On February 27, 2025 Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) reported financial results for the three and twelve months ended December 31, 2024 and provided an update on recent corporate progress (Press release, Aurinia Pharmaceuticals, FEB 27, 2025, View Source [SID1234650688]).

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Fourth Quarter 2024 Financial Results

Total Revenue: For the three months ended December 31, 2024, total revenue was $59.9 million, up 33% from $45.1 million in the same period of 2023.
– Net Product Sales: For the three months ended December 31, 2024, net product sales of LUPKYNIS, the first FDA-approved oral therapy for the treatment of adult patients with active lupus nephritis, were $57.6 million, up 36% from $42.3 million in the same period of 2023.

– License, Collaboration and Royalty Revenue: For the three months ended December 31, 2024, license, collaboration and royalty revenue, which includes manufacturing services revenue and royalties from Aurinia’s collaboration partner, Otsuka, was $2.3 million, down 18% from $2.8 million in the same period of 2023.

Net Income (Loss): For the three months ended December 31, 2024, net income (loss) was $1.4 million, compared to $(26.9) million in the same period of 2023.
Cash Flow Provided by Operating Activities: For the three months ended December 31, 2024, cash flow provided by operating activities was $30.1 million, up 110% from $14.3 million in the same period of 2023.
Full Year 2024 Financial Results

Total Revenue: For the twelve months ended December 31, 2024, total revenue was $235.1 million, up 34% from $175.5 million in 2023.
– Net Product Sales: For the twelve months ended December 31, 2024, net product sales were $216.2 million, up 36% from $158.5 million in 2023.

– License, Collaboration and Royalty Revenue: For the twelve months ended December 31, 2024, license, collaboration and royalty revenue, which includes a milestone payment, manufacturing services revenue and royalties from Otsuka, was $18.9 million, up 11% from $17.0 million in 2023.

Net Income (Loss): For the twelve months ended December 31, 2024, net income (loss) was $5.8 million, compared to $(78.0) million in 2023.
Cash Flow Provided by (Used in) Operating Activities: For the twelve months ended December 31, 2024, cash flow provided by (used in) operating activities was $44.4 million, compared to $(33.5) million in 2023.
Cash Position

As of December 31, 2024, Aurinia had cash, cash equivalents, restricted cash and investments of $358.5 million, compared to $350.7 million at December 31, 2023. For the year ended December 31, 2024, the Company repurchased 6.1 million of its common shares for $41.0 million.

Full Year 2025 Total Revenue and Net Product Sales Guidance

For 2025, Aurinia expects total revenue in the range of $250 million to $260 million and net product sales in the range of $240 million to $250 million.

Research and Development Update

In September 2024, Aurinia initiated a Phase 1 study of AUR200, its potentially best-in-class dual inhibitor of B cell activating factor (BAFF) and a proliferation inducing ligand (APRIL). BAFF and APRIL are cytokines that stimulate B cell proliferation and activity and are upregulated in many autoimmune diseases. In preclinical studies, AUR200 potently inhibited B cell proliferation and production of IgA and IgM antibodies and exhibited pharmacokinetic and pharmacodynamic properties consistent with once-monthly dosing. Aurinia expects to report initial results from its Phase 1 study of AUR200 in the second quarter of 2025.

"We are pleased to have delivered strong LUPKYNIS sales growth in 2024," stated Peter Greenleaf, President and Chief Executive Officer of Aurinia. "We expect 2025 to be an exciting year for Aurinia. We remain focused on increasing LUPKYNIS’s adoption among the many lupus nephritis patients who could benefit from this important treatment, while, at the same time, advancing our important pipeline product, AUR200, which has the potential to treat a wide range of autoimmune diseases."

Webcast & Conference Call Details

A webcast and conference call will be hosted today, February 27th, at 8:30 a.m. ET. The link to the audio webcast is available here. To join the conference call, please dial 877-407-9170/+1 201-493-6756. A replay of the webcast will be available on Aurinia’s website.

On February 27, 2025 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2024 (Press release, Arcellx, FEB 27, 2025, View Source [SID1234650687]).

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"2024 was a transformational year for Arcellx as our ASH (Free ASH Whitepaper) data presentations for anito-cel, in partnership with Kite, continued to demonstrate anito-cel’s differentiated clinical profile for the potential treatment of patients with relapsed or refractory multiple myeloma," said Rami Elghandour, Arcellx’s Chairman and Chief Executive Officer. "In our Phase 1 and iMMagine-1 studies, data demonstrate that anito-cel has the potential to provide a meaningful benefit to a broad population of patients. Our Phase 1 study demonstrated that the deep responses observed with anito-cel also translated to durable benefit for patients with a 30.2 month median progression-free survival in a challenging patient cohort and the preliminary data from our pivotal iMMagine-1 study demonstrated anito-cel also delivered deep responses for late line myeloma patients with a 97% overall response rate and 62% complete response rate at a median follow-up of 9.5 months. Most notably, as presented during ASH (Free ASH Whitepaper), the safety profile for anito-cel continues to be manageable with no delayed neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome in the155 patients dosed across the Phase 1 and iMMagine-1 studies. Physician engagement and enthusiasm for anito-cel continues to build following these data presentations and as more sites gain experience with anito-cel through iMMagine-3, our earlier-line study. Kite is manufacturing for iMMagine-3, and turnaround times are consistent with Kite commercial products. This year, we look forward to presenting updated data from our iMMagine-1 study mid-year, preparing for the commercial launch of anito-cel in multiple myeloma in 2026, enrolling our program in generalized myasthenia gravis, and expanding our ARC-SparX program in acute myeloid leukemia to include an additional SparX antigen target. Thank you to our Arcellx team who are willing to embrace challenges and think creatively to make an impact for the patients we serve."

Recent Business Progress

Presented positive preliminary data for the Phase 2 pivotal iMMagine-1 study of anito-cel in patients with relapsed or refractory multiple myeloma (RRMM) at the 66th ASH (Free ASH Whitepaper) Annual Meeting and Exposition. The data presented on December 9, 2024 demonstrated deep and durable responses with a predictable and manageable safety profile in a high-risk fourth-line or higher (4L+) RRMM population, including triple- and penta-class refractory disease. The data were from an October 31, 2024 data cutoff date, with a median follow-up of 9.5 months for the 86 patients who were evaluable for efficacy based on a follow-up of at least two months after treatment with anito-cel, and 98 patients were evaluable for safety based on a follow-up of at least one month after treatment with anito-cel.

Overall response rate was 97% (83/86) with a complete response/stringent complete response rate of 62% (53/86) and a very good partial response or higher rate of 81% (70/86), per International Myeloma Working Group (IMWG) criteria as investigator-assessed. Of those evaluable for minimal residual disease (MRD) testing, 93.1% (54/58) achieved MRD negativity at a minimum of 10 -5 sensitivity. Median progression-free survival (mPFS) and overall survival (OS) were not reached; 6-month PFS and OS rates were 93.3% and 96.5%, respectively, and 12-month PFS and OS rates were 78.5% and 96.5%, respectively. No delayed or non-ICANS neurotoxicities, including no Parkinsonism, no cranial nerve palsies, and no Guillain-Barré syndrome, were observed in the 155 patients dosed with anito-cel.

Fourth Quarter and Full Year 2024 Financial Highlights

Cash, cash equivalents, and marketable securities:

As of December 31, 2024, Arcellx had cash, cash equivalents, and marketable securities of $625.7 million. Arcellx anticipates that its cash, cash equivalents, and marketable securities will fund its operations into 2027.

Collaboration revenue:

Collaboration revenue were $15.3 million and $63.1 million for the quarters ended December 31, 2024 and 2023, respectively, a decrease of $47.8 million. This decrease was primarily driven by the December 2023 expansion to the license and collaboration agreement with Kite Pharma, Inc. Collaboration revenue were $107.9 million and $110.3 million for the twelve months ended December 31, 2024 and 2023, respectively, a decrease of $2.4 million.

R&D expenses:

Research and development expenses were $44.6 million and $28.8 million for the quarters ended December 31, 2024 and 2023, respectively, an increase of $15.8 million. Research and development expenses were $157.1 million and $133.8 million for the twelve months ended December 31, 2024 and 2023, respectively, an increase of $23.3 million. The increases were primarily driven by increased personnel costs, which include non-cash stock-based compensation expense, and increased costs relating to anito-cel and other pipeline programs.

G&A expenses:

General and administrative expenses were $23.8 million and $19.4 million for the quarters ended December 31, 2024 and 2023, respectively, an increase of $4.4 million. General and administrative expenses were $88.4 million and $66.4 million for the twelve months ended December 31, 2024 and 2023, respectively, an increase of $22.0 million. The increases were primarily driven by increased personnel costs, which include non-cash stock-based compensation expense, increased costs relating to commercial readiness, and increased depreciation expense.

Net losses:

Net losses were $47.1 million and $19.8 million for the quarters ended December 31, 2024 and 2023, respectively. Net losses were $107.3 million and $70.7 million for the twelve months ended December 31, 2024 and 2023, respectively.

About Multiple Myeloma

Multiple Myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. Multiple myeloma is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple co-morbidities and toxicities that can quickly escalate and become life-endangering.

About anitocabtagene autoleucel (anito-cel)

Anitocabtagene autoleucel (anito-cel, previously CART-ddBCMA) is the first BCMA-directed CAR T-cell therapy to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact binder known as the D-Domain. The small, stable D-Domain binder enables high CAR expression without tonic signaling and is designed to quickly release from the BCMA target. This combination may allow for the effective elimination of multiple myeloma cells without severe immunotoxicity. Anito-cel has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.

On February 27, 2025 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, reported that Daniel Faga, president and chief executive officer of Anaptys, and/or other members of its executive leadership team, are scheduled to participate in multiple upcoming investor conferences (Press release, AnaptysBio, FEB 27, 2025, View Source [SID1234650686]):

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TD Cowen 45th Annual Healthcare Conference, Boston, MA

Format – Fireside chat and one-on-one investor meetings
Date and Time – Tuesday, Mar. 4, 2025 at 1:10pm ET / 10:10am PT
Leerink Partners 2025 Global Healthcare Conference, Miami, FL

Format – Fireside chat and one-on-one investor meetings
Date and Time – Tuesday, Mar. 11, 2025 at 10:40am ET / 7:40am PT
Barclays 27th Annual Global Healthcare Conference, Miami, FL

Format – Presentation and one-on-one investor meetings
Date and Time – Wednesday, Mar. 12, 2025 at 8:30am ET / 5:30am PT
Live webcasts of the fireside chats and presentation will be available on the investor section of the Anaptys website at View Source Replays of the webcasts will be available for at least 30 days following the events.

On February 27, 2025 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the fourth quarter and full year ended 2024 and provided a corporate update (Press release, Aclaris Therapeutics, FEB 27, 2025, View Source [SID1234650685]).

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"2024 was a transformative year that has positioned Aclaris with multiple clinical catalysts expected in 2025 across our expanded pipeline," stated Dr. Neal Walker, Chief Executive Officer and Chair of the Board of Directors of Aclaris. "We are particularly excited about the upcoming Phase 2 data for bosakitug in both severe asthma and chronic rhinosinusitis with nasal polyps anticipated from our partner CTTQ, which we expect will provide important insights into our future development of bosakitug in respiratory diseases. We also anticipate top-line data from our Phase 2a trial of ATI-2138 in atopic dermatitis in the first half of 2025. With multiple clinical catalysts expected throughout 2025 across our pipeline of differentiated assets with mechanisms shown to have proven activity in the diseases we are addressing, we look to drive continued innovation for the patients we seek to treat."

Fourth Quarter 2024 Highlights and Recent Updates

Pipeline:

Announced Exclusive, Global License Agreement with Biosion, Inc., Adding Potential Best-in-Class Biologic Assets to Pipeline: Aclaris acquired worldwide rights (excluding Greater China) to bosakitug (ATI-045), a potential best-in-class, clinical-stage, novel anti-TSLP monoclonal antibody, and ATI-052, a potential best-in-class, pre-clinical stage, novel bispecific antibody that is directed against both TSLP and IL4R. As a result of this license agreement, the Company recorded a one-time $86.9 million in-process research and development charge. (press release here)
Confirmed Expectation of Phase 2 Data in the First Half of 2025 for Bosakitug in Chinese Patients with Certain Pulmonary Disorders: Aclaris’ regional partner, Chia Tai Tianqing Pharmaceutical Group, Co., Ltd. (CTTQ), is conducting concurrent Phase 2 studies in China for patients with severe asthma, CRSwNP, and chronic obstructive pulmonary disease. Data from trials in severe asthma and CRSwNP expected in first half of 2025 to inform internal development programs.
Initiated Clinical Trial Activities for a Phase 2b Trial of Bosakitug in Atopic Dermatitis (AD); Enrollment Expected to Begin in the First Half of 2025: This trial will investigate the safety, tolerability, pharmacokinetics, efficacy, and pharmacodynamics of bosakitug in patients with moderate to severe AD.
Confirmed Expectation of Top Line Results in the First Half of 2025 for Phase 2a Trial in AD of ATI-2138, an Investigational Oral Covalent ITK/JAK3 Inhibitor: This ongoing Phase 2a open-label trial is being conducted to investigate the safety, tolerability, pharmacokinetics, efficacy, and pharmacodynamics of ATI-2138 in patients with moderate to severe AD.
Announced Plan to File an Investigational New Drug (IND) Application for ATI-052 in the First Quarter of 2025: Following allowance of the IND, Aclaris expects to initiate a Phase 1 clinical trial evaluating single ascending doses and multiple ascending doses of ATI-052.
Announced New Publication Highlighting the Unique Properties of ATI-2138: New publication provides important clinical and non-clinical evidence of the potential for ATI-2138 to be a best-in-class inhibitor of key signal transduction kinases due to its unique mechanism of action. (press release here)
Corporate:

Completed $80 Million Private Placement in November 2024 to Bolster Cash Runway: Aclaris’ cash runway expected into 2028. (press release here)
Provided Update on Senior Leadership:
Dr. Neal Walker, formerly interim Chief Executive Officer, has been named Chief Executive Officer. Dr. Walker is a co-founder of Aclaris and has served as a member of the Board of Directors since its inception. He previously served as Aclaris’ Chief Executive Officer until 2022 before being appointed as interim Chief Executive Officer in January 2024. Dr. Walker serves as Chair of the Board of Directors of Aclaris.
Hugh Davis, Ph.D. joined Aclaris as President and Chief Operating Officer. Dr. Davis brings over 35 years of experience in biologics development, clinical pharmacology, and business development to Aclaris. He most recently served as Biosion’s Chief Business & Development Officer and President.
William Roberts has been appointed as Senior Vice President, Corporate Communications and Investor Relations. Mr. Roberts brings 30 years of corporate communications, investor relations, and scientific experience in the biotech/biopharma industry to the Company. He most recently served as the Communications Officer of G1 Therapeutics, which was recently acquired by Pharmacosmos Group.
Fourth Quarter and Full Year 2024 Financial Results

As of December 31, 2024, Aclaris had aggregate cash, cash equivalents and marketable securities of $203.9 million compared to $181.9 million as of December 31, 2023. The Company believes that its cash, cash equivalents and marketable securities as of December 31, 2024 will be sufficient to fund its operations into 2028, without giving effect to any potential business development transactions or financing activities.

Net loss was $96.6 million for the fourth quarter of 2024 compared to $1.5 million for the fourth quarter of 2023. Net loss was $132.1 million for the year ended December 31, 2024 compared to $88.5 million for the year ended December 31, 2023.

Total revenue was $9.2 million for the fourth quarter of 2024 compared to $17.6 million for the fourth quarter of 2023. The decrease was primarily driven by a one-time upfront payment under the license agreement with Sun Pharmaceutical Industries, Inc. received in the fourth quarter of 2023, offset by the achievement of a commercial milestone under the license agreement with Eli Lilly and Company in the fourth quarter of 2024. Total revenue was $18.7 million for the year ended December 31, 2024 compared to $31.2 million for the year ended December 31, 2023.

Research and development (R&D) expenses were $9.0 million for the quarter ended December 31, 2024 compared to $26.6 million for the prior year period. The $17.6 million decrease was primarily the result of lower zunsemetinib development expenses, lepzacitinib preclinical and clinical development activities, and compensation-related expenses. For the year ended December 31, 2024, R&D expenses were $33.6 million compared to $98.4 million for the year ended December 31, 2023.

General and administrative (G&A) expenses were $5.0 million for the quarter ended December 31, 2024 compared to $8.2 million for the corresponding prior year period. The decrease was primarily due to a reduction in personnel and stock-based compensation expenses. For the year ended December 31, 2024, G&A expenses were $22.2 compared to $32.4 million for the year ended December 31, 2023, primarily due to lower compensation-related costs.

Licensing expenses were $8.6 million for the quarter ended December 31, 2024 compared to $5.7 million for the prior year period. The increase was primarily attributable to a milestone achieved during the fourth quarter of 2024, the entirety of which was payable to a third party. For the year ended December 31, 2024, licensing expenses were $12.7 million compared to $14.7 million for the year ended December 31, 2023.

Revaluation of contingent consideration resulted in a $1.3 million gain for the quarter ended December 31, 2024 compared to a $26.3 million gain for the prior year period. For the year ended December 31, 2024, revaluation of contingent consideration resulted in a charge of $2.5 million compared to a $26.9 million gain for the year ended December 31, 2023.

During the quarter and year ended December 31, 2024, the Company recorded $86.9 million of in-process research and development expenses, representing the fair value of consideration expensed in connection with the in-license of bosakitug (ATI-045) and ATI-052, as well as transaction costs incurred. During the quarter ended December 31, 2023, the Company recorded an intangible asset impairment charge of $6.6 million representing the full balance of its in-process research and development intangible asset.

On February 26, 2025 Starpharma (ASX: SPL, US OTC: SPHRY), an innovative biotechnology company with two decades of experience in advancing dendrimer technology from the lab to the patient, reported its Interim Report and Half-Year Financial Results for the period ended 31 December 2024 (H1 FY25) (Press release, Starpharma, FEB 26, 2025, View Source [SID1234650765]).

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Starpharma’s Chief Executive Officer, Cheryl Maley, commented:
"In the first half of FY25, Starpharma has made good progress in executing our strategy, aimed at maximising the value of our DEP assets, accelerating early asset development, and building longterm sustainability. I know our internal progress may not always be evident externally, but I can vouch for the dedication and hard work of everyone at Starpharma as we work towards achieving our strategic objectives.

"We have made important progress in advancing our DEP clinical assets, particularly DEP SN38, which has shown promising clinical outcomes in patients with high unmet need. A key regulatory milestone during the period was the recent meeting with the US Food and Drug Administration (FDA) regarding DEP SN38. The FDA provided feedback on the path to market for DEP SN38, confirming that the 505(b)(2) regulatory approval pathway is appropriate for DEP SN38 and the potential for Fast Track designation and accelerated approval. The positive response from the US regulator increases our confidence in the potential of DEP SN38 for treating platinum-resistant ovarian cancer. During the six months to December, our partner engagement through ongoing meetings and conference participation highlighted the importance of FDA feedback to potential partners for the commercialisation of DEP SN38.