in GeneSight and unaffected hereditary cancer tests. While we are actively working on initiatives to re-accelerate testing volumes, this will take time; therefore we are lowering our 2025 financial guidance. We are taking immediate steps to reduce overall expenditures while prioritizing investment in new product development and programs intended to drive revenue growth," said Sam Raha, President and CEO, of Myriad Genetics.

"As a new leadership team we are focused on unlocking Myriad Genetics’ potential by implementing a compelling strategy, strengthening our organizational capabilities, and improving execution."

Financial and Operational Highlights
•Test volumes of 385,000 in the first quarter of 2025 increased 1% year-over-year.
•The following table summarizes year-over-year testing volume changes in the company’s core product categories:
Three Months Ended March 31,
(in thousands) 2025 2024 % Change
Product volumes:
Hereditary cancer
73 71 3 %
Tumor profiling(1)
12 14 (14) %
Prenatal 173 172 1 %
Pharmacogenomics
127 124 2 %
Total 385 381 1 %
(1) Tumor Profiling decreased for the three months ended March 31, 2025 compared to the same period in the prior year due primarily to a decrease in testing volume for EndoPredict due to the sale of the company’s international EndoPredict business in August 2024.

•The following table summarizes year-over-year revenue changes in the company’s core product categories:
Three Months Ended March 31,
(in millions)
2025 2024 % Change
Product revenues:
Hereditary cancer
$ 86.3 $ 88.1 (2) %
Tumor profiling(1)
29.3 30.9 (5) %
Prenatal 49.3 44.3 11 %
Pharmacogenomics
31.0 38.9 (20) %
Total $ 195.9 $ 202.2 (3) %
(1) Tumor Profiling decreased for the three months ended March 31, 2025 compared to the same period in the prior year due primarily to a decrease in testing volume for EndoPredict due to the sale of the company’s international EndoPredict business in August 2024.

•Operating expenses in the first quarter of 2025 were $163.2 million, decreasing $2.3 million year-over-year. Adjusted operating expenses in the first quarter of 2025 increased $1.5 million year-over-year to $140.6 million, reflecting the company’s commitment to disciplined cost management while maintaining investments in key strategic areas, such as research and development.
•Operating loss in the first quarter of 2025 was $29.0 million, an increase of $1.1 million year-over-year; adjusted operating loss in the first quarter of 2025 was $5.5 million.

Cash Flow and Liquidity
First quarter 2025 cash flow used in operations was $16.3 million; adjusted cash flow used in operations in the first quarter of 2025 was $10.4 million, a decrease of $1.1 million year-over-year. Capital expenditures and capitalization of internal use software costs were $8.3 million in the first quarter 2025.

As of the end of the first quarter 2025, the company had cash and cash equivalents of $92 million and the ability to access an incremental $42 million of availability under its asset-based credit facility (ABL Facility). The continued availability and amount thereof under the ABL Facility is subject to maintaining compliance with the fixed charge coverage ratio and maintaining $20 million of cash in a controlled account with the administrator of the ABL Facility.

Business Performance and Highlights
Oncology
The Oncology business delivered revenue of $77.7 million in the first quarter of 2025.
•First quarter 2025 hereditary cancer testing volume and revenue in Oncology grew 5% and 0% year-over-year, respectively, as MyRisk with RiskScore testing volume in the affected population grew 11% year-over-year. A year-over-year decrease in average revenue per hereditary cancer test in the first quarter 2025 was largely due to a difference in change in estimated revenue related to prior periods.
•First quarter 2025 Prolaris test revenue declined 2% year-over-year as Myriad Genetics continues to educate clinicians on how it believes the Prostate Cancer Guidelines from the National Comprehensive Cancer Network (NCCN) underscore the critical role of Myriad Genetics’ portfolio of offerings across the patient’s prostate cancer journey.
•New clinical data highlighting the performance of the Precise MRD test was presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) conference in April 2025 and additional clinical data will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in May 2025.
•Myriad continues to make progress and intends to commercially launch its first AI-driven prostate cancer test, in partnership with PATHOMIQ, by end of 2025.

Women’s Health
The Women’s Health business delivered revenue of $87.2 million in the first quarter of 2025.
•Prenatal testing revenue in the first quarter of 2025 grew 11% year-over-year reflecting growth across both carrier screen and non-invasive prenatal testing. First quarter saw positive early adoption of the mid-fourth quarter 2024 launch of the Prequel Early Gestational Age test.
•First quarter 2025 hereditary cancer testing revenue for the unaffected population decreased 4% year-over-year on stable volume as the company continues to develop and deploy its electronic medical records (EMR) efforts and further expand its breast cancer risk assessment programs across its current and new provider base.

Pharmacogenomics
GeneSight test revenue was $31.0 million in the first quarter of 2025. As anticipated, first quarter this test revenue was impacted by UnitedHealthcare’s decision to discontinue coverage of multi-gene panel pharmacogenetic testing, effective in the first quarter of 2025, as well as Myriad Genetics’ actions to streamline the Pharmacogenomics organization.

Financial Guidance
Myriad Genetics does not provide forward-looking guidance in accordance with accounting principles generally accepted in the United States (GAAP) for the measures on which it provides forward-looking non-GAAP guidance as the company is unable to provide a quantitative reconciliation of forward-looking non-GAAP measures to the most directly comparable forward-looking GAAP measure, without unreasonable effort, because of the inherent difficulty in accurately forecasting the occurrence and financial impact of the various adjusting items necessary for such reconciliations that have not yet occurred, are dependent on various factors, are out of the company’s control, or cannot be reasonably predicted. Such adjustments include, but are not limited to, real estate optimization and transformation initiatives, certain litigation charges and loss contingencies, costs related to acquisitions/divestitures and the related amortization, impairment and related charges, depreciation, equity compensation, tax benefits, and other adjustments. For example, stock-based compensation may fluctuate based on the timing of employee stock transactions and unpredictable fluctuations in the company’s stock price. Any associated estimate of these items and its impact on GAAP performance could vary materially.

Below is a table updating Myriad Genetics’ full-year 2025 financial guidance*:
(in millions, except per share amounts) INITIAL 2025 Guidance
CURRENT 2025 Guidance
FY 2025 Comments
Revenue $840 – $860
$807 – $823
Lowered 2025 revenue range mid-point by $35 million reflecting an updated outlook for our pharmacogenomics business and hereditary cancer testing in our Women’s Health business.
Gross Margin %
69.5% – 70.5% 68.5% – 69.5% Gross margins expected to fluctuate in any quarter given product mix and pricing trends.
Adjusted Operating Expenses
$575 – $595
$555 – $565
Change reflects moderating planned expenditures for remainder of 2025.
Adjusted EBITDA** $25 – $35
$19 – $27
Adjusted EPS*** $0.07 – $0.11
$(0.02) – $0.02
*
Assumes currency rates as of May 6, 2025.
** Adjusted EBITDA is defined as Net Income (loss) plus income tax expense (benefit), total other income (expense), non-cash operating expenses, such as amortization of intangible assets, depreciation, impairment of long-lived assets, and share-based compensation expense, and one-time expenses such as expenses from real estate optimization initiatives, transformation initiatives, legal settlements, and divestitures and acquisitions.
***
Full-year 2025 adjusted EPS is based on a 94 million share count.

These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release.

Conference Call and Webcast
A conference call will be held today, Tuesday, May 6, 2025, at 4:30 p.m. ET to discuss Myriad Genetics’ financial results and business developments for the first quarter 2025. A live webcast of the conference call can be accessed on Myriad Genetics’ Investor Relations website at investor.myriad.com. To participate in the live conference call via telephone, please register at View Source Upon registering, a dial-in number and unique PIN will be provided to join the conference call. Following the conference call, an archived webcast of the call will be available at investor.myriad.com.

On May 6, 2025 Zetagen Therapeutics, Inc., a private, clinical stage, biopharmaceutical company focused on developing breakthrough therapies, via local administration, for metastatic and primary breast cancers, reported they have successfully completed enrollment in their phase 2a study, which will evaluate ZetaMet (Zeta-BC-003) in the treatment of spinal metastatic lytic breast cancer lesions (ClinicalTrials.gov #NCT05280067) (Press release, Zetagen Therapeutics, MAY 6, 2025, View Source [SID1234652599]).

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"We are excited to reach another critical milestone in the development of ZetaMet (Zeta-BC-003), " said Joe C. Loy, Zetagen’s Chief Executive Officer. "We launched this study with stage IV breast cancer patients suffering from spinal metastases, because we recognize their severe pain and how debilitating it is, and that current treatments remain largely palliative. Our objective with ZetaMet is to eliminate cancer cells responsible for bone destruction, alleviate pain, stimulate the regeneration of bone lost to lytic lesions, enhance overall quality of life, while improving survival rates."

The 26-week study, conducted at the University of British Columbia, (UBC) Vancouver, BC, Canada, will evaluate the safety and efficacy of ZetaMet (Zeta-BC-003) in treating vertebral bone defects created by lytic metastatic breast cancer. The study will measure the reduction of skeletal related events (SRE), pain, change in vertebral body defect size, and postoperative prescribed opioid use. The Company anticipates reporting top-line results early fourth quarter of 2025.

Zetagen expects to submit an Investigation New Drug (IND) application to the U.S. Food and Drug Administration ("FDA"), leveraging its FDA’s Breakthrough designations.

About ZetaMet (Zeta-BC-003)
ZetaMet’s (Zeta-BC-003) small molecule mechanism of action (MOA) via a novel molecular pathway initiates a circuit which results in tumor cell death.

ZetaMet (Zeta-BC-003) is the first-of-its-kind, synthetic, small-molecule delivered via a proprietary controlled-release carrier intended to resolve metastatic breast cancer bone lesions, administered locoregionally, to inhibit pain while regenerating new bone, with the potential to increase survival rates.

The US Food & Drug Administration (FDA) has recognized Zetagen’s discoveries with multiple Breakthrough Designations including ZetaMet (Zeta-BC-003).

Zetagen with FDA and Health Canada (HC) approval via their Expanded Access (Compassionate Use) programs has treated several patients with ZetaMet (Zeta-BC-003) with results published in multiple peer-reviewed journals.

Peer-reviewed 2-year follow up clinical data published in 2023 on ZetaMet (Zeta-BC-003) demonstrated resolution of 7 lytic lesions (radiated and non-radiated), reduction in pain, significant reduction in opioid pain medication (4-fold), prevention of vertebral fracture, and increased survival rate in a patient living with Stage 4 breast cancer.[i] To view this publication via open access, go to: View Source

On May 6, 2025 Mission Bio, a pioneer in single-cell multi-omics solutions, reported it has partnered with Integrated DNA Technologies (IDT), a global leader in genomics solutions, to introduce an advanced workflow for precise on- and off-target confirmation in gene editing applications (Press release, Mission Bio, MAY 6, 2025, View Source [SID1234652616]). This collaboration integrates IDT’s award-winning rhAmpSeq technology with Mission Bio’s Tapestri Platform to provide a comprehensive, high-resolution approach for assessing genome editing outcomes at the single-cell level.

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Genome editing is crucial for the development of the next wave of cell and gene therapies, enabling precise and multiple genetic modifications. However, ensuring the specificity and efficiency of these edits remains a critical challenge, particularly in cell and gene therapy (CGT) applications. Traditional bulk sequencing methods provide valuable insights into genome editing efficiency, but lack the resolution to capture single-cell heterogeneity, zygosity, and co-occurrence of edits within individual cells.

To address these challenges, Mission Bio and IDT have developed an integrated workflow combining IDT’s rhAmpSeq amplicon-based targeted resequencing chemistry with Mission Bio’s droplet-based single-cell DNA sequencing technology. This novel solution builds on published approaches that researchers have previously used to leverage the platforms. The new, integrated approach enhances the efficiency for obtaining both on-target efficacy assessment and off-target detection in a single-cell context, offering researchers an unprecedented level of accuracy and confidence in genome editing analysis.

In a proof-of-concept study, to be presented at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, the integrated workflow was interrogated using well-characterized cell lines and selected for their extensive orthogonal bulk sequencing data. By targeting loci with known indel variations ranging from single-base pair changes to large insertions and deletions (>2,000 bp), the study demonstrated the robustness of rhAmpSeq chemistry in single-cell applications. The resulting targeted panel proved compatible with both bulk and single-cell assays, ensuring seamless adoption across various research needs.

"Our single-cell multi-omics has been used in combination with bulk sequencing to add specificity that otherwise would not be possible," said Brian Kim, CEO of Mission Bio. "By integrating our capabilities with IDT’s trusted approach to bulk sequencing, we’re adding together multiple layers of genetic insights in a single drop."

The Tapestri Platform’s automated single-cell genome editing analytics pipeline provided a quantitative assessment of editing efficiency, off-target editing levels and zygosity, offering a critical layer of data resolution beyond bulk sequencing. By integrating rhAmpSeq’s advanced primer design with Mission Bio’s single-cell DNA resequencing technology, the new workflow establishes a cost-effective, scalable, and highly customizable solution for genome editing validation in CGT applications.

"Safety is key to unlocking the true promise of CRISPR, and this collaboration with Mission Bio reflects another step forward to advancing the delivery of safer and more efficient genome editing platforms," said Sandy Ottensmann, VP/GM, Gene Writing and Editing at IDT. "By pairing the precision of rhAmpSeq with the single-cell resolution of Tapestri, we aim to support cell and gene therapy developers in driving the future of safe, life-changing therapies to benefit more patients."

Researchers and industry professionals can learn more about this cutting-edge approach at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting this month. Mission Bio and IDT will be presenting a poster detailing the study’s findings, demonstrating the power of integrated single-cell sequencing for genome editing validation.

The poster "Comprehensive On- and Off-target Validation Using Integrated rhAmpSeq and Targeted DNA Resequencing Single-Cell Technology for Gene Editing Applications" will be presented on Thursday, May 15 at 5:30 pm CST.

An oral presentation delivered by Dr. Ayal Hendel of Bar-Ilan University, "Single-Cell Profiling of Genome-Editing Alterations and Functional Outcomes in CRISPR-Engineered Cells," will take place on Saturday, May 17 at 9:00 am CST.

Visit Mission Bio’s Booth #1749 at ASGCT (Free ASGCT Whitepaper) to see how this technology can accelerate your research.

For more information, visit missionbio.com or contact [email protected].

On May 6, 2025 Leidos reported Strong First Quarter Results and Reaffirms Full-Year Guidance (Press release, Leidos, MAY 6, 2025, View Source [SID1234654244]).

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