On May 3, 2024 Abeona Therapeutics Inc. (Nasdaq: ABEO) ("Abeona" or the "Company") reported the pricing of an underwritten offering of 12,285,056 shares of its common stock and, in lieu of common stock, pre-funded warrants to purchase 6,142,656 shares of its common stock, at an offering price of $4.07 per share, which is equal to the closing price on Thursday, May 2, 2024, or $4.0699 per pre-funded warrant, which represents the per share offering price for the common stock less the $0.0001 per share exercise price for each pre-funded warrant (Press release, Abeona Therapeutics, MAY 3, 2024, View Source [SID1234642623]). The pre-funded warrants will be immediately exercisable at a nominal exercise price of $0.0001 per share and may be exercised at any time until the pre-funded warrants are exercised in full. The closing of the offering is expected to occur on or about May 7, 2024, subject to the satisfaction of customary closing conditions.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The offering included participation from both new and existing investors, including Adage Capital Partners, L.P., Janus Henderson Investors, Nantahala Capital, Suvretta Capital, Vivo Capital, and other healthcare-dedicated investors.

Stifel is acting as the sole bookrunner for the offering.

The gross proceeds to Abeona from this offering are expected to be approximately $75 million, before deducting underwriting discounts and commissions and other offering expenses. Abeona intends to use the net proceeds from the offering primarily to fund preparations for resubmission of its BLA and for commercialization of its product candidate pz-cel, as well as for working capital and general corporate purposes.

The securities described above are being offered pursuant to a shelf registration statement on Form S-3 (File No. 333-256850) that was filed with the Securities and Exchange Commission (the "SEC") on June 7, 2021 and amended on August 27, 2021 and October 19, 2021, and was declared effective by the SEC on October 22, 2021. When available, the prospectus supplement and the accompanying prospectus that form a part of the registration statement will be filed with the SEC and available on the SEC’s website at www.sec.gov. Copies of the prospectus supplement and the accompanying prospectus may also be obtained when available by contacting Stifel, Nicolaus & Company, Incorporated, Attention: Prospectus Department, One Montgomery Street, Suite 3700, San Francisco, CA 94104, by telephone at (415) 364-2720 or by email at [email protected].

The securities described above have not been qualified under any state blue sky laws. This press release does not constitute an offer to sell or the solicitation of offers to buy any securities of Abeona being offered, and shall not constitute an offer, solicitation or sale of any security in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On May 3, 2024 Clarity Pharmaceuticals (ASX: CU6) ("Clarity", "the Company"), a clinical stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, reported that new data will be presented at the upcoming AUA, ASCO (Free ASCO Whitepaper) and SNMMI 2024 Annual Meetings, covering clinical results and trials in progress (Press release, Clarity Pharmaceuticals, MAY 3, 2024, View Source [SID1234642582]). The data showcases the extensive scope of Clarity’s Targeted Copper Theranostic (TCT) platform in developing multiple products for imaging and treating cancer patients, with focus on prostate cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "The AUA, ASCO (Free ASCO Whitepaper) and SNMMI Annual Meetings are among the most prestigious conferences in urology, oncology and nuclear medicine, respectively. We are delighted that our data has been accepted for presentation at these world leading conferences. The abstracts accepted for presentations, including two oral presentations, highlight the exciting opportunities for our next-generation TCT radiopharmaceuticals as we continue to generate incredible data on what we strongly believe to be best in class assets for the treatment and diagnosis of cancer.

"We are looking forward to sharing this data as we continue to adhere to the highest standards of clinical research, work with world-class clinical sites and generate exciting results to date while expanding the platform into multiple indications. We are also looking forward to further developing our existing as well as novel TCT products as we get closer to our ultimate goal of improving treatment outcomes for children and adults with cancer."

Data to be presented at 2024 AUA, ASCO (Free ASCO Whitepaper) and SNMMI Annual Meetings
Product Abstract Title Conference
64Cu-SAR-bisPSMA CLARIFY: Positron Emission Tomography using 64Cu-SAR-bisPSMA in patients with high-risk prostate cancer prior to radical prostatectomy, a phase 3 diagnostic study AUA – Oral presentation
Session Date
Sunday, May 5th

Session Time
1:00-3:00 PM CT

Session Title
Clinical Trials in Progress—Cancer

64Cu-SAR-bisPSMA COBRA: Assessment of safety and efficacy of 64Cu-SAR-bisPSMA in patients with biochemical recurrence of prostate cancer following definitive therapy ASCO – Poster presentation
Session Date
Sunday, June 2nd

Session Time
9:00 AM – 12:00 PM CT

Session Title/Track
Genitourinary Cancer—Prostate, Testicular, and Penile

Sub Track
Prostate Cancer–Local-Regional Disease

Abstract # 5100

Poster Bd # 506

64Cu-SAR-bisPSMA CLARIFY: Positron emission tomography using 64Cu-SAR-bisPSMA in patients with high-risk prostate cancer prior to radical prostatectomy (a phase 3 diagnostic study) ASCO – Poster presentation
Session Date
Sunday, June 2nd

Session Time
9:00 AM – 12:00 PM CT

Session Title/Track
Genitourinary Cancer—Prostate, Testicular, and Penile

Sub Track
Prostate Cancer–Local-Regional Disease

Abstract # TPS5129

Poster Bd # 524a

67Cu-SAR-bisPSMA SECuRE: A dose escalation/expansion study to assess the anti-tumor efficacy of 67Cu-SAR-bisPSMA in patients with metastatic castrate resistant prostate cancer

ASCO – Poster presentation
Session Date
Sunday, June 2nd

Session Time
9:00 AM – 12:00 PM CT

Session Title/Track
Genitourinary Cancer—Prostate, Testicular, and Penile

Sub Track
Prostate Cancer– Advanced/Castrate-Resistant

Abstract # TPS5116

Poster Bd # 517b

64Cu-SAR-bisPSMA COBRA: Assessment of safety and efficacy of 64Cu-SAR-bisPSMA in patients with biochemical recurrence of prostate cancer following definitive therapy SNMMI – Oral presentation
Session Date
Tuesday, June 11th

Session Time
8:00 AM – 9:15 AM ET

Session Title
Prostate Cancer – Radiomics/Data Analysis

67Cu-SAR-bisPSMA SECuRE: A dose escalation/expansion study to assess the anti-tumor efficacy of 67Cu-SAR-bisPSMA in patients with metastatic castrate resistant prostate cancer SNMMI – Poster presentation
67Cu-SAR-BBN COMBAT: A study of 64Cu-SAR-BBN and 67Cu-SAR-BBN for identification and treatment of GRPr-expressing metastatic castrate resistant prostate cancer ineligible for therapy with 177Lu-PSMA-617 SNMMI – Poster presentation
64Cu-SAR-BBN SABRE: Assessment of safety and efficacy of 64Cu-SAR-BBN in PSMA-negative biochemical recurrent prostate cancer patients SNMMI – Poster presentation
Posters and presentations containing study results will be available on Clarity’s official website after their presentation at the 2024 AUA, ASCO (Free ASCO Whitepaper) and SNMMI Annual Meetings: claritypharmaceuticals.com/pipeline/scientific_presentations

Abstracts for the 2024 ASCO (Free ASCO Whitepaper) Annual Meeting will be released on the 23rd of May at 5 PM ET and will be available at the conference website: conferences.asco.org/am/abstracts

On May 2, 2024 Moderna Therapeutics reported financial results and provided business updates for the first quarter of 2024 (Press release, Moderna Therapeutics, MAY 2, 2024, View Source/news/news-details/2024/Moderna-Reports-First-Quarter-2024-Financial-Results-and-Provides-Business-Updates/default.aspx" target="_blank" title="View Source/news/news-details/2024/Moderna-Reports-First-Quarter-2024-Financial-Results-and-Provides-Business-Updates/default.aspx" rel="nofollow">View Source [SID1234644823]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"As we anticipate the launches of our Spikevax 2024-2025 formula and RSV vaccine, we are exercising financial discipline and have intensified our focus on building and utilizing AI technologies to further streamline operations and enhance productivity," said Stéphane Bancel, Chief Executive Officer of Moderna. "With 10 late-stage programs, and additional new programs advancing toward pivotal studies, we continue to expect numerous product milestones this year across our vaccines and therapeutics portfolio. This is the start of a banner year for our vaccine platform as we continue to advance mRNA medicines for patients. This is just the beginning."

Recent progress includes:

Commercial Updates

COVID-19: The Company reported $167 million in Spikevax (COVID-19 vaccine) sales in the first quarter of 2024, which includes $100 million of U.S. sales and $67 million of international sales.

In the U.S., the Company is reaffirming its 2024 product sales outlook as it enters the second year of the commercial endemic COVID market. Moderna’s focus is on working with public health officials, health care providers and pharmacies to increase vaccination coverage rates to reduce the substantial burden of COVID-19. Moderna is also working with the U.S. FDA and regulators to align the timing of flu and COVID-19 vaccine approvals, which is expected to result in higher vaccination uptake if vaccines are available sooner and offered at the same time as the flu shot.

In the European Union, Moderna is participating in the EU Health Emergency and Response Authority’s tendering procedure for up to 36 million doses of mRNA COVID-19 vaccines per year for up to four years.

In the Rest of World, the Company is prioritizing markets for greater commercial focus and in April announced a contract with the Ministry of Health in Brazil (Ministério da Saúde) to supply 12.5 million mRNA COVID-19 vaccines as an integral part of Brazil’s 2024 national vaccination campaign against COVID-19.

RSV: The Company anticipates initial regulatory approvals of its RSV vaccine (mRNA-1345) beginning in the first half of 2024.

Moderna is targeting fall 2024 for its U.S. RSV vaccine launch, which will build upon the success of its commercial efforts in the fall COVID-19 market. The Company is encouraged by early indications of widespread consumer awareness and established demand in the RSV market, which Moderna will enter with a strong competitive profile with robust clinical efficacy data, a well-established safety and tolerability profile for its mRNA technology, and as the only pre-filled syringe (PFS) product available.

The PFS ready-to-use formulation will save pharmacists and clinicians time, potentially alleviating wait times and reducing the burden on pharmacy staff. In a study funded by Moderna, the PFS presentation was three to four times more efficient than vaccines requiring reconstitution, measured in doses per hour and based on mean time of preparation.

First Quarter 2024 Financial Results

Revenue: Total revenue for the first quarter of 2024 was $167 million, compared to $1.9 billion in the same period in 2023. The decline was primarily due to reduced sales of the Company’s COVID-19 vaccine. Net product sales for the first quarter of 2024 were $167 million, representing a 91% decline compared to the same period in 2023. This decline aligns with the anticipated transition to a seasonal COVID-19 vaccine market; in the prior year period, the Company recognized revenue primarily from delivered doses deferred from 2022.

Cost of Sales: Cost of sales for the first quarter of 2024 totaled $96 million, which included third-party royalties of $8 million, inventory write-downs of $30 million, and unutilized manufacturing capacity and winddown costs of $27 million. Cost of sales for the first quarter of 2024 decreased by $696 million, or 88%, compared to the same period in 2023. Cost of sales as a percentage of net product sales was 58% for the first quarter of 2024, compared to 43% for the first quarter of 2023. The decrease in cost of sales in 2024 was primarily driven by lower sales volume coupled with reduced unutilized manufacturing capacity, inventory write-downs and losses on firm purchase commitments and related cancellation fees. Conversely, the increase in cost of sales as a percentage of net product sales in 2024 was driven by the lower sales level compared to the prior year.

Research and Development Expenses: Research and development expenses for the first quarter of 2024 decreased by 6% to $1.1 billion, compared to the same quarter of 2023. This reduction was primarily due to the absence of upfront collaboration payments being made in 2024. The upfront payments made in the first quarter of 2023 were related to the Company’s strategic collaborations with Generation Bio and Life Edit Therapeutics. Additionally, there was a decrease in clinical development and manufacturing expenses, driven by lower spending on clinical trials for the Company’s COVID-19, RSV and seasonal flu programs, which aligns with its planned trial schedules. These decreases were partially offset by higher personnel-related costs, resulting from an increased headcount in the research and development functions.

Selling, General and Administrative Expenses: Selling, general and administrative expenses for the first quarter of 2024 decreased by 10% to $274 million, compared to the first quarter of 2023. This decrease is a result of cost discipline and the efficiencies resulting from investments the Company made in foundational capabilities over the last year, which allowed for a significant reduction of purchased services and the use of external consultants. The Company continues to invest in digital and commercial capabilities and has intensified its focus on building and utilizing AI technologies to further streamline operations and enhance productivity.

Income Taxes: Income tax expense for the first quarter of 2024 was $10 million, in contrast to an income tax benefit of $384 million in the same period last year. The shift primarily resulted from the continued application of a valuation allowance on the majority of the Company’s deferred tax assets, first established in the third quarter of 2023. The Company only maintained a valuation allowance on certain state deferred tax assets prior to the third quarter of 2023.

Net Income (Loss): Net loss was $(1.2) billion for the first quarter of 2024, compared to net income of $79 million for the first quarter of 2023.

Earnings (Loss) Per Share: Diluted loss per share was $(3.07) for the first quarter of 2024, compared to diluted earnings per share of $0.19 for the first quarter of 2023.

Cash Position: Cash, cash equivalents and investments as of March 31, 2024, were $12.2 billion, compared to $13.3 billion as of December 31, 2023. The decrease in the Company’s cash position during the first quarter of 2024 was largely attributable to research and development expenses and operating activities.

2024 Financial Framework

Expectations for full year 2024 remain consistent with prior expectations.

Revenue: The Company reaffirms its 2024 expected revenue of approximately $4 billion from its respiratory franchise, and now expects approximately $0.3 billion in net sales in the first half of the year, reflecting the seasonality of the respiratory business.

Cost of Sales: Cost of sales is expected to be approximately 35% of product sales for the year.

Research and Development Expenses: Full-year 2024 research and development expenses are anticipated to be approximately $4.5 billion.

Selling, General and Administrative Expenses: Selling, general and administrative expenses for 2024 are projected to be approximately $1.3 billion.

Income Taxes: The Company continues to expect its full-year tax expense to be negligible.

Capital Expenditures: Capital expenditures for 2024 are expected to be approximately $0.9 billion.

Cash and Investments: Year-end cash and investments for 2024 are projected to be approximately $9 billion.

Recent Progress and Upcoming Late-Stage Pipeline Milestones

With 10 late-stage programs, and additional new programs advancing toward pivotal studies, Moderna continues to advance its pipeline and expects numerous product milestones in 2024 across its vaccines and therapeutics portfolio. Late-stage includes eight programs that have progressed to Phase 3 clinical studies and two rare disease programs that are moving toward registrational studies.

Respiratory vaccines:

Respiratory syncytial virus (RSV) vaccine: Moderna’s RSV vaccine candidate (mRNA-1345) is in an ongoing Phase 2/3 clinical trial, ConquerRSV. Based on positive data from the study, Moderna has filed for regulatory approvals for the prevention of RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD) in adults over 60 years of age. As mentioned above, the Company is awaiting regulatory approvals and the U.S. ACIP recommendation in 2024.

At its Vaccines Day event in March, Moderna announced the initiation of multiple Phase 3 expansion studies of its RSV vaccine in adults over 50 years of age to evaluate co-administration and revaccination. Additional trials (Phase 1 – Phase 3) have been initiated for high-risk adults, as well as maternal and pediatric populations. Interim data from these studies could be available as early as 2024.
Seasonal flu vaccine: Moderna’s seasonal flu vaccine (mRNA-1010) demonstrated consistently acceptable safety and tolerability across three Phase 3 trials. In the most recent Phase 3 trial (P303), mRNA-1010 met all immunogenicity endpoints, demonstrating higher titers compared to a currently licensed standard-dose flu vaccine. In an older adult extension study of P303, mRNA-1010 is being studied against high dose Fluzone HD; the trial is fully enrolled. The Company is in ongoing discussions with regulators and intends to file in 2024.
Next-generation COVID-19 vaccine: A recent announcement of positive interim results from the NEXTCove Phase 3 trial showed that Moderna’s next-generation COVID-19 vaccine (mRNA-1283) elicited a higher immune response against both the Omicron BA.4/BA.5 and original virus strains of SARS-CoV-2 compared to Moderna’s licensed COVID-19 vaccine (mRNA-1273.222). The next-generation vaccine is designed to be refrigerator-stable and paves the way for a combination vaccine against influenza and COVID-19 (mRNA-1083). This is Moderna’s fourth infectious disease vaccine program with positive Phase 3 data. The Company is engaging with regulators on next steps.
Seasonal flu + COVID vaccine: Moderna’s Phase 3 trial of its combination vaccine against seasonal flu and COVID-19 (mRNA-1083) is fully enrolled. The Company anticipates data from the study in 2024.
Latent and other vaccines:

Cytomegalovirus (CMV) vaccine: The pivotal Phase 3 study of Moderna’s CMV vaccine candidate (mRNA-1647) is fully enrolled and accruing cases, evaluating its efficacy, safety and immunogenicity in the prevention of primary infection in women of childbearing age. The first interim analysis for the evaluation of vaccine efficacy is expected as early as the end of 2024.
Epstein-Barr virus (EBV) vaccine: Moderna’s EBV vaccine candidate for the prevention of infectious mononucleosis (mRNA-1189) showed positive immunogenicity and safety data in a Phase 1 study. The Company is advancing this candidate toward pivotal development.
Varicella-Zoster virus (VZV) vaccine: In a Phase 1/2 trial, Moderna’s VZV vaccine candidate for the prevention of shingles (mRNA-1468) elicited comparable or higher T cell responses relative to Shingrix. The Company is advancing toward a pivotal Phase 3 trial.
Herpes simplex virus (HSV) vaccine: The Phase 1/2 study of Moderna’s HSV vaccine candidate (mRNA-1608) is fully enrolled.
Norovirus vaccine: Moderna’s vaccine candidate for the prevention of norovirus (mRNA-1403) showed positive immunogenicity and safety data in a Phase 1 study. The Company is advancing toward a pivotal Phase 3 trial.
Oncology therapeutics:

Individualized Neoantigen Therapy (INT): Moderna continues to demonstrate the potential clinical benefit of its INT program (mRNA-4157). In partnership with Merck, two Phase 3 trials in resected high-risk (stage III/IV) melanoma and completely resected stage II, IIIA or IIIB non-small cell lung cancer are ongoing.

Moderna and Merck have initiated three new randomized clinical studies in additional tumor types in 2024, including a Phase 2 adjuvant treatment in patients with renal cell carcinoma, or kidney cancer; a Phase 2 adjuvant treatment in patients with high-risk muscle-invasive bladder cancer; and a Phase 2/3 neoadjuvant and adjuvant treatment in patients with cutaneous squamous cell carcinoma, the second most common form of skin cancer.
Rare disease and other therapeutics:

Propionic acidemia (PA) & methylmalonic acidemia (MMA) therapeutics: Interim data for a first-in-human, Phase 1/2, open-label, dose optimization study and extension study, evaluating the safety and efficacy of an investigational mRNA therapy for PA (mRNA-3927), was published in Nature. The Company expects to advance its PA and MMA (mRNA-3705) programs into registrational studies in 2024.
PD-L1 therapeutic: Following a strategic review, and as a result of its decision to prioritize investments in other programs, Moderna is discontinuing development of its preclinical PD-L1 program (mRNA-6981), and is no longer evaluating other mRNA candidates in this area.
Moderna Corporate Updates

Announced the advancement of multiple vaccine programs to late-stage clinical trials at its fifth Vaccines Day Investor Event on March 27, 2024.
Announced a development and commercialization funding agreement on March 27, 2024, with Blackstone Life Sciences for up to $750 million to advance Moderna’s flu program.
Announced a new collaboration with OpenAI to advance mRNA medicine using generative AI.
Entered into a non-exclusive intellectual property licensing agreement with an upfront payment and low double-digit royalty on the net sales of a COVID-19 product from a pharmaceutical company in the territory of Japan.
Agreed with Metagenomi to terminate gene editing collaboration as Moderna continues to strategically prioritize its research and development investments.
The Moderna Annual Meeting of Shareholders will be held on May 6, 2024, at 8:00 a.m. ET.
Company Accolade

Moderna was named for the first time to the LinkedIn Top Companies list of the best workplaces to grow a career in the U.S.
Key 2024 Investor and Analyst Event Dates

ASCO Investor Event: June 3
R&D Day: September 12
Investor Call and Webcast Information

Moderna will host a live conference call and webcast at 8:00 a.m. ET on May 2, 2024. To access the live conference call via telephone, please register at the link below. Once registered, dial-in numbers and a unique pin number will be provided. A live webcast of the call will also be available under "Events and Presentations" in the Investors section of the Moderna website.

Telephone: https://register.vevent.com/register/BI4b70dc922b874480847b6928c1fb9579
Webcast: View Source
The archived webcast will be available on Moderna’s website approximately two hours after the conference call and will be available for one year following the call.

On May 2, 2024 Ingenium Therapeutics reported the FDA granted an orphan drug designation to IGNK001 for the treatment of patients with acute myeloid leukemia (AML) (Press release, Ingenium Therapeutics, MAY 2, 2024, View Source [SID1234643523]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are delighted to have received orphan drug designation for IGNK001 from the FDA, recognizing the potential of our innovative [natural killer (NK)] cell therapy technology," said Ko Jin-ok, chief executive officer of Ingenium Therapeutics, in a press release. "[IGNK001] has already received approval from the Korean Ministry of Food and Drug Safety for phase 2 clinical trials, and we are preparing to initiate clinical trials in the US targeting 80 subjects with the goal of completing the studies by December 2027."

IGNK001 is an allogeneic NK cell therapy.2 While traditional methods for NK cell therapy use feeder cells to cultivate small numbers of isolated NK cells, this manufacturing process for IGNK001 leads to memory NK cells with higher expression of activating receptors. The novel method expands NK cells through targeted differentiation of other immune cells. This feeder cell-free process yields large quantities of healthy NK cells while avoiding exhaustion.

In addition to memory NK cells that have higher expression of activating receptors, the IGNK001 manufacturing process results in higher granzyme B and perforin 1 production compared with normal NK cells. These cells have also shown to have increased production of IFN-γ.

The mass-produced, allogeneic NK cell therapy has already demonstrated potent anticancer effects with high purity and activity.1 Its safety and efficacy have also been evaluated in a number of clinical trials.

Most recently, a single-center, open-label, random comparison, phase 2b study (NCT02477787) evaluated haploidentical hematopoietic cell transplantation and subsequent donor NK cell infusion in patients with high-risk, refractory AML and myelodysplastic syndromes.3 Patients were required to be aged 19 years or older with a Karnofsky performance status of at least 70.

In the experimental arm (n = 40), patients were given donor-derived NK cell infusions twice on days 13 and 20 after haploidentical hematopoietic cell transplantation. On day 13, the donor NK cell infusion was given at a dose which ranged from 1 x 108/kg to 2 x 108/kg, and on day 20, the donor NK cell infusion was given at a cell dose of up to 5 x 108/kg. The control arm included 36 patients who underwent haploidentical hematopoietic cell transplantation alone.

Investigators assessed the primary end point of the number of patients who experienced AML progression or recurrence after hematopoietic cell transplantation. Secondary end points explored in the study included the number of patients who achieved engraftment after hematopoietic cell transplantation, the number of patients who developed acute graft-vs-host disease (GVHD) and chronic GVHD, the number of patients who experienced donor NK cell infusion-associated toxicity, and the number of patients who died after hematopoietic cell transplantation without AML progression.

Findings from the intention-to-treat analysis showed that there was lower 30-month cumulative incidence of disease progression among those in the NK group vs the control group. These rates were 35% vs 61%, respectively (subdistribution HR, 0.50; P = .040).4

Three months post hematopoietic cell transplantation, those who received NK cells had a 1.8- and 2.6-fold higher median absolute blood count of NK cells and T cells compared with those who received hematopoietic cell transplantation alone. Looking at a single-cell RNA sequencing analysis in 7 patients, findings showed there to be an increase in memory-like NK cells among those given the NK cell infusion. This in turn expanded the CD8-positive effector memory T cells.

These findings, which led to IGNK001’s approval from the Korean Ministry of Food and Drug Safety for phase 2 clinical trials, now also are leading to the initiation of a sponsored clinical trial to support the conditional marketing approval of IGNK001.

On May 2, 2024 Galapagos NV (Euronext & NASDAQ: GLPG) reported its first quarter 2024 financial results and provided a business update and outlook for the remainder of 2024 (Press release, Galapagos, MAY 2, 2024, View Source [SID1234642637]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are moving forward with a renewed focus, a differentiated and expanding R&D pipeline, and competitive technology platforms to bring innovation to patients around the world. We continue to build a strong team of global leaders and we expanded our U.S. footprint," said Dr. Paul Stoffels1, CEO and Chairman of the Board of Directors of Galapagos. "As we look forward to the year ahead, we strive to make important progress in our clinical programs, achieving regulatory milestones to initiate clinical development of our CAR-T programs in the U.S., and expanding our decentralized CAR-T network in the U.S. and Europe. In parallel, we will continue to build our early-stage pipeline of cell therapy and small molecule investigational medicines in oncology and immunology, both internally and through external partnerships."

Thad Huston, CFO and COO of Galapagos, concluded: "We are committed to investing in our internal R&D pipeline, while we continue to actively pursue business development opportunities. We are broadening our oncology and immunology portfolio and will capitalize on opportunities that are aligned with our strategic objectives."

First quarter 2024 and recent business update

At the Annual and Extraordinary Shareholders’ Meetings held on 30 April 2024, all proposed resolutions were approved (see: press release of 30 April 2024).
Presented our innovative decentralized CAR-T manufacturing and 7-day vein-to-vein approach to hematological cancer care at the EBMT-EHA and EBMT annual meetings with new preliminary translational and previously published encouraging clinical data of our CD19 CAR-T candidates. The data support the promise of GLPG5101 in relapsed/refractory non-Hodgkin lymphoma (NHL) and GLPG5201 in relapsed/refractory chronic lymphocytic leukemia (CLL) and Richter transformation (RT), in addressing the critical needs of patients facing poor prognosis.
Discontinued the development of CD19 CAR-T candidate in refractory systemic lupus erythematosus.
Transferred the Jyseleca business to Alfasigma per 31 January 2024.
Signed a strategic collaboration and license agreement with BridGene Biosciences to further strengthen our growing early-stage oncology precision medicine pipeline.
Entered into a strategic collaboration agreement with Thermo Fisher Scientific for decentralized CAR-T manufacturing in the San Francisco area.
Participated with an investment of $40.0 million in the Series C financing round of precision oncology pioneer Frontier Medicines.
Financial performance
First quarter 2024 key figures (consolidated)
(€ millions, except basic & diluted income per share)

Three months ended 31 March % Change

2024 2023
Total net revenues 62.4 58.6 +7%
Cost of sales (2.5) –
R&D expenses (71.6) (52.5) +36%
G&Aii and S&Miii expenses (30.8) (27.1) +14%
Other operating income 9.4 6.8 +37%
Operating loss (33.1) (14.2)
Fair value adjustments and net exchange differences 30.6 (9.7)
Net other financial result 25.4 12.5
Income taxes 0.6 0.2
Net profit/loss (-) from continuing operations 23.5 (11.2)
Net profit from discontinued operations, net of tax 66.7 34.4
Net profit of the period 90.2 23.2
Basic and diluted earnings per share (€) 1.4 0.4
Current financial investments, cash & cash equivalents 3,557.9 4,005.5 (*)
(*) Including €15.4 million of net accrued interest income

DETAILS OF THE FINANCIAL RESULTS OF THE FIRST THREE MONTHS OF 2024
As a consequence of the transfer of our Jyseleca business to Alfasigma, the revenues and costs related to Jyseleca for the first quarter of 2024 are presented separately from the results of our continuing operations in the line ‘Net profit from discontinued operations, net of tax’ in our consolidated income statement. The comparative first quarter of 2023 has been restated accordingly for the presentation of the results related to the Jyseleca business.

Results from our continuing operations
Total operating loss from continuing operations for the three months ended 31 March 2024 was €33.1 million, compared to an operating loss of €14.2 million for the three months ended 31 March 2023.

Total net revenues for the three months ended 31 March 2024 amounted to €62.4 million, compared to €58.6 million for the three months ended 31 March 2023. The revenue recognition related to the exclusive access rights granted to Gilead for our drug discovery platform amounted to €57.6 million for the first three months of both 2024 and 2023. Our deferred income balance at 31 March 2024 includes €1.2 billion allocated to our drug discovery platform that is recognized linearly over the remaining period of our 10-year collaboration.
Cost of sales for the three months ended 31 March 2024 amounted to €2.5 million and related to the supply of Jyseleca to Alfasigma under the transition agreement. The related revenues are booked in total net revenues.
R&D expenses in the first three months of 2024 amounted to €71.6 million, compared to €52.5 million for the first three months of 2023. This increase was primarily explained by higher costs for CAR-T and small molecule programs in oncology.
G&A and S&M expenses amounted to €30.8 million in the first three months of 2024, compared to €27.1 million in the first three months of 2023. This increase was primarily due to an increase in legal and professional fees, mainly related to business development activities.
Other operating income amounted to €9.4 million in the first three months of 2024, compared to €6.8 million for the same period last year. This increase is mainly due to €2.2 million of recharges for transition services to Alfasigma for the months of February and March 2024.
Net financial income in the first three months of 2024 amounted to €56.0 million, compared to net financial income of €2.8 million for the first three months of 2023.

Fair value adjustments and net currency exchange gains in the first three months of 2024 amounted to €30.6 million, compared to fair value adjustments and net currency exchange losses of €9.7 million for the first three months of 2023, and were primarily attributable to €12.4 million of unrealized currency exchange gains on our cash and cash equivalents and current financial investments at amortized cost in U.S. dollars, and to €17.6 million of positive changes in fair value of current financial investments.
Net other financial income in the first three months of 2024 amounted to €25.4 million, compared to net other financial income of €12.5 million for the first three months of 2023, and was primarily attributable to €25.2 million of interest income, which increased significantly due to the increase in interest rates.
Net profit from continuing operations for the first three months of 2024 was €23.5 million, compared to a net loss from continuing operations of €11.2 million for the first three months of 2023.

Results from discontinued operations

(€ millions)

Three months ended 31 March % Change

2024 2023
Product net sales 11.3 26.7 -58%
Collaboration revenues 26.0 93.6 -72%
Total net revenues 37.3 120.3 -69%
Cost of sales (1.9) (3.6) -47%
R&D expenses (13.4) (51.0) -74%
G&A and S&M expenses (9.2) (31.1) -71%
Other operating income 53.9 1.5
Operating profit 66.7 36.1
Net financial result 0.1 (1.3)
Income taxes (0.1) (0.4)
Net profit from discontinued operations 66.7 34.4
Total operating profit from discontinued operations amounted to €66.7 million in the first three months of 2024, compared to an operating profit of €36.1 million in the same period last year.

Product net sales of Jyseleca in Europe were €11.3 million for the first three months of 2024 consisting of sales to customers in January 2024. Product net sales to customers for the first three months of 2023 amounted to €26.7 million. As from 1 February 2024, all economics linked to the sales of Jyseleca in Europe are to the benefit of Alfasigma.
Collaboration revenues for the development of filgotinib with Gilead amounted to €26.0 million for the first three months of 2024, compared to €93.6 million for the same period last year. The sale of the Jyseleca business to Alfasigma on 31 January 2024 led to the full recognition in revenue of the remaining deferred income related to filgotinib.
Cost of sales related to Jyseleca net sales were €1.9 million for the first three months of 2024. Cost of sales related to Jyseleca net sales for the first three months of 2023 amounted to €3.6 million.
R&D expenses for the development of filgotinib for the first three months of 2024 amounted to €13.4 million, compared to €51.0 million in the first three months of 2023. As from 1 February 2024, all filgotinib development expenses are recharged to Alfasigma.
G&A and S&M expenses related to the Jyseleca business amounted to €9.2 million in the first three months of 2024, compared to €31.1 million in the first three months of 2023. As from 1 February 2024, all remaining G&A and S&M expenses relating to Jyseleca are recharged to Alfasigma.
Other operating income for the first three months of 2024 amounted to €53.9 million (€1.5 million for the same period last year) and comprised €53.2 million related to the preliminary calculation of the gain on the sale of the Jyseleca business to Alfasigma. This preliminary result at 31 March 2024 of the transaction is considering the following elements:
€50.0 million of upfront payment received at closing of the transaction of which €40.0 million was paid on an escrow account. This amount will be kept in escrow for a period of one year after the closing date of 31 January 2024. We gave customary representations and warranties which are capped and limited in time (at 31 March 2024, this €40.0 million is presented as "Escrow account" in our balance sheet).
€13.2 million of cash received at closing of the transaction from Alfasigma for preliminary settlement for net cash and working capital and an additional adjustment estimated at €1.1 million related to settlement for completion accounts.
€47.0 million of estimated fair value on 31 January 2024 of the future earn-outs payable by Alfasigma to us (the fair value of these future earn-outs at 31 March 2024 is presented on the lines "Non-current contingent consideration receivable" and "Trade and other receivables").
€40.0 million of liability towards Alfasigma on 31 January 2024 for R&D cost contributions of which €5.0 million was paid in the first quarter of 2024 (at 31 March 2024, €35.0 million of liabilities for R&D cost contribution is presented in our balance sheet in "Other non-current liabilities" for €10.0 million and on the line "Trade and other liabilities" for €25.0 million).
Net profit from discontinued operations related to Jyseleca amounted to €66.7 million for the first three months of 2024, compared to a net profit amounting to €34.4 million for the first three months of 2023.

Cash, cash equivalents and current financial investments totaled €3,557.9 million as of 31 March 2024, as compared to €3,684.5 million as of 31 December 2023. Total net decrease in cash and cash equivalents and current financial investments amounted to €126.6 million during the first three months of 2024, compared to a net decrease of €88.6 million during the first three months of 2023. This net decrease was composed of (i) €125.2 million of operational cash burn, (ii) €36.9 million for the acquisition of financial assets held at fair value through profit or loss, (iii) €38.7 million of net cash in related to the sale of the Jyseleca business to Alfasigma of which €40.0 million has been transferred to an escrow account, offset by (iv) €36.8 million of positive exchange rate differences, positive changes in fair value of current financial investments and variation in accrued interest income.

Outlook 2024

Financial outlook
For the full year 2024, we reconfirm our cash burn guidance of €280 million to €320 million (compared to €414.8 million for the full year 2023), not including future potential business development opportunities.

R&D Outlook
We are progressing three CAR-T Phase 1/2 studies in hemato-oncology:
GLPG5101 in relapsed/refractory NHL;
GLPG5201 in relapsed/refractory CLL, and RT; and
GLPG5301 in relapsed/refractory multiple myeloma.
We are progressing two Phase 2 studies with TYK2 inhibitor GLPG3667, in systemic lupus erythematosus and in dermatomyositis.
We plan to file Investigational New Drug applications in the U.S. to progress clinical development of our CAR-T programs in hemato-oncology.
We will further upscale our CAR-T network and operations in the U.S. and Europe.
Business development
We will continue to evaluate business development opportunities that fit our strategy to accelerate and expand our pipeline of potential best-in-class investigational medicines in our therapeutic focus areas of oncology and immunology.

Conference call and webcast presentation
We will host a conference call and webcast presentation on 3 May 2024, at 14:00 CET / 8:00 am ET. To participate in the conference call, please register in advance using this link. Dial-in numbers will be provided upon registration. The conference call can be accessed 10 minutes prior to the start of the call by using the conference access information provided in the email received after registration, or by selecting the "call me" feature.

The live webcast is available on glpg.com or via the following link. The archived webcast will be available for replay shortly after the close of the call on the investor section of the website.