On March 17, 2025 CEL-SCI Corporation ("CEL-SCI" or the "Company") (NYSE American: CVM), a Phase 3 cancer immunotherapy company, reported the pricing of a best-efforts offering of 16,000,000 shares of its common stock (or pre-funded warrants ("Pre-Funded Warrants") in lieu thereof) (Press release, Cel-Sci, MAR 17, 2025, View Source [SID1234651204]). Total gross proceeds from the offering, before deducting the placement agent’s fees and other offering expenses, are expected to be approximately $2,560,000. The offering is expected to close on March 18, 2025, subject to satisfaction of customary closing conditions.

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The Company intends to use the net proceeds from the offering to fund the continued development of Multikine, general corporate purposes, and working capital.

ThinkEquity is acting as sole placement agent for the offering.

The securities will be offered and sold pursuant to a shelf registration statement on Form S-3 (File No. 333-265995), including a base prospectus, filed with the U.S. Securities and Exchange Commission (the "SEC") on July 1, 2022, and declared effective on July 15, 2022. The offering will be made only by means of a written prospectus. A final prospectus supplement and accompanying prospectus describing the terms of the offering will be filed with the SEC on its website at www.sec.gov. Copies of the prospectus supplement and the accompanying prospectus relating to the offering may also be obtained, when available, from the offices of ThinkEquity, 17 State Street, 41st Floor, New York, New York 10004.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On March 17, 2025 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported financial results and business highlights for the fourth quarter and year ended December 31, 2024 (Press release, Sana Biotechnology, MAR 17, 2025, View Source [SID1234651186]).

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"The updated preliminary 12-week clinical data for UP421, showing stable C-peptide production and a positive mixed meal tolerance test over time, build upon the earlier reported 4-week data and increase our confidence that we can successfully transplant hypoimmune-modified pancreatic islets into a type 1 diabetes patient without any immunosuppression, a result we view as transformational for the company and the field," said Steve Harr, Sana’s President and Chief Executive Officer. "With these data and our progress in manufacturing, we are increasingly optimistic about the potential for SC451 – a gene-modified, stem cell-derived pancreatic islet cell therapy with a goal of single treatment that leads to normal blood glucose with no more insulin injections or immunosuppression, and we look forward to presenting more data from our type 1 diabetes program in 2025 and to filing an IND as early as 2026. We expect to share clinical data later this year from two clinical-stage programs, SC291 and SC262, and we are making meaningful progress in moving forward SG299 (in vivo CAR T cell, with no lymphodepleting chemotherapy, for the treatment of B-cell mediated autoimmune diseases and B-cell cancers) with an expected IND filing as early as 2026. Overall, our investments in research across the hypoimmune platform, stem cell biology, and in vivo delivery continue to strengthen our ability to make potentially transformative medicines, and we look optimistically to our future."

Recent Corporate Highlights

Announced positive initial results from an investigator-sponsored, first-in-human study transplanting UP421, an allogeneic primary islet cell therapy engineered with hypoimmune platform (HIP) technology, into a patient with type 1 diabetes without the use of any immunosuppression.

UP421 is a primary human HIP-modified pancreatic islet cell therapy for patients with type 1 diabetes. The goal of this investigator-sponsored trial (IST) is to understand safety, immune evasion, islet cell survival, and beta cell function, as measured by C-peptide production, of HIP-modified pancreatic islet cells in type 1 diabetics without any immunosuppression. The trial is being conducted under a clinical trial authorization (CTA) at Uppsala University Hospital with Dr. Per-Ola Carlsson as the principal investigator.
Results of the study at four- and 12-weeks after cell transplantation demonstrate the survival and function of pancreatic beta cells as measured by the presence of circulating C-peptide, a biomarker indicating that transplanted beta cells are producing insulin. C-peptide levels also increase with a mixed meal tolerance test (MMTT) during testing at these timepoints, consistent with insulin secretion in response to a meal. Magnetic resonance imaging (MRI) scanning also demonstrates a sustained signal at the site of transplanted cells over time, which is consistent with graft survival. The study identified no safety issues, and the HIP-modified islet cells evaded immune detection.
Sana expects to report additional data from this study, including longer-term follow-up, as the year progresses in a peer-reviewed publication and/or at scientific conferences. The preliminary 12-week results remain subject to source data verification.
Advancing our pipeline across multiple indications and modalities:

Type 1 Diabetes – Sana continues the clinical development of gene-modified primary islet cells (UP421) and the pre-clinical development of SC451, a HIP-modified, stem cell-derived pancreatic islet cell therapy. In addition to the human data for UP421 outlined above, Sana shared data for SC451 showing 15-month durability of glycemic control in a mouse model and no histologic abnormalities. Sana expects to share additional data in 2025 and file an IND as early as 2026.
Allogeneic CAR T cells – The GLEAM study is a Phase 1 study evaluating SC291, a HIP-modified CD19-directed allogeneic CAR T cell therapy, in patients with B-cell mediated autoimmune diseases, including refractory systemic lupus erythematosus and antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis. The VIVID study is a Phase 1 clinical trial evaluating SC262, a HIP-modified CD22-directed CAR T cell therapy, in patients with relapsed and/or refractory B-cell malignancies who have received prior CD19-directed CAR T therapy.
Data from the suspended ARDENT trial evaluating SC291 in relapsed or refractory non-Hodgkin lymphoma (NHL) and chronic lymphocytic leukemia (CLL) demonstrated the ability to safely dose SC291 with the desired deep B-cell depletion. The goal in the GLEAM study is to demonstrate similar deep B-cell depletion with subsequent clinical benefit for patients with B-cell mediated autoimmune diseases.
Sana is enrolling patients in both the GLEAM and VIVID trials and expects to share data from each study in 2025.
in vivo CAR T cells – SG299, which uses our fusogen platform, allows for cell-specific, in vivo delivery of various payloads. SG299 is a CD8-targeted fusosome that delivers to CD8+ T cells the genetic material to make CD19-directed CAR T cells while avoiding delivery to potentially troublesome tissues such as the liver and gonadal tissue. Sana recently shared data showing that an SG299 surrogate with another component can lead to deep B-cell depletion in non-human primates without the use of any lymphodepleting chemotherapy. Sana expects to file an IND for SG299 as early as 2026, and we look forward to developing it in a range of B-cell cancers and B-cell mediated autoimmune diseases.
Published preclinical data in Cell Stem Cell demonstrating that HIP-modified allogeneic islet cells provided lasting endocrine function in a fully immunocompetent non-human primate with type 1 diabetes, enabling the achievement of exogenous insulin independence without immunosuppression:

Sana developed HIP-modified allogeneic islet cells, which cluster into effective endocrine organoids termed "pseudo islet grafts" (p-islets). HIP p-islets engrafted and provided stable endocrine function, enabling insulin independence without immunosuppression.
The allogeneic HIP p-islet graft survived for the six-month duration of the study with no indication of immune recognition of the HIP p-islet engraftment at any time.
To demonstrate that there was no regeneration or recovery of an endogenous islet cell population in the diabetic NHP, HIP p-islets were eliminated after 6 months using an anti-CD47 antibody, demonstrating proof of principle of CD47 overexpression and a potential safety switch.
These data complement the clinical data seen with UP421, highlighting the potential of the HIP-modified pancreatic islets for patients with type 1 diabetes.
Strengthened leadership with the appointment of new Chief Scientific Officer and Chief People Officer

Appointed Dhaval Patel, M.D., Ph.D., as Executive Vice President and Chief Scientific Officer. Dr. Patel has decades of experience in research, drug discovery, drug development, and clinical care – including roles at UCB, Novartis, University of North Carolina, and the Duke University School of Medicine – and over the course of his career has participated in the development of 10 approved drugs in multiple indications.
Appointed Tricia Stewart as Executive Vice President and Chief People Officer. Ms. Stewart has decades of experience in the human resources function in both the biotechnology and services industries, including roles at Genentech, Roche, and Fibrogen, and has developed programs to transform company culture, organizational structure, total rewards, employee engagement, and talent management.
Fourth Quarter 2024 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of December 31, 2024 were $152.5 million compared to $205.2 million as of December 31, 2023. The decrease of $52.7 million was primarily driven by cash used in operations of $223.2 million and cash used for the purchase of property and equipment of $33.4 million, partially offset by net proceeds from equity financings of $181.0 million, proceeds from stock option exercises and Sana’s employee stock purchase plan of $11.0 million, and net proceeds of $7.7 million from a loan to fund tenant improvements for Sana’s manufacturing facility in Bothell, Washington during the year ended December 31, 2024.
Research and Development Expenses: For the three and twelve months ended December 31, 2024, research and development expenses, inclusive of non-cash expenses, were $47.0 million and $217.6 million, respectively, compared to $63.0 million and $268.8 million for the same periods in 2023. The decreases of $16.0 million and $51.2 million for the three and twelve months ended December 31, 2024, respectively, compared to the same periods in 2023 were primarily due to lower personnel-related and laboratory costs due to a decrease in headcount and decreased research costs related to the portfolio prioritizations in the fourth quarters of 2024 and 2023, lower costs for third-party manufacturing at contract development and manufacturing organizations, and a decline in facility and other allocated costs. These decreases were partially offset by increased clinical development costs. Research and development expenses include non-cash stock-based compensation of $3.9 million and $23.4 million for the three and twelve months ended December 31, 2024, respectively, and $4.9 million and $23.2 million for the same periods in 2023.
Research and Development Related Success Payments and Contingent Consideration: For the three and twelve months ended December 31, 2024, Sana recognized non-cash gains of $13.4 million and $8.9 million, respectively, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate, compared to a non-cash expense of $6.8 million and a non-cash gain of $49.0 million for the same periods in 2023. The value of these potential liabilities fluctuate significantly with changes in Sana’s market capitalization and stock price.
General and Administrative Expenses: General and administrative expenses for the three and twelve months ended December 31, 2024, inclusive of non-cash expenses, were $17.3 million and $64.0 million, respectively, compared to $20.8 million and $73.3 million for the same periods in 2023. The decreases of $3.5 million and $9.3 million for the three and twelve months ended December 31, 2024, respectively, compared to the same periods in 2023 were primarily due to a decrease in legal fees, a loss on lease termination associated with Sana’s previously planned manufacturing facility in Fremont, California (Fremont facility) recorded in 2023, a decrease in personnel-related costs related to the portfolio prioritizations in 2024 and 2023, a decrease in facility costs, and a decrease in insurance and consulting expenses. These decreases were partially offset by an increase in non-cash stock-based compensation. General and administrative expenses include non-cash stock-based compensation of $2.5 million and $14.3 million for the three and twelve months ended December 31, 2024, respectively, and $3.1 million and $12.3 million for the same periods in 2023.
Net Loss: Net loss for the three and twelve months ended December 31, 2024 was $49.1 million, or $0.21 per share, and $266.8 million, or $1.16 per share, respectively, compared to $88.1 million, or $0.45 per share, and $283.3 million, or $1.46 per share, for the same periods in 2023.

On March 17, 2025 Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, reported recent business highlights and fourth quarter and full year 2024 financial results (Press release, Sangamo Therapeutics, MAR 17, 2025, View Source [SID1234651187]).

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"I am pleased with Sangamo’s pipeline progress since the start of 2024. We advanced our two prioritized neurology therapies towards the clinic, securing our first ever neurology IND; we showed we are a collaborator of choice for neurotropic capsids, with the announcement of two blue-chip pharma agreements for our STAC-BBB capsid, with negotiations advancing for a third potential agreement; and we have a clear regulatory pathway to Accelerated Approval in Fabry disease, which could reduce the time to potential approval by approximately three years," said Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics. "We believe our neurology pipeline represents important potential value. In addition, we continue to engage in Fabry business development negotiations, in an effort to capitalize the business for the future. This will be an important year for the Company as we plan to begin patient enrollment and dosing in mid-2025 for our clinical study in iSFN, which we believe has the potential to transform the chronic neuropathic pain landscape, and as we prepare for an anticipated BLA submission in Fabry disease in the second half of the year."
Recent Business Highlights

Corporate Updates
•Announced in December a capsid license agreement with Astellas Gene Therapies, Inc. (Astellas) to deliver genomic medicines for neurological diseases. Agreement grants Astellas a worldwide exclusive license to STAC-BBB for up to five potential neurological disease targets. Received a $20 million upfront license fee from Astellas and eligible to earn up to $1.3 billion in additional licensed target fees and milestone payments across all five potential neurology disease targets, as well as tiered royalties on potential net sales.
•Announced in December that Sangamo is scheduled to regain full rights to giroctocogene fitelparvovec, an investigational gene therapy product candidate for the treatment of adults with moderately severe to severe hemophilia A that it has co-developed with, and licensed to, Pfizer, Inc. (Pfizer), following a decision by Pfizer to terminate the global collaboration and license agreement between the parties. Sangamo continues to explore how to maximize the value of the SB-525 program, including a search for a potential new collaboration partner.
Core Neurology Pipeline
Chronic Neuropathic Pain – ST-503
•IND application cleared by the FDA for ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to iSFN, a type of chronic neuropathic pain.
•Preparing for a Phase 1/2 study of ST-503 to assess the safety, tolerability and preliminary efficacy of a one-time dose administered intrathecally to patients with intractable pain due to iSFN.
•Expect to commence patient enrollment and dosing in mid-2025, with preliminary proof of efficacy data anticipated in Q4 2026.

Prion Disease

•Clinical Trial Authorisation (CTA) enabling activities continue to advance for Sangamo’s product candidate to treat prion disease, leveraging STAC-BBB.
•Published a manuscript in bioRxiv titled, "Zinc Finger Repressors mediate widespread prion depletion from the nonhuman primate brain and profoundly extend survival in prion disease mice" demonstrating nonclinical proof of concept for this approach. A single intravenous infusion of Sangamo’s ZFR significantly reduced expression of prion mRNA and protein in the mouse brain, extended mouse survival and improved an array of molecular, histological, biomarker and behavior readouts – even when administered post-symptomatically to mice with prion disease. In addition, a single intravenous administration of the prion ZFR, delivered via STAC-BBB to nonhuman primates, resulted in potent and widespread reduction of prion expression in transduced neurons throughout the brain.
•A CTA submission is expected in Q1 2026, with preliminary clinical data anticipated in Q4 2026.
Novel Adeno-Associated Virus (AAV) Capsid Delivery Technology
•Actively engaged in advanced contract negotiations with a potential collaborator for a third STAC-BBB license agreement for use in delivering intravenously administered genomic medicines for certain specified neurological diseases.
Clinical – Fabry Disease
•Presented updated Phase 1/2 STAAR study data at the 21st Annual WORLDSymposium in San Diego, CA in February 2025 showing sustained benefit, improvements in kidney function and a favorable safety profile, following a single administration of isaralgagene civaparvovec in 33 adults with Fabry disease.
•Elevated expression of alpha-galactosidase A (α-Gal A) activity maintained for nearly four years for the longest treated patient as of the September 12, 2024 data cutoff date.
•Positive mean estimated glomerular filtration rate (eGFR) slope of 3.061 mL/min/1.73m2/year (95% confidence interval: 0.863, 5.258) was observed in the 23 patients who had reached at least one-year follow-up, indicating notable improvements in renal function.
•All 18 patients who began the study on enzyme replacement therapy (ERT) have been withdrawn from, and remain off, ERT as of March 17, 2025.
•Significant improvements continued to be observed in the short form-36 (SF-36) quality of life (QoL) scores reported, with a mean change in General Health score of 10.6. Significant improvements in physical component, bodily pain, physical, vitality, social function, and emotional SF-36 scores were also observed.
•Enrollment and dosing are complete in the Phase 1/2 STAAR study.
•The FDA has provided a clear regulatory pathway to Accelerated Approval for isaralgagene civaparvovec, agreeing that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the Accelerated Approval Program, using eGFR slope at 52 weeks across all patients as an intermediate clinical endpoint.
•The 52-week eGFR slope data from all enrolled patients in the Phase 1/2 STAAR study will be available in the first half of 2025. A potential Biologics License Application (BLA) submission is anticipated in the second half of 2025.
•Sangamo is advancing BLA preparation activities for isaralgagene civaparvovec, while continuing to engage in business development negotiations for a potential Fabry commercialization agreement.

Fourth Quarter and Full Year 2024 Financial Results
Consolidated net loss for the fourth quarter ended December 31, 2024 was $23.4 million, or $0.11 per share, compared to consolidated net loss of $60.3 million, or $0.34 per share, for the same period in 2023. For the year ended December 31, 2024, consolidated net loss was $97.9 million, or $0.49 per share, compared to consolidated net loss of $257.8 million, or $1.48 per share, for the year ended December 31, 2023.
Revenues
Revenues for the fourth quarter ended December 31, 2024 were $7.6 million, compared to $2.0 million for the same period in 2023.
The increase of $5.5 million in revenues was primarily attributed to $6.5 million and $0.8 million in revenues relating to our license agreements with Astellas Gene Therapies, Inc., or Astellas, and Genentech, Inc., respectively, partially offset by revenue decreases in other collaborations.
Revenues were $57.8 million in 2024, compared to $176.2 million in 2023.
The decrease of $118.4 million in revenues in 2024 compared to 2023 was primarily attributed to decreases of $134.8 million and $12.2 million in revenues relating to our collaboration agreements with Biogen and Novartis, respectively, due to the termination of these collaboration agreements in June 2023, a decrease of $20.5 million in revenue relating to our collaboration agreement with Kite, which expired pursuant to its terms in April 2024, a decrease of $4.7 million in revenue relating to our license agreements with Sigma and Ligand, and a decrease of $2.7 million in revenue relating to our other license agreements. These decreases were partially offset by $50.0 million in revenue relating to our license agreement with Genentech and $6.5 million in revenue relating to our license agreement with Astellas.
GAAP and Non-GAAP Operating Expenses
(In millions)
Three Months Ended
December 31, Year Ended
December 31,
2024 2023 2024 2023
Research and development $ 23.6 $ 50.7 $ 111.5 $ 234.0
General and administrative 9.9 13.1 44.8 61.2
Impairment of long-lived assets — 0.3 5.5 65.5
Impairment of goodwill and indefinite-lived intangible assets — — — 89.5
Total operating expenses 33.5 64.1 161.8 450.2
Impairment of long-lived assets — (0.3) (5.5) (65.5)
Impairment of goodwill and indefinite-lived intangible assets — — — (89.5)
Depreciation and amortization (1.2) (1.8) (5.1) (15.1)
Stock-based compensation expense (3.3) (6.1) (12.4) (27.4)
Non-GAAP operating expenses $ 29.0 $ 55.9 $ 138.8 $ 252.7

Total operating expenses on a GAAP basis for the fourth quarter ended December 31, 2024 were $33.5 million compared to $64.1 million for the same period in 2023. Non-GAAP operating expenses, which exclude impairment charges, depreciation and amortization and stock-based compensation expense as shown in the reconciliation table above, for the fourth quarter ended December 31, 2024 were $29.0 million, compared to $55.9 million for the same period in 2023.
Total operating expenses on a GAAP basis in 2024 were $161.8 million compared to $450.2 million in 2023. Non-GAAP operating expenses, which exclude impairment charges, depreciation and amortization and stock-based compensation expense as shown in the reconciliation table above, were $138.8 million in 2024 compared to $252.7 million in 2023.
The decrease in total operating expenses on a GAAP basis was primarily driven by cost reductions resulting from the strategic realignment of the business, which included a lower headcount due to the restructuring of operations and corresponding reductions in workforce announced during 2023. Additionally, the decrease reflects intentional reprioritization of research and development investments, with a focused shift toward advancing the neurology pipeline. Other contributing factors included lower impairment charges recorded in the current year, lower preclinical and clinical expenses due to program deferrals, a decrease in restructuring charges related to the 2023 restructuring of operations, a decrease in depreciation due to reduced carrying values as a result of impairment charges recorded in 2023, and a decrease in external professional services, facilities, and infrastructure-related costs.

Cash and Cash Equivalents

Cash and cash equivalents as of December 31, 2024 were $41.9 million, compared to cash, cash equivalents and marketable securities of $81.0 million as of December 31, 2023. Based on our current operating plan, we believe that our cash and cash equivalents as of December 31, 2024, together with $10.1 million generated to date through our at-the-market offering program in 2025 and the $5.0 million payment expected from Pfizer by the end of March, will be sufficient to fund our planned operations into the middle of the second quarter of 2025.
Financial Guidance for 2025

•2025 operating expenses on a non-GAAP basis are expected to be roughly in line with 2024, reflecting our intention to operate a lean neurology-focused business and to continue advancing isaralgagene civaparvovec towards a potential BLA submission, while engaging in business development negotiations for a potential Fabry commercialization agreement.
•On a GAAP basis, we expect total operating expenses in the range of approximately $135 million to $155 million in 2025, which includes estimated non-cash stock-based compensation expense, and depreciation and amortization.
•We expect non-GAAP total operating expenses, excluding estimated non-cash stock-based compensation expense of approximately $7 million, and estimated depreciation and amortization of approximately $3 million, in the range of approximately $125 million to $145 million in 2025, consistent with the prior year.

Upcoming Events

Sangamo plans to participate in the following event:
•Jefferies Global Healthcare Conference, June 3-5, 2025
•Wells Fargo Healthcare Conference, September 3-5, 2025
Access links for available webcasts for investor conferences will be available on the Sangamo website in the Investors and Media section under Events. Available materials will be found on the Sangamo website after the event under Presentations.

Conference Call

The Sangamo management team will hold a corporate call to further discuss program and financial updates on Monday, March 17, at 4:30pm Eastern Time.

Participants should register for, and access, the call using this link. While not required, it is recommended you join 10 minutes prior to the event start. Once registered, participants will be given the option to either dial into the call with the number and unique passcode provided or to use the dial-out option to connect their phone instantly.
An updated corporate presentation is available in the Investors and Media section under Presentations.
The link to access the live webcast can also be found on the Sangamo website in the Investors and Media section under Events. A replay will be available following the conference call, accessible at the same link.

On March 17, 2025 Taiho Pharmaceutical Co., Ltd. (hereinafter "Taiho Pharmaceutical") and Araris Biotech AG (hereinafter "Araris"), a Swiss biotechnology company developing next-generation antibody drug conjugates (ADCs), reported that they have entered into a definitive agreement pursuant to which Taiho Pharmaceutical will fully acquire Araris (hereinafter "the Acquisition") (Press release, Taiho, MAR 17, 2025, View Source [SID1234651188]). Following necessary procedures, the Acquisition is expected to be completed in first half of 2025. The Acquisition follows a research collaboration between Taiho Pharmaceutical and Araris signed in November 2023.

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Under the terms of the agreement, Taiho Pharmaceutical will pay USD 400 million at closing, with the potential for additional milestone payments of up to USD 740 million.

Araris is a spin-off company of the Paul-Scherrer-Institute in Switzerland and pioneering the development of best-in-class ADCs with superior design, high linker solubility and simple manufacturing that address the shortcomings of current generation ADCs. ADCs are designed to selectively deliver cytotoxic drugs (payloads) to cancer cells by attaching them to antibodies that bind specifically to cancer cells through linkers. Foundational to its approach is its novel, proprietary ADC linker platform AraLinQ. This platform has generated highly uniform, stable and potent ADC therapeutic candidates that have demonstrated a wider range of safety and increased antitumor effect compared to conventional ADCs in preclinical studies.i,ii Furthermore, Araris is advancing three products for the treatment of hematological and solid tumors developed using its unique AraLinQ technology, which are currently in the preclinical stage. These products are anticipated to enter into clinical trials between 2025 and 2026.

Taiho Pharmaceutical, in addition to antimetabolites, has established and created novel drugs through its proprietary small molecule drug discovery platform, Cysteinomix, enhancing cancer treatment and contributing to patient care. By acquiring Araris’ innovative ADC drug discovery technology platform along with Cysteinomix, Taiho Pharmaceutical will further expand its ongoing development portfolio in the field of oncology.

Masayuki Kobayashi, President and Representative Director of Taiho Pharmaceutical commented, "We are very pleased to have entered into this agreement with Araris. AraLinQTM is an innovative technology that enables next-generation ADC drug discovery. We are confident that the addition of Araris’ knowledge, experience and technology platform in ADC drug discovery, as well as its development pipeline, will lead to further expansion and strengthening of Taiho’s drug discovery capabilities and portfolio. Going forward, together with Araris, we will strive to develop innovative drugs that can contribute to patients globally."

Dragan Grabulovski, CEO and Co-founder of Araris commented, "Araris’ unique ADC technology represents a quantum leap for the ADC field, potentially offering precise payload delivery of multiple mechanisms of action simultaneously to the tumor, with less toxicity. We are proud to combine with Taiho, our partner since November 2023, whose significant cancer expertise will support us in turbo-charging the clinical development of our potent ADC candidates in hematological and solid tumors."

Dima Kuzmin, Managing Partner of 4BIO Capital and Chairman of Araris, added: "This acquisition validates Araris’ position as one of the most exciting ADC companies in the market. Having worked with Araris closely during its collaboration, Taiho knows the potential of Araris’ proprietary AraLinQ technology and is best placed to accelerate these transformative treatments to patients."

With the Acquisition, Araris will become a wholly owned subsidiary of Taiho Pharmaceutical and will continue its business, research and development activities at its current location in Zurich, Switzerland.

About AraLinQTM

AraLinQTM is Araris’ linker technology, which enables site-specific payload attachment to a privileged attachment site on a specific amino acid (Q295) within the antibody IgGFc framework. When a payload is attached to this site, the antibody still maintains nearly identical performance (e.g. pharmacokinetics and effector functions) to the unconjugated, original antibody. Furthermore, the linker-payload is connected to the antibody through a very strong peptide bond resulting in exceptional stability in the blood stream and therefore, avoidance of healthy tissue damage. However, once entering a cancer cell via antibody mediated internalization, the linker can be easily broken to release the payload and kill the cancer cell. All three of these properties are key factors to enable the most efficient payload delivery and maximum ADC efficacy.

AraLinQTM linkers are hydrophilic, rendering them soluble in water-based solutions like blood. Improved solubility leads to decreased clumping, allowing the ADC to properly bind to cancerous cells without the need to further edit the antibody/payload structures. In addition, this linker can have unique branching structures which makes it possible to create ADCs that carry multiple payloads of different types. AraLinQTM can also generate ADCs in one step using "off-the-shelf" antibodies that are native or engineered. The process is fast, cost-efficient and can be easily upscaled without the need for custom antibody synthesis.

Advisors

MTS Health Partners, L.P is serving as a financial advisor and Wilson Sonsini Goodrich & Rosati PC and Homburger AG is serving as the legal advisor for Taiho Pharmaceutical. Centerview Partners UK LLP is serving as financial advisor to Araris, and Cooley LLP and BGPartner Ltd are serving as legal advisors.

On March 17, 2025 XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its fourth quarter and full year 2024 financial results and highlighted recent activities (Press release, Xoma, MAR 17, 2025, View Source [SID1234651191]).

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"Our balanced approach to building the scale of XOMA Royalty’s portfolio by selectively acquiring royalty economics across the lifecycle of drug development is beginning to bear fruit," stated Owen Hughes, Chief Executive Officer of XOMA Royalty. "Our growing commercial royalty portfolio of six assets is supported by VABYSMO (faricimab), OJEMDA, and MIPLYFFA, while our Phase 3 portfolio, which now totals 11 assets, promises several key readouts in 2025, including ersodetug from Rezolute, seralutinib from Gossamer Bio, and Ovaprene (non-hormonal vaginal ring) from Daré Biosciences. With over $100 million in cash on hand and a clear path to sustainable cashflow from royalties alone, we are well-positioned to further our goal of driving value for patients and shareholders alike."

Royalty and Milestone Acquisitions

Partner
Asset and Transaction Detail

Twist Bioscience XOMA Royalty completed a $15 million royalty monetization agreement with Twist, acquiring 50% of the future milestones and royalties in 60-plus partnered early-stage programs across 30 companies enabled by Twist Bioscience’s Biopharma Solutions business unit.
Daré Bioscience XOMA Royalty added economic interests to three best- or first-in-category assets to its portfolio for a $22 million upfront payment. XACIATO vaginal gel 2% is commercially available and marketed by Organon. Bayer holds the U.S. rights to commercialize Ovaprene, a hormone-free monthly intravaginal contraceptive, currently in Phase 3 clinical trials. XOMA Royalty also acquired a synthetic royalty in Sildenafil Cream, 3.6%, a Phase 3-ready asset for female sexual arousal disorder.

Talphera, Inc. XOMA Royalty acquired an economic interest in DSUVIA (sufentanil sublingual tablet) from Talphera, Inc., for $8 million. XOMA Royalty is entitled to royalties from DSUVIA sales. Alora Pharmaceuticals discontinued its DSUVIA commercial activities in November 2024. We remain eligible for payments from sales to the U.S. Department of Defense.
Company Acquisitions

Acquired Company
Rationale

Kinnate Biopharma Kinnate stockholders received $2.5879 per share in cash plus a Contingent Value Right (CVR) on April 3, 2024. The acquisition added approximately $7.8 million in cash and five assets to the XOMA Royalty portfolio.
Pulmokine Inc. XOMA Royalty secured a milestone and royalty interest in Gossamer Bio and Chiesi Farmaceutici’s seralutinib held by Pulmokine, a private company. Seralutinib is a Phase 3 asset being studied in pulmonary arterial hypertension (PAH), and Gossamer expects to initiate a registrational Phase 3 study in pulmonary hypertension associated with interstitial lung disease (PH-ILD) in 20251. Acquisition cost was $20 million upfront.
Product Approvals

Partner
Event

Day One Biopharmaceuticals The U.S. Food and Drug Administration (FDA) approved Day One’s OJEMDA (tovorafenib) for use in patients with pediatric low-grade glioma (pLGG). XOMA Royalty earned a $9.0 million milestone upon the approval and recorded $2.7 million in income resulting from OJEMDA sales in 2024. In addition, XOMA Royalty received an $8.1 million payment related to Day One’s sale of its priority review voucher.
Zevra Therapeutics The FDA approved Zevra’s MIPLYFFA (arimoclomol) capsules as an orally delivered treatment for Niemann-Pick disease type C (NPC). MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older.

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Out-licensing Activities

Partner
Event

Alexion In December 2024, following its acquisition of Amolyt, Alexion (an AstraZeneca company) exercised Amolyt’s option to continue developing anti-PTH1R monoclonal antibodies that originated from XOMA’s discovery efforts as potential treatments for primary hyperparathyroidism and humoral hypercalcemia of malignancy. XOMA Royalty will be eligible to receive up to $10.5 million in milestone payments and royalties ranging from low single to low double-digits on net commercial sales. Upon Alexion’s exercise of the option, XOMA Royalty earned a $0.5 million payment.
Kinnate In early 2025, XOMA Royalty secured license agreements with several parties for the five unpartnered Kinnate assets. Per the terms of the acquisition, a portion of any upfront payments received by XOMA Royalty will be distributed to the Kinnate CVR holders.
Subsequent Events

Partner
Event

Rezolute
Received Breakthrough Therapy Designation from FDA for ersodetug (RZ358) for the treatment of hypoglycemia due to congenital hyperinsulinism (cHI)2.

Announced the independent Data Monitoring Committee reviewed the safety data from eight infants ages 3 months to 1 year enrolled in the open-label portion of the sunRIZE Phase 3 study of ersodetug for the treatment of hypoglycemia due to cHI. Their conclusion was the safety profile was such that infants may now be enrolled in the double-blind, placebo-controlled study3.

Castle Creek
XOMA Royalty added a royalty interest in D-Fi (FCX-007), a Phase 3 asset being developed by Castle Creek Biosciences, to the portfolio. D-Fi is being studied in dystrophic epidermolysis bullosa (DEB), a rare progressive and debilitating skin disorder. D-Fi has been granted Orphan Drug Designation for the treatment of DEB, as well as Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations by the FDA.

XOMA Royalty contributed $5 million to Castle Creek Biosciences’ $75 million syndicated royalty financing transaction.

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Affitech Research AS XOMA Royalty paid $6 million in milestones to Affitech related to VABYSMO (faricimab-svoa) achieving specific sales thresholds. This was the final payment due to Affitech.
Anticipated 2025 Events of Note

Partner
Event

Rezolute
Completion of enrollment in sunRIZE Phase 3 clinical trial, which is investigating ersodetug in infants and children with cHI. Topline results are expected in the fourth quarter of 20252.

First patient dosed in Phase 3 registrational study for ersodetug for the treatment of hypoglycemia due to tumor hyperinsulinism4.

Gossamer / Chiesi
Presentation of topline results from the Phase 3 PROSERA study, a global registrational clinical trial in patients with WHO Function Class II and III pulmonary arterial hypertension (PAH).5

Initiation of a registrational Phase 3 study in pulmonary hypertension associated with interstitial lung disease (PH-ILD) in 2025.1

Takeda First patient dosed in Takeda’s Phase 3 clinical trial investigating mezagitamab as a treatment for adults with chronic primary immune thrombocytopenia (ITP).
Daré Bioscience Commencement of one of two registrational Phase 3 clinical trials investigating Sildenafil Cream, 3.6%, for the treatment of female sexual arousal disorder6.
Fourth Quarter and Full Year 2024 Financial Results

Tom Burns, Chief Financial Officer of XOMA Royalty, commented, "Based upon the anticipated incoming cash payments from royalties alone, we have line of sight on becoming cash flow positive on a consistent basis. The transient expenses associated with the Kinnate and Pulmokine acquisitions that impacted our 2024 financial results are coming to a close. We expect our R&D and G&A expenses to normalize in the second half of 2025."

Income and Revenue: XOMA Royalty recorded total income and revenues of $8.7 million and $28.5 million for the fourth quarter and full year of 2024, respectively. In 2023, XOMA Royalty recorded total income and revenues of $1.8 million and $4.8 million for the fourth quarter and full year, respectively. The increase for the full year of 2024 was primarily driven by an increase in our income from purchased receivables.

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Research and Development (R&D) Expenses: R&D expenses were $0.9 million and $2.9 million in the fourth quarter and full year of 2024, respectively. R&D expenses in the fourth quarter and full year of 2023 were $25,000 and $0.1 million, respectively. The increase of $2.8 million for the full year of 2024 is due to clinical trial costs related to KIN-3248 that were incurred subsequent to XOMA Royalty’s acquisition of Kinnate in April 2024. The Company currently is winding down this trial.

General and Administrative (G&A) Expenses: G&A expenses were $7.0 million and $34.5 million for the fourth quarter and full year of 2024, respectively, compared with $7.3 million in the fourth quarter and $25.6 million for the full year of 2023. The increase of $8.9 million for the full year of 2024 was primarily due to $7.4 million in costs associated with the acquisition of Kinnate, which primarily included $3.6 million in severance costs, $2.9 million in legal and consulting costs, $0.4 million in information technology costs, and $0.3 million in insurance costs. In addition, stock-based compensation expenses increased in 2024 by $1.2 million primarily due to the performance stock unit (PSU) grant awarded to Mr. Hughes in connection with his appointment as full-time CEO in January 2024.

In the fourth quarter and full year of 2024, G&A expenses included $2.2 million and $10.3 million, respectively, of non-cash stock-based compensation expenses. In the fourth quarter and full year of 2023, G&A expenses included $2.6 million and $9.1 million, respectively, of non-cash stock-based compensation expenses.

Credit Losses on Royalty and Commercial Payment Receivables (credit losses): In the fourth quarter of 2024, credit losses were $7.9 million related to the 2024 Talphera transaction. For the year ended December 31, 2024, credit losses totaled $30.9 million, consisting of $14.0 million related to the 2018 Agenus transaction, $9.0 million related to the 2019 Aronora transaction, and $7.9 million related to the Talphera transaction. For the year ended December 31, 2023, credit losses were $1.6 million related to the 2019 Bioasis transaction. There were no credit losses in the fourth quarter of 2023.

Interest Expense: Interest expense was $3.4 million and $13.8 million for the fourth quarter and full year of 2024, respectively. Interest expense in the fourth quarter and full year of 2023 was $0.6 million. Interest expense relates to the Blue Owl Loan established in December 2023.

Other Non-Comparable Transactions: Transactions for which there were no comparable period-over-period transactions include the following: In 2023, arbitration settlement costs of $4.1 million were paid in relation to a proceeding with one of XOMA Royalty’s licensees and a $14.2 million non-cash impairment charge was recorded in relation to the intangible ObsEva asset. In 2024, the Company recognized a gain on the acquisition of Kinnate of $19.3 million and an $8.1 million change in fair value of embedded derivative related to the Viracta transaction.

Other Income, net: The Company reported other income, net, of $1.0 million and $6.9 million for the fourth quarter and full year of 2024, as compared to $0.4 million and $1.6 million in the corresponding periods of 2023. The $5.3 million increase during the full year of 2024 was primarily driven by a $4.8 million increase in investment income due to higher balances on XOMA Royalty’s investments.

Net Loss: Net loss for the fourth quarter and full year ended December 31, 2024, was $4.0 million and $13.8 million, respectively, primarily resulting from the $30.9 million in non-cash credit losses on purchased receivables. Net loss for the fourth quarter and full year ended December 31, 2023, was $20.1 million and $40.8 million, respectively, which included $15.8 million in non-cash credit losses and impairment charges.

On December 31, 2024, XOMA Royalty had cash and cash equivalents of $106.4 million (including $4.8 million in restricted cash). On December 31, 2023, XOMA Royalty had cash and cash equivalents of $159.6 million (including $6.3 million in restricted cash). In 2024, XOMA Royalty received $46.3 million in cash receipts including $20.0 million in royalties and commercial payments, $19.3 million in other receipts from purchased receivables, and $7.1 million from licensees. In addition, as of December 31, 2024, the Company netted approximately $7.8 million from its acquisition of Kinnate. In 2024, XOMA Royalty deployed $65 million to acquire new milestone and royalty assets and paid $5.5 million in dividends on the XOMA Royalty Perpetual Preferred stocks.