On May 13, 2024 Pheast Therapeutics ("Pheast"), a biotech developing novel macrophage checkpoint therapies to defy cancer, reported the first presentation of preclinical data for PHST001, an anti-CD24 antibody drug candidate that is designed to block a key macrophage "don’t eat me" signal on cancer cells (Press release, Pheast Therapeutics, MAY 13, 2024, View Source [SID1234643177]). The data to be presented at the 20th Annual PEGS Boston Summit demonstrate that PHST001 can powerfully induce an anti-cancer immune response and drive therapeutic efficacy in in vivo models.

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"Pheast’s lead drug candidate, PHST001, is an exceptionally well-engineered, well-expressed antibody against an exciting new target, CD24," said Roy Maute, Ph.D., Cofounder and CEO, Pheast Therapeutics. "Our preclinical studies show potent efficacy in challenging mouse model systems, which supports the possibility of clinical efficacy in cancers where other immunotherapies have failed and the unmet need remains high. The Pheast team is making rapid progress to support filing an investigational new drug application with the goal to enter the clinic in patients in the first half of next year."

CD24 is a newly discovered immune checkpoint that is highly expressed by many human cancers, including ovarian and triple negative breast cancer (TNBC). High expression of CD24 is a negative prognostic factor in multiple cancer indications. CD24 interacts with the macrophage receptor Siglec-10, and shields cancer cells from attack by macrophages. Pheast has engineered PHST001 to bind recombinant CD24 and cell surface CD24 with high affinity and specificity and to block Siglec-10 binding.

Pheast scientists have demonstrated that blocking CD24 with PHST001 induces macrophage phagocytosis of multiple cancer subtypes in vitro, and have shown potent efficacy for PHST001 in vivo. In some preclinical models, PHST001 monotherapy is sufficient to shrink and fully eradicate tumors in all treated mice.

"These data demonstrate Pheast’s robust in-house antibody discovery and protein engineering capabilities, allowing rapid discovery and development of therapeutic monoclonal antibodies," said John Burg, Ph.D., Senior Director, Protein Sciences. "Our innovative platform for discovery of tumor-specific immune checkpoints for precision immuno-oncology treatments will support exploring additional targets and developing new pipeline programs."

Dr. Burg will present these data on Monday, May 13, 2024, at 1:15 pm ET in a talk titled, "Engineering a Therapeutic Monoclonal Antibody Targeting CD24, a Macrophage Checkpoint."

On May 13, 2024 Circio Holding ASA (OSE: CRNA), a biotechnology company developing circular RNA-based gene therapy, reported that it has presented two posters that demonstrate in vivo proof-of-concept for its powerful and differentiated circVec platform approach to gene therapy (Press release, Circio, MAY 13, 2024, View Source [SID1234643139]). The two posters were presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2024 annual meeting 7-11 May in Baltimore, USA

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"Circio has generated results demonstrating that the circVec 2.1 design performs very well in vitro. We have now confirmed this in vivo with statistically significant higher expression level and durability for circVec 2.1 DNA vectors compared to standard linear mRNA-based expression. These results provide an important technical proof-of-concept for Circio´s technology platform in an animal model. We now have confirmation for our expectation that this could translate into improved gene therapies for patients in the future," said Dr. Thomas B Hansen, CTO at Circio. "In recent experiments, Circio has observed up to four months circVec durability in vivo. This substantially outperforms mRNA vector expression. Following these results, we can rapidly advance to design and test circVec in several AAV and DNA-based vectors. This will validate these very promising data in therapeutically relevant formats."

At ASGCT (Free ASGCT Whitepaper), Circio also presented the dual-function ‘remove-&-replace’ concept for Alpha-1-antitrypsin deficiency (AATD). This genetic disease causes severe symptoms in the lung and liver. There are currently no satisfactory therapeutic options available for this indication and AATD still represents a major unmet medical need. There are over 200,000 AATD patients affected in the USA and EU alone. With the technologically differentiated circVec remove-&-replace format, Circio has developed a unique gene therapy concept that can deal with both the lung and liver-associated symptoms in one single therapeutic.

"AATD is a challenging genetic disease to treat. This is in part due to the two distinct pathologies in the liver and lung," said Dr. Victor Levitsky, CSO at Circio. "We have now established and technically validated circVec constructs that can both replenish functional wild-type AAT and specifically remove more than 90% of the mutated protein. This is challenging to achieve because the functional and mutant forms are very similar. By using circular RNA-based AAT expression, Circio is uniquely able to separate the two species for mutant-specific knockdown, thereby solving two problems with one single product."

Optimization and In Vivo Performance of circVec, a Vector-Based Circular RNA Expression Platform; O´Leary et al. ASGCT (Free ASGCT Whitepaper) 2024

Expressing AAT from circular RNA-encoding vectors as a promising gene therapy approach for Alpha 1-antitrypsin deficiency; O´Leary et al. ASGCT (Free ASGCT Whitepaper) 2024

On May 13, 2024 Sutro Biopharma, Inc. (Sutro or the Company) (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), reported its financial results for the first quarter of 2024, its recent business highlights, and a preview of select anticipated milestones (Press release, Sutro Biopharma, MAY 13, 2024, View Source [SID1234643156]).

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"The Sutro team executed on multiple fronts in the first quarter of 2024, advancing luvelta through the clinic in multiple indications of high unmet need, continuing to progress our pipeline and collaboration programs, and establishing a new licensing relationship with Ipsen. The upfront funding from the Ipsen deal and our recent financing also augmented our strong cash position," said Bill Newell, Sutro’s Chief Executive Officer. "We plan to deliver on important catalysts throughout 2024, reporting on expanded patient data with luvelta in combination with bevacizumab, the initiation of a registrational trial for pediatric patients with a rare form of acute myeloid leukemia (AML), and a Phase 2 trial in non-small cell lung cancer (NSCLC). We continue to build upon our momentum and are well positioned on our goal to rapidly deliver precisely designed ADCs to patients in need."

Recent Business Highlights and Select Anticipated Milestones

Luveltamab Tazevibulin (luvelta), FolRα-Targeting ADC Franchise:

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Part 1 (dose-optimization) of the registration-directed trial, REFRαME-O1, for treatment of platinum-resistant ovarian cancer (PROC), has completed enrollment. Part 2 (randomized portion) is now enrolling, with an anticipated ~140 sites in ~20 countries planned to be opened by the end of 2024.
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Enrollment of REFRαME-P1, a registration-enabling trial for pediatric patients with CBFA2T3::GLIS2 (CBF/GLIS; RAM phenotype) AML, is expected to be initiated in the second half of 2024.
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An Investigational New Drug (IND) application for the treatment of NSCLC has been cleared by U.S. Food and Drug Administration (FDA). The Phase 2 trial is expected to begin enrolling patients in the second half of 2024. Initial data is expected in the first half of 2025.
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A Phase 2 expansion study in combination with bevacizumab is well underway. Enrollment is expected to be complete in the first half of 2024.

Additional Pipeline Development and Collaboration Updates:

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In April 2024, Sutro announced a global licensing agreement for STRO-003, a ROR1-targeting ADC, with Ipsen. Sutro is eligible to receive up to $899 million in upfront and potential milestone payments, including up to $92 million in near-term payments, of which $75 million, including an equity investment, have been received in April. Sutro is also eligible to receive tiered royalties ranging from low double-digit to mid-teen digit percentages on annual global sales of STRO-003.
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Sutro plans to submit an IND for STRO-004, a tissue factor-targeting ADC, in 2025.
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Sutro continues to seek to maximize the value of its proprietary cell-free platform by working with partners on programs in multiple disease spaces and geographies and has generated from collaborators an aggregate of approximately $864 million in payments through March 31, 2024, including equity investments.
Corporate Updates:

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Additionally, Sutro strengthened its cash position with an underwritten offering of 14,478,764 shares of its common stock at a price of $5.18 per share, resulting in gross proceeds of $75.0 million. The offering was led by a high-quality group of new and existing healthcare-focused institutional investors.
Upcoming Events: Sutro will participate in two upcoming investor conferences. Webcasts of the presentations will be accessible through the News & Events page of the Investor Relations section of the Company’s website at www.sutrobio.com. Archived replays will be available for at least 30 days after the events.

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The Citizens JMP Life Sciences Conference in New York, May 13-14, 2024
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Jefferies Healthcare Conference in New York, June 5-6, 2024
First Quarter 2024 Financial Highlights

Cash, Cash Equivalents and Marketable Securities

As of March 31, 2024, Sutro had cash, cash equivalents and marketable securities of $267.6 million, and approximately 0.7 million shares of Vaxcyte common stock with a fair value of $45.6 million.

Unrealized Gain from Increase in Value of Vaxcyte Common Stock

The non-operating, unrealized gain of $3.7 million for the quarter ended March 31, 2024 was due to the increase since December 31, 2023 in the estimated fair value of Sutro’s holdings of Vaxcyte common stock. Vaxcyte common stock held by Sutro will be remeasured at fair value based on the closing price of Vaxcyte’s common stock on the last trading day of each reporting period, with any non-operating, unrealized gains and losses recorded in Sutro’s statements of operations.

Revenue

Revenue was $13.0 million for the quarter ended March 31, 2024, as compared to $12.7 million for the same period in 2023, with the 2024 amount related principally to the Astellas collaboration, and the Tasly and Vaxcyte agreements. Future collaboration and license revenue under existing agreements, and from any additional collaboration and license partners, will fluctuate as a result of the amount and timing of revenue recognition of upfront, milestones, and other agreement payments.

Operating Expenses

Total operating expenses for the quarter ended March 31, 2024 were $69.6 million, as compared to $54.9 million for the same period in 2023. The 2024 quarter includes non-cash expenses for stock-based compensation of $6.1 million and depreciation and amortization of $1.8 million, as compared to $6.0 million and $1.6 million, respectively, in the comparable 2023 period. Total operating expenses for the quarter ended March 31, 2024 were comprised of research and development expenses of $56.9 million and general and administrative expenses of $12.7 million.

On May 13, 2024 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, reported its first quarter 2024 unaudited financial results and key corporate highlights (Press release, Legend Biotech, MAY 13, 2024, View Source [SID1234643178]).

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"Legend made great progress in the first quarter, culminating in our exciting announcements in recent weeks. We received label expansions for CARVYKTI in the U.S., Europe, and Brazil that have changed the treatment paradigm for multiple myeloma and will enable more patients to receive our transformative therapy earlier in the course of their disease," said Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech. "With more patients needing access to CARVYKTI, we have increased our manufacturing capacity and have scaled up our operations to reach our goal of 10,000 annual doses by the end of 2025. The expansion of our partnership with Novartis demonstrates our commitment to ensuring every patient who needs CARVYKTI can access it."

Regulatory Updates

The U.S. Food and Drug Administration (FDA) approved CARVYKTI for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD) and are refractory to lenalidomide following the Oncologic Drug Advisory Committee’s (ODAC) unanimous (11 to 0) vote recommending the approval of CARVYKTI.
The European Commission (EC) granted approval for the label expansion of CARVYKTI for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least one prior therapy, including an immunomodulatory agent and a proteasome inhibitor, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide.
The Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), approved CARVYKTI for the treatment of adult patients with multiple myeloma, who previously received a proteasome inhibitor and are refractory to lenalidomide, as well as adult patients with relapsed or refractory multiple myeloma, who previously received a proteasome inhibitor, an immunomodulatory agent and anti-CD38 antibody.
Key Business Developments

Legend and Johnson & Johnson* entered into a Master Manufacturing and Supply Services Agreement with Novartis Pharmaceuticals Corporation to supplement our existing manufacturing capabilities and increase commercial supply of CARVYKTI
Published inaugural Environmental, Social & Governance (ESG) report which aligns with the Sustainable Accounting Standards Board (SASB) Biotechnology and Pharmaceutical sector standards, shares ESG data collection and disclosure roadmap, and future growth strategy for good corporate citizenship
* In December 2017, Legend Biotech entered into an exclusive worldwide collaboration and license agreement with Janssen Biotech, Inc., a Johnson & Johnson company, to develop and commercialize cilta-cel (the Janssen Agreement).

First Quarter 2024 Financial Results

License Revenue: License revenue was $12.2 million for the first quarter of 2024 and consisted of the recognition of deferred revenue in connection with the global license agreement with Novartis Pharma AG to develop, manufacture, and commercialize LB2102 and other potential CAR-T therapies selectively targeting DLL3. Legend did not recognize any license revenue for the first quarter of 2023.
Collaboration Revenue: Collaboration revenue was $78.5 million for the first quarter of 2024 compared to $36.3 million for the first quarter of 2023. The increase was primarily due to an increase in revenue generated from sales of CARVYKTI in connection with the Janssen Agreement.
Collaboration Cost of Revenue: Collaboration cost of revenue was $49.1 million for the first quarter of 2024 compared to $35.6 million for the first quarter of 2023. The increase was primarily due to Legend Biotech’s share of the cost of sales in connection with CARVYKTI sales under the Janssen Agreement.
Cost of License and Other Revenue: Cost of license and other revenue for the three months ended March 31, 2024 was $5.6 million and consisted of costs in connection with the global license agreement with Novartis Pharma AG to develop, manufacture, and commercialize LB2102 and other potential CAR-T therapies selectively targeting DLL3. The Company did not incur any cost of license and other revenue for the three months ended March 31, 2023.
Research and Development Expenses: Research and development expenses were $101.0 million for the first quarter of 2024 compared to $84.9 million for the first quarter of 2023. The increase was primarily driven by continuous research and development activities in cilta-cel, including start up costs for clinical production in Belgium and continued investment in Legend’s solid tumor programs.
Administrative Expenses: Administrative expenses were $31.9 million for the first quarter of 2024 compared to $22.2 million for the first quarter of 2023. The increase was primarily due to the expansion of administrative functions and infrastructure to increase manufacturing capacity.
Selling and Distribution Expenses: Selling and distribution expenses were $24.2 million for the first quarter of 2024 compared to $18.0 million for the first quarter of 2023. The increase was primarily driven by costs associated with commercial activities for cilta-cel, including the expansion of the sales force and second line indication launch preparation.
Net Loss: Net loss was $59.8 million for the first quarter of 2024, compared to a net loss of $112.1 million for the first quarter of 2023.
Cash Position: Cash and cash equivalents, time deposits, and short-term investments were $1.3 billion as of March 31, 2024.
Webcast/Conference Call Details:
Legend Biotech will host its quarterly earnings call and webcast today at 8:00 am ET. To access the webcast, please visit this weblink.

A replay of the webcast will be available on Legend Biotech’s website at View Source

On May 13, 2024 Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage biopharmaceutical company focused on developing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer, reported financial results for the first quarter ended March 31, 2024, and provided recent corporate updates (Press release, Galera Therapeutics, MAY 13, 2024, View Source [SID1234643140]).

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"Our review of strategic options continues, as we strive to maximize value for our stockholders," said Mel Sorensen, M.D., Galera’s President and CEO. "Potential options may include mergers, asset sales, divestiture, licensing arrangements, or other strategic transactions and may encompass a potential development path for avasopasem. The process could ultimately culminate in the dissolution of the Company."

General Corporate Updates

Galera remains actively engaged with Stifel, Nicolaus & Company, Inc. to undertake a comprehensive review of strategic alternatives for both the Company and its portfolio of dismutase mimetics. The Company has not set a fixed timeline for completing this evaluation process and does not intend to disclose further updates unless and until it is determined that further disclosure is appropriate or necessary.
On May 3, 2024, the Company announced that its Board of Directors (the "Board") unanimously resolved to adopt a limited duration stockholder rights agreement (the "Rights Agreement") to protect stockholder interests. The Board resolved to adopt the Rights Agreement in response to recent accumulations of the Company’s common stock, and the Rights Agreement is intended to enable all Galera stockholders to realize the full potential value of their investment in Galera and to protect the interests of the Company and its stockholders by reducing the likelihood that any person or group gains control of Galera without paying an appropriate control premium. In addition, the Rights Agreement provides the Board with time to make informed decisions that are in the best long-term interests of Galera and its stockholders. It does not deter the Board from considering any offer or proposal that is fair and otherwise in the best interest of Galera stockholders.
First Quarter 2024 Financial Highlights

Research and development expenses were $1.5 million in the first quarter of 2024, compared to $7.3 million for the same period in 2023. The decrease was primarily attributable to a decrease in avasopasem and rucosopasem development costs. The Company has ceased all clinical trial activity and suspended the clinical development of its product candidates as it explores potential strategic alternatives.
General and administrative expenses were $3.1 million in the first quarter of 2024, compared to $6.6 million for the same period in 2023. The decrease was primarily attributable to the cessation of avasopasem commercial preparations and medical affairs activities and reduced personnel-related expenses due to the workforce reduction announced in August 2023.
Galera reported a net loss of $(4.4) million, or $(0.08) per share, for the first quarter of 2024, compared to a net loss of $(17.7) million, or $(0.50) per share, for the same period in 2023.
As of March 31, 2024, Galera had cash and cash equivalents of $13.5 million. Galera expects that its existing cash and cash equivalents will enable Galera to fund its operating expenses and capital expenditure requirements into the third quarter of 2025.