On February 19, 2025 Clarity Pharmaceuticals (ASX: CU6) ("Clarity" or "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 the United States (US) Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) for 67Cu-SAR-bisPSMA for the treatment of adult patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) who have been previously treated with androgen receptor pathway inhibition (ARPI) (Press release, Clarity Pharmaceuticals, FEB 19, 2025, View Source [SID1234650344]).

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!

This milestone builds on Clarity’s earlier receipt of 2 FTDs for the diagnostic 64Cu-SAR-bisPSMA in patients with suspected metastasis of prostate cancer who are candidates for initial definitive therapy1, as well as patients with biochemical recurrence (BCR) of prostate cancer following definitive therapy2, with 2 Phase III registration trials underway (CLARIFY [NCT06056830]3 and AMPLIFY, respectively). These 3 FTDs demonstrate the quality of the data generated to date on the 64Cu-SAR-bisPSMA and 67Cu-SAR-bisPSMA products in addressing serious unmet needs in prostate cancer. The FTDs will enable Clarity to accelerate the development of its comprehensive program with the optimised SAR-bisPSMA agent to be used in patients with prostate cancer throughout the management of their cancer, from initial to late-stage disease, with an opportunity to completely change the entire treatment landscape for the large prostate cancer market.

The FDA’s FTD is designed to expedite the development and regulatory review of novel drugs addressing serious conditions with significant unmet medical needs. For SAR-bisPSMA, it provides a number of product development advantages. The designations pave the way for a faster review process once Clarity submits its product approval applications. Additionally, it enables more frequent communication with the FDA, allowing for rapid resolution of queries during development. Furthermore, Clarity can submit completed sections of its application as they are ready, rather than waiting for the entire package to be finished before it can be lodged with the FDA. These benefits would reduce the review time needed to bring this innovative and proprietary molecule to the prostate cancer imaging and therapy markets.

The data for this FTD submission was based on the preliminary results to date from the Phase I/IIa SECuRE study (NCT04868604)4, which is investigating the safety and efficacy of 67Cu-SAR-bisPSMA for the treatment of mCRPC patients. The first 3 cohorts in the dose escalation phase of the trial were successfully completed with no dose limiting toxicities (DLTs) reported in any of the participants dosed (15 participants). No adverse events (AEs) related to 64Cu-SAR-bisPSMA were observed. Most AEs related to 67Cu-SAR-bisPSMA were low grade (grade 1 or 2). The most common AE reported was mild dry mouth (grade 1, 5/15 participants, 33.3%).

Preliminary data shows that the majority of participants in the SECuRE study enrolled to date had bone metastasis (77%), high median prostate-specific antigen (PSA) level at baseline (112.86 ng/mL, range 0.1-1503.1) and were heavily pre-treated (59% of participants received 3 or more lines of therapy). Despite how heavily pre-treated these participants were, and how much disease they had, 73% of them across all cohorts (including the lowest dose cohort of 67Cu-SAR-bisPSMA at 4 GBq, single dose) showed reductions in PSA levels. The majority of patients that had an increase in PSA were on the single, lowest dose cohort 1 (4 GBq). PSA reductions of greater than 50% were seen in 45% of all trial participants, despite the overwhelming majority of participants only receiving a single dose of 67Cu-SAR-bisPSMA (4, 8 or 12 GBq) in the trial. In cohorts 2, 3 and 4 (8 and 12 GBq single dose and 12 GBq multi-dose, respectively), in which most participants also only received 1 dose of 67Cu-SAR-bisPSMA, PSA reductions of greater than 35% were observed in almost 75% of participants and PSA was reduced by 80% or more in almost half of the participants so far, as patients continue in follow-up.

The trial is currently progressing through the highest dose cohort where participants were administered multiple doses of 12 GBq of 67Cu-SAR-bisPSMA. Recruitment into cohort 4 is complete, and the remaining 3 participants are currently in the safety and efficacy follow-up period after receiving their first 2 doses. Following completion of the follow-up up period, the safety review committee meeting is planned for March 2025. The largest drop in prostate-specific antigen (PSA) in cohort 4 to date is a decline of 98% (from a baseline of 157.4 ng/mL). This participant, who had failed multiple lines of therapy prior to receiving 67Cu-SAR-bisPSMA (androgen deprivation therapy [ADT], ARPI and an investigational agent), has already had a radiographic partial response based on the investigator’s assessment of Response Evaluation Criteria in Solid Tumours v1.1 (RECIST) criteria. Preliminary analysis showed a reduction of 60.6% in tumour volume evaluated by PSMA positron emission tomography [PET] imaging with 64Cu-SAR-bisPSMA.

Cohort expansion of the SECuRE trial to assess the combination of 67Cu-SAR-bisPSMA with enzalutamide

For a long time, Clarity has been working closely with many important global medical experts in the field of prostate cancer, including the Company’s Clinical Advisory Board members, Prof Louise Emmett and Prof Oliver Sartor, to optimise the development of all of its products in prostate cancer. Those discussions have led to a recent protocol amendment for the SECuRE trial, which aims to investigate ways to further improve the treatment outcomes for these patients. The protocol amendment is aligned with the positive results of the Enza-p trial presented by Prof Emmett first at the European Society for Medical Oncology in 20235 and more recently at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers (ASCO GU) Symposium in 20256, which confirmed the hypothesis that targeting both androgen signalling and PSMA receptors concurrently would improve anti-cancer activity in mCRPC. The latest SECuRE protocol amendment increased the number of participants in the cohort expansion phase from 14 to 24 patients in the mCRPC pre-chemotherapy setting, with a subset of patients to receive the combination therapy of 67Cu-SAR-bisPSMA with enzalutamide. This protocol amendment has now been approved at many of the participating trial sites, and the changes are expected to further enhance the already positive results of 67Cu-SAR-bisPSMA observed in the SECuRE trial to date. This strategy focuses on the commercialisation of the product firstly in the largest market for prostate cancer therapies in mCRPC, with pre-chemotherapy being 3 times larger than the post-chemotherapy setting, and creates opportunities for the use of 67Cu-SAR-bisPSMA with a range of ARPIs in future clinical development. Addressing the radiopharmaceuticals supply issues with current treatments in such a large indication requires a streamlined approach for which Clarity’s 64Cu/67Cu platform is well and uniquely suited.

Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "Receiving 3 FTDs for the one molecule, SAR-bisPSMA, within the last 6 months is an incredible achievement for Clarity, highlighting how impressive our science and development are, the significance of the diagnostic and therapeutic data so far, and the high unmet need for better therapies and diagnostics in prostate cancer.

"The dual-targeted bisPSMA molecule was developed at the benchtop of Australian science with the intent of overcoming the shortfalls of the current generation of PSMA-targeting products. It was optimised with two PSMA ligands, which increases not only the amount of product in the lesions, but also how long the product is retained in the lesions over time, making it an ideal candidate for both diagnosis and therapy. The clinical data in both diagnostic and therapeutic indications that we are generating is remarkable, confirming the results that we initially saw in preclinical development. The granting of FTDs by the US FDA for 3 distinct indications in prostate cancer that we are aiming to address with this product is testament to the incredible work of our team and collaborators. This latest FTD will allow us flexibility to develop 67Cu-SAR-bisPSMA in both pre- and post-chemotherapy patients in the mCRPC setting, with initial focus on the largest market segment. The SECuRE study will also provide invaluable information on the potential of 67Cu-SAR-bisPSMA to be combined with enzalutamide and other ARPIs in future, creating opportunities for the broader use of 67Cu-SAR-bisPSMA in those patients with such high unmet medical need.

"These designations will allow us to work closely with the FDA to facilitate the development process and accelerate the approval of what could become best-in-class therapy and diagnostic agents, and our team and collaborators are committed to making this our priority in order to achieve our ultimate goal of improving treatment outcomes for people with cancer."

About SAR-bisPSMA
SAR-bisPSMA derives its name from the word "bis", which reflects a novel approach of connecting two PSMA-targeting agents to Clarity’s proprietary sarcophagine (SAR) technology that securely holds copper isotopes inside a cage-like structure, called a chelator. Unlike other commercially available chelators, the SAR technology prevents copper leakage into the body. SAR-bisPSMA is a Targeted Copper Theranostic (TCT) that can be used with isotopes of copper-64 (Cu-64 or 64Cu) for imaging and copper-67 (Cu-67 or 67Cu) for therapy.

64Cu-SAR-bisPSMA is an unregistered product. The safety and efficacy of 64Cu-SAR-bisPSMA has not been assessed by health authorities such as the U.S. FDA or the Therapeutic Goods Administration (TGA). There is no guarantee that this product will become commercially available.

About the SECuRE trial
SECuRE (NCT04868604)4 is a Phase I/IIa theranostic trial for identification and treatment of an advanced form of prostate cancer, mCRPC. It is a multi-centre, single arm, dose escalation study with a cohort expansion. The aim of this trial is to determine the safety and tolerability of both 64Cu-SAR-bisPSMA and 67Cu-SAR-bisPSMA, as well as the efficacy of 67Cu-SAR-bisPSMA as a therapy. A recent protocol amendment has increased the number of participants in the cohort expansion phase from 14 to 24, and a subset of participants will receive the combination of 67Cu-SAR-bisPSMA with enzalutamide.

About Prostate Cancer
Prostate cancer is the second most common cancer diagnosed in men globally and the fifth leading cause of cancer death in men worldwide7. Prostate cancer is the second-leading causes of cancer death in American men. The American Cancer Institute estimates in 2025 there will be about 313,780 new cases of prostate cancer in the U.S. and around 35,770 deaths from the disease.

On February 19, 2025 Charles River Laboratories International, Inc. (NYSE: CRL) reported its results for the fourth quarter and full-year 2024 and provided guidance for 2025 (Press release, Charles River Laboratories, FEB 19, 2025, View Source [SID1234650374]). For the quarter, revenue was $1.00 billion, a decrease of 1.1% from $1.01 billion in the fourth quarter of 2023.

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 impact of foreign currency translation reduced reported revenue by 0.1%, and an acquisition contributed 0.9% to consolidated fourth-quarter revenue. A divestiture reduced reported revenue by 0.1%. Excluding the effect of these items, revenue declined 1.8% on an organic basis. On a segment basis, organic revenue growth in the Manufacturing Solutions (Manufacturing) segment was more than offset by lower revenue in the Discovery and Safety Assessment (DSA) and Research Models and Services (RMS) segments.

In the fourth quarter of 2024, the GAAP operating margin decreased to (16.7)% from 13.1% in the fourth quarter of 2023. The primary driver of the GAAP decrease was a non-cash goodwill impairment of $215.0 million in the fourth quarter of 2024 related to the Biologics Solutions reporting unit, which includes the Biologics Testing and CDMO businesses. On a non-GAAP basis, the fourth-quarter operating margin increased to 19.9% from 19.1%, due primarily to higher revenue and operating income in the Manufacturing segment and lower unallocated corporate costs.
On a GAAP basis, the net loss available to common shareholders for the fourth quarter of 2024 was $(215.7) million, or $(4.22) per share, a decrease from net earnings of $187.1 million, or $3.62 per diluted share, for the same period in 2023. The GAAP decreases were primarily driven by the non-cash goodwill impairment, which totaled $4.20 per share, as well as a loss of $0.32 per share on certain venture capital and other strategic investments. This compares to a gain of $2.04 per share on certain venture capital and other strategic investments in the fourth quarter of 2023, which included a gain on our original strategic investment in Noveprim Group.
On a non-GAAP basis, net income was $136.6 million for the fourth quarter of 2024, an increase of 7.4% from $127.2 million for the same period in 2023. Fourth-quarter diluted earnings per share on a non-GAAP basis were $2.66, an increase of 8.1% from $2.46 per share for the fourth quarter of 2023. The increases in non-GAAP net income and earnings per share were driven by higher operating income, as well as favorable below-the-line items, including reductions in the tax rate, interest expense, and diluted shares outstanding.
James C. Foster, Chair, President and Chief Executive Officer, said, "Throughout 2024, we have launched initiatives to generate more revenue, aggressively reduce costs, and further strengthen our business. As we move into 2025, we see many of our global biopharmaceutical clients continuing to move forward with their restructuring and pipeline reprioritization activities, which are expected to constrain early-stage spending by many of these clients again in 2025. We believe these trends are stabilizing, and therefore, our view of the global biopharmaceutical demand environment remains unchanged. In addition, small and mid-sized biotechnology clients continued to benefit from a more favorable funding environment in 2024, and we expect biotechnology demand trends will be stable to slightly improved this year."

"During this period of softer demand, we remain committed to executing on our revenue and cost-savings initiatives and protecting shareholder value. We are taking decisive actions to emerge from this period as a stronger, leaner, and more profitable company, and an even more responsive partner for our clients," Mr. Foster concluded.

Fourth-Quarter Segment Results
Research Models and Services (RMS)
Revenue for the RMS segment was $204.3 million in the fourth quarter of 2024, an increase of 4.3% from $195.8 million in the fourth quarter of 2023. The Noveprim acquisition in November 2023 contributed 4.8% to fourth-quarter RMS reported revenue, and the impact of foreign currency translation reduced revenue by 0.1%. Organic revenue decreased by 0.4%, due primarily to lower revenue for research models services and the Cell Solutions business, as well as lower sales for non-human primates (NHPs) in China. The decline was partially offset by higher sales of small research models, principally driven by higher pricing.
In the fourth quarter of 2024, the RMS segment’s GAAP operating margin decreased to 6.7% from 18.9% in the fourth quarter of 2023. On a non-GAAP basis, the operating margin decreased to 22.8% from 23.1%. The GAAP and non-GAAP operating margin declines were primarily driven by research models services and NHP sales, partially offset by higher pricing for small research models and from cost savings associated with restructuring initiatives. On a GAAP basis, the lower operating margin also reflects higher costs associated with the Company’s restructuring initiatives, including severance and site consolidation costs, as well as higher amortization expense related to the Noveprim acquisition.
Discovery and Safety Assessment (DSA)

Revenue for the DSA segment was $603.3 million in the fourth quarter of 2024, a decrease of 3.6% from $625.8 million in the fourth quarter of 2023. The divestiture of a small Safety Assessment site reduced reported revenue by 0.2% and the impact of foreign currency translation increased DSA revenue by 0.1%. Organic revenue decreased by 3.5%, driven primarily by lower sales volume, as well as slightly lower pricing.

In the fourth quarter of 2024, the DSA segment’s GAAP operating margin decreased to 10.4% from 20.2% in the fourth quarter of 2023. On a non-GAAP basis, the operating margin decreased to 24.7% from 26.0% in the fourth quarter of 2023. The GAAP and non-GAAP operating margin declines were primarily driven by lower revenue, partially offset by cost savings associated with restructuring initiatives. On a GAAP basis, the lower operating margin also reflects an NHP inventory write down, as well as higher acquisition-related adjustments associated with Noveprim.

Manufacturing Solutions (Manufacturing)

Revenue for the Manufacturing segment was $194.9 million in the fourth quarter of 2024, an increase of 1.6% from $191.9 million in the fourth quarter of 2023. The impact of foreign currency translation reduced Manufacturing revenue by 0.5%. Organic revenue increased 2.1%, primarily driven by the Microbial Solutions business. This was partially offset by lower revenue in the CDMO business.

Primarily as a result of the non-cash goodwill impairment, the Manufacturing segment’s GAAP operating margin decreased to (93.6)% from 18.5% in the fourth quarter of 2023. On a non-GAAP basis, the operating margin increased to 28.7% from 25.4% in the fourth quarter of 2023 driven primarily by improved operating leverage from higher revenue in the Microbial Solutions business, as well as the benefit of cost savings associated with restructuring initiatives.

Full-Year Results
For 2024, revenue decreased by 1.9% to $4.05 billion from $4.13 billion in 2023. Revenue declined by 2.8% on an organic basis.
The GAAP operating margin decreased to 5.6% from 14.9% in 2023, and on a non-GAAP basis, the operating margin decreased to 19.9% from 20.3%.
On a GAAP basis, net income available to common shareholders was $10.3 million in 2024, a decrease of 97.8% from $474.6 million in 2023. Diluted earnings per share on a GAAP basis in 2024 were $0.20, a decrease of 97.8% from $9.22 in 2023. The GAAP decreases were primarily driven by the non-cash goodwill impairment of $215.0 million, or $4.16 per share, as well as the loss on certain venture capital and other strategic investments of $0.15 per share in 2024. This compares to a gain of $1.87 per share on certain venture capital and other strategic investments in 2023.
On a non-GAAP basis, net income was $532.9 million in 2024, a decrease of 2.9% from $548.9 million in 2023. Diluted earnings per share on a non-GAAP basis in 2024 were $10.32, a decrease of 3.3% from $10.67 in 2023.
Research Models and Services (RMS)
For 2024, RMS revenue was $829.4 million, an increase of 4.7% from $792.3 million in 2023. Revenue declined by 0.1% on an organic basis.

On a GAAP basis, the RMS segment operating margin decreased to 13.8% in 2024 from 19.5% in 2023. On a non-GAAP basis, the operating margin increased to 23.7% in 2024 from 23.0% in 2023.

Discovery and Safety Assessment (DSA)
For 2024, DSA revenue was $2.45 billion, a decrease of 6.3% from $2.62 billion in 2023. Revenue declined by 6.2% on an organic basis.

On a GAAP basis, the DSA segment operating margin decreased to 18.1% in 2024 from 23.2% in 2023. On a non-GAAP basis, the operating margin decreased to 25.7% in 2024 from 27.5% in 2023.

Manufacturing Solutions (Manufacturing)
For 2024, Manufacturing revenue was $769.3 million, an increase of 6.6% from $721.4 million in 2023. Organic revenue growth was 6.8%.

On a GAAP basis, the Manufacturing segment operating margin decreased to (9.3)% in 2024 from 12.2% in 2023. The GAAP operating margin was impacted by the non-cash goodwill impairment. On a non-GAAP basis, the operating margin increased to 27.4% in 2024 from 21.8% in 2023.

Provides 2025 Guidance
The Company is providing financial guidance for 2025. The 2025 revenue outlook assumes relatively stable biopharmaceutical demand trends compared to those experienced during the second half of 2024, including continued budgetary constraints from global biopharmaceutical clients and stable to slightly improved demand from small and mid-sized biotechnology clients. In addition, we expect that DSA pricing will be a headwind to revenue growth throughout 2025, and that lower commercial revenue in the CDMO business will impact the Manufacturing segment’s growth rate. Earnings per share in 2025 will principally be affected by the lower revenue, partially offset by the benefit of cost savings associated with the Company’s restructuring initiatives. The Company plans to repurchase approximately $350 million in common stock in 2025.
The Company’s 2025 guidance for revenue and earnings per share is as follows:

2025 GUIDANCE
Revenue growth/(decrease), reported
(7.0)% – (4.5)%
Impact of divestitures/(acquisitions), net
N/M
(Favorable)/unfavorable impact of foreign exchange
1.0% – 1.5%
Revenue growth/(decrease), organic (1)
(5.5)% – (3.5)%
GAAP EPS estimate
$4.30 – $4.80
Acquisition-related amortization and other acquisition- and integration-related costs (2)
~$3.50
Costs associated with restructuring actions (3)
~$1.00
Other items (4)
~$0.30
Non-GAAP EPS estimate
$9.10 – $9.60

Footnotes to Guidance Table:
(1) Organic revenue growth is defined as reported revenue growth adjusted for completed acquisitions and divestitures, as well as foreign currency translation.
(2) These adjustments include amortization related to intangible assets, as well as the purchase accounting step-up on inventory and certain long-term biological assets. In addition, these adjustments include some costs related to the evaluation and integration of acquisitions and divestitures.
(3) These adjustments primarily include site consolidation (including site transition costs), severance, impairment, and other costs related to the Company’s restructuring actions.
(4) These items primarily relate to certain third-party legal costs related to investigations by the U.S. government into the NHP supply chain related to our Safety Assessment business.

Webcast
Charles River has scheduled a live webcast on Wednesday, February 19th, at 8:30 a.m. ET to discuss matters relating to this press release. To participate, please go to ir.criver.com and select the webcast link. You can also find the associated slide presentation and reconciliations of GAAP financial measures to non-GAAP financial measures on the website.

On February 19, 2025 Oncolytics Biotech Inc. (NASDAQ: ONCY) (TSX: ONC), a leading clinical-stage company specializing in immunotherapy for oncology, reported to make good progress in 2025 with key regulatory and clinical advancements, reinforcing pelareorep’s potential in hard-to-treat cancers (Press release, Oncolytics Biotech, FEB 19, 2025, View Source [SID1234650391]). Oncolytics is pleased to highlight two significant developments for its immunotherapy, pelareorep: the safety and regulatory clearance to advance enrollment in its pancreatic cancer study and the recent presentation of new efficacy and safety data at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium in late January.

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’re hitting critical milestones that validate our progress and set the stage for what we believe will be an exciting year," said Wayne Pisano, Interim CEO and Chair of Oncolytics’ Board of Directors. "With positive feedback from regulators in place, we’re advancing our pancreatic cancer study toward full enrollment, and our ASCO (Free ASCO Whitepaper) GI presentations highlighted pelareorep’s strong safety and efficacy results in two hard-to-treat cancers. We remain focused on bringing new treatment options to patients while creating value for shareholders as we move forward in 2025."

German Regulatory Agency Gives Green Light for Pancreatic Cancer Study to Continue as Planned

Approval to Fully Enroll the Cohort Secured: Germany’s Paul-Ehrlich-Institute (PEI) has given Oncolytics the go-ahead to continue enrolling patients in its pancreatic cancer trial (GOBLET Cohort 5) after a positive safety review.
What This Means: Pelareorep, in combination with modified FOLFIRINOX with and without atezolizumab, is now progressing toward full enrollment, with 30 patients set to participate in Stage 1 across the two treatment arms.
Next Steps: Oncolytics will continue to collect safety data, and an initial efficacy readout is expected later this year.
ASCO GI 2025 Data Confirms Pelareorep’s Potential in Pancreatic and Anal Cancers

At ASCO (Free ASCO Whitepaper) GI 2025, Oncolytics presented new clinical results demonstrating pelareorep’s potential in two challenging cancer types:

Anal Cancer: Patients receiving pelareorep + atezolizumab continue to show stronger responses than expected based on published studies with checkpoint inhibitors alone.
Pancreatic Cancer: Pelareorep previously demonstrated a strong efficacy signal when administered with gemcitabine, nab-paclitaxel, and atezolizumab. The most recent data supports a favorable safety profile when combining pelareorep with a different chemotherapy regimen (modified FOLFIRINOX) with and without the checkpoint inhibitor atezolizumab, potentially expanding its treatment applications.
Why This Matters: These findings further de-risk pelareorep’s development and could pave the way for larger registration-enabling clinical trials in these indications.

Looking Ahead: More Catalysts in 2025

Oncolytics is entering a pivotal year with multiple upcoming milestones, including:

Additional data readouts from ongoing trials in gastrointestinal cancers, including translational results that further characterize pelareorep’s mechanism of action.
Interactions with Regulatory Agencies that could accelerate future trials and move pelareorep closer to potential registration-enabling studies in breast cancer and gastrointestinal cancers.
"We’re seeing clinical validation across multiple studies," added Pisano. "With encouraging regulatory interactions in hand and data readouts ahead, 2025 is shaping up to be an exciting year for Oncolytics and our investors. As we have shown in GOBLET, BRACELET-1, and numerous previous studies, pelareorep has a favorable safety profile and efficacy signals across multiple indications with a high unmet need. We are excited about the potential for moving to a registration-enabling study in breast cancer and advancing our clinical program in gastrointestinal cancers."

About GOBLET

The GOBLET (Gastrointestinal tumOrs exploring the treatment comBinations with the oncolytic reovirus peLarEorep and anTi-PD-L1) study is a phase 1/2 multiple indication study in advanced or metastatic gastrointestinal tumors. The study is being conducted at 17 centers in Germany and is being managed by AIO-Studien-gGmbH. The co-primary endpoints of the study are objective response rate (ORR) and/or disease control rate and safety. Key secondary and exploratory endpoints include additional efficacy assessments and evaluation of potential biomarkers. Favorable safety and positive clinical efficacy signals have been seen in the pancreatic and anal cancer cohorts.

About GOBLET Cohort 5

The modified FOLFIRINOX (mFOLFIRINOX) cohort of the Phase 1/2 GOBLET study is designed to evaluate newly diagnosed metastatic pancreatic ductal adenocarcinoma patients treated with pelareorep + mFOLFIRINOX with or without atezolizumab. A three-patient safety run-in was incorporated to evaluate the safety and tolerability of each treatment arm: pelareorep + mFOLFIRINOX + atezolizumab and pelareorep + mFOLFIRINOX. A total of fifteen evaluable patients will be randomized to each arm in Stage 1 of this Simon two-stage study. The co-primary endpoints are objective response rate and safety. If Stage 1 success criteria are met, one or both treatment arms may be expanded to Stage 2, in which 17 additional evaluable patients per arm will be enrolled. Blood and tumor samples will also be collected for translational evaluations.

On February 18, 2025, Intra-Cellular Therapies, Inc., a Delaware corporation (the "Company"), filed its definitive proxy statement on Schedule 14A (as such may be supplemented from time to time, the "Proxy Statement") with the Securities and Exchange Commission (the "SEC") with respect to the special meeting of the Company’s stockholders (the "Special Meeting") to be held in connection with transactions contemplated by that certain Agreement and Plan of Merger (the "Merger Agreement"), dated as of January 10, 2025, by and among the Company, Johnson & Johnson, a New Jersey corporation ("Johnson & Johnson"), and Fleming Merger Sub, Inc., a Delaware corporation and a wholly owned subsidiary of Johnson & Johnson ("Merger Sub"), pursuant to which, subject to the terms and conditions thereof, Merger Sub will merge with and into the Company (the "Merger"), with the Company surviving as a wholly owned subsidiary of Johnson & Johnson (Filing, 8-K, Intra-Cellular Therapies, MAR 18, 2025, View Source [SID1234651221]).

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 Special Meeting is scheduled for March 27, 2025, beginning at 9:00 a.m. Eastern Time. The Company’s stockholders of record as of the close of business on February 13, 2025 will be eligible to vote at the Special Meeting. The information contained in this Current Report on Form 8-K (this "Form 8-K") should be read in conjunction with the Proxy Statement, which should be read in its entirety.

Litigation Relating to the Merger

On February 26, 2025 a purported stockholder of the Company filed a lawsuit in the New York State Supreme Court against the Company and its Board of Directors, captioned Morgan v. Intra-Cellular Therapies, Inc., et al., Index No. 651153/2025 (the "Morgan Complaint"). On February 27, 2025, a purported stockholder of the Company filed a lawsuit in the New York State Supreme Court against the Company and its Board of Directors, captioned O’Neill v. Intra-Cellular Therapies, Inc., et al., Index No. 651115/2025 (the "O’Neill Complaint"). The Morgan Complaint and the O’Neill Complaint generally allege, among other things, that the Proxy Statement negligently misrepresents and/or fails to disclose material information in violation New York law. The Morgan Complaint and the O’Neill Complaint each seek, among other things, injunctive relief, rescissory or other unspecified monetary damages, and an award of attorneys’ fees and expenses.

On March 3, 2025, a purported stockholder of the Company filed a lawsuit in the Superior Court of New Jersey, Somerset County, against the Company, its Board of Directors and Johnson & Johnson, captioned Drulias v. Mates, et al., Case No. SOM C-012016-25 (the "Drulias Complaint" and together with the Morgan Complaint and the O’Neill Complaint, the "Complaints"). The Drulias Complaint generally alleges, among other things, that the Company’s Board of Directors breached its fiduciary duties under Delaware law in entering into the Merger, that the Company and its Board of Directors failed to disclose material information in the Proxy Statement, and that Johnson & Johnson aided and abetted those breaches. The Drulias Complaint seeks, among other things, a declaration that the defendants have breached their fiduciary duties and/or aided and abetted such breaches, an order requiring corrective disclosures, injunctive relief, unspecified compensatory and/or rescissory damages, and an award of attorneys’ fees and expenses.

As of the date of this Form 8-K, attorneys representing multiple purported stockholders of the Company have also delivered demand letters to the Company (collectively, the "Demand Letters") alleging that the disclosures contained in the Proxy Statement are deficient and requesting that the Company supplement such disclosures prior to the Special Meeting. The Demand Letters threaten the Company with lawsuits in the event that the purported deficiencies in the Proxy Statement are not addressed.

It is possible that additional, similar complaints may be filed, that the Complaints described above may be amended, or that additional demand letters will be received by the Company. If this occurs, the Company does not intend to announce the filing or receipt of each additional, similar complaint or demand letter or any amended complaint unless required by law.

The Company believes that the claims asserted in the Complaints and the Demand Letters are without merit. However, in order to moot the unmeritorious disclosure claims, alleviate the costs, risks and uncertainties inherent in potential litigation and provide additional information to its stockholders, the Company has determined to voluntarily supplement the Proxy Statement as described in this Form 8-K. Nothing in this Form 8-K shall be deemed an

admission of the legal necessity or materiality under applicable laws of any of the disclosures set forth herein. To the contrary, the Company specifically denies all allegations set forth in the Complaints and the Demand Letters that any additional disclosure in the Proxy Statement was or is required.

Supplemental Disclosures

The following disclosures supplement the disclosures contained in the Proxy Statement and should be read in conjunction with the disclosures contained in the Proxy Statement, which should be read in its entirety. To the extent the information set forth herein differs from or updates information contained in the Proxy Statement, the information set forth herein shall supersede or supplement the information in the Proxy Statement. All page references are to pages in the Proxy Statement, and terms used below, unless otherwise defined, have the meanings set forth in the Proxy Statement.

(a)
The disclosure under the section entitled " — Opinions of ITI’s Financial Advisors – Opinion of Centerview Partners LLC" is hereby amended and supplemented by deleting the bulleted list of selected companies beginning on page 49 of the Proxy Statement, and replacing it with the following chart:

Company
EV / 2027 Revenue Multiple

Acadia Pharmaceuticals Inc.

1.8x
Alkermes plc.

2.9x
Amicus Therapeutics, Inc.

3.1x
Apellis Pharmaceuticals, Inc.

3.6x
Axsome Therapeutics, Inc.

3.0x
Neurocrine Biosciences, Inc.

3.6x
Sarepta Therapeutics, Inc.

3.1x
TG Therapeutics, Inc.

4.7x

(b)
The disclosure under the section entitled " — Opinions of ITI’s Financial Advisors – Opinion of Centerview Partners LLC" is hereby amended and supplemented by adding the following double underlined language to a new rightmost column of the table set forth on page 51 of the Proxy Statement:

Date Announced
Target

Acquiror TV / Three-Year
Forward Revenue
Multiple
December 2022 Horizon Therapeutics Public Limited Company Amgen Inc. 5.4x
August 2022 Global Blood Therapeutics, Inc. Pfizer Inc. 7.3x
May 2022 Biohaven Pharmaceutical Holding Company Ltd. Pfizer Inc. 6.0x
February 2021 GW Pharmaceuticals plc Jazz Pharmaceuticals Public
Limited Company 6.0x
December 2020 Alexion Pharmaceuticals, Inc. AstraZeneca PLC 5.2x
January 2018 Bioverativ Inc. Sanofi SA 7.0x
January 2017 Actelion Ltd. Johnson & Johnson 9.9x
January 2016 Baxalta Incorporated Shire plc 4.6x
January 2015 NPS Pharmaceuticals, Inc. Shire plc 6.9x

(c)
The disclosure under the section entitled " — Opinions of ITI’s Financial Advisors – Opinion of Centerview Partners LLC" is hereby amended and supplemented by adding the following double underlined language to the last paragraph beginning on page 51 of the Proxy Statement:

In performing this analysis, Centerview calculated a range of equity values for shares of our common stock by (a) discounting to present value as of December 31, 2024 using discount rates ranging from 10.25% to 12.25% (reflecting

Centerview’s analysis of ITI’s weighted average cost of capital) and using a mid-year convention: (i) the forecasted risk-adjusted, after-tax unlevered free cash flows of ITI over the period beginning on January 1, 2025 and ending on December 31, 2047, utilized by Centerview based on the Projections, (ii) a range of implied terminal values of ITI, calculated by Centerview by adjusting ITI’s fiscal year 2047 risk-adjusted, after-tax unlevered free cash flow based on the Projections to account for normalized levels of working capital and research and development expenses, and applying a selected range of perpetuity growth rates of 0.0% to 3.0% (which range Centerview was instructed to use by ITI’s management), based on information provided by ITI management as of January 9, 2025 and set forth in the internal data and (iii) tax savings from usage of ITI’s estimated federal net operating losses as of December 31, 2024 of $468 million and research and development tax credits as of December 31, 2024 of $51 million and ITI’s future losses, in each case which amount was provided by ITI management as set forth in the internal data and (b) adding to the foregoing results ITI’s estimated net cash as of December 31, 2024 of approximately $1 billion, which amount was provided by ITI management as set forth in the internal data. Centerview divided the result of the foregoing calculations by the number of fully diluted outstanding shares of ITI’s common stock (determined using the treasury stock method and taking into account approximately 106.2 million shares of ITI’s common stock, approximately 3.1 million outstanding in-the-money options with a weighted average exercise price of $31.23, approximately 2.0 million restricted stock units, and approximately 0.2 million performance stock units (including assumptions regarding the vesting of such performance stock units)), based on information provided by ITI management as set forth in the internal data. This analysis resulted in a range of implied equity values per share of our common stock of $102.90 to $127.70, rounded to the nearest $0.05. Centerview then compared this range to the Merger Consideration of $132.00 per share to be paid to the holders of our common stock (other than Excluded Shares) pursuant to the Merger Agreement.

(d)
The disclosure under the section entitled " — Opinions of ITI’s Financial Advisors – Opinion of Centerview Partners LLC" is hereby amended and supplemented by adding the following double underlined language to the second and third bullets under the first full paragraph beginning on page 52 of the Proxy Statement:

•
Analyst Price Targets Analysis. Centerview reviewed stock price targets for shares of ITI’s common stock in 14 publicly available Wall Street research analyst reports as of January 8, 2025 which indicated low and high stock price targets for ITI ranging from $87.00 to $135.00 per share (with a median price target of $101.00 per share).

•
Precedent Premiums Paid Analysis. Centerview performed an analysis of premiums paid in the selected transactions involving publicly traded scaled commercial stage biotechnology companies, as set forth above in "Selected Precedent Transaction Analysis". The premiums in this analysis were calculated by comparing the per share acquisition price in each transaction to the closing price of the target company’s common stock for the date one day prior to the date on which the trading price of the target company’s common stock was perceived by Centerview to be affected by a potential transaction, which indicated low and high premiums of 37% and 102% (with a median of 51%). Based on the analysis above and other considerations that Centerview deemed relevant in its professional judgment, Centerview applied a premium range of 35% to 85% to ITI’s closing stock price on January 8, 2025 (the last full trading day prior to the date on which the Board of Directors met to approve the Merger) of $82.56, which resulted in an implied price range of approximately $111.45 to $152.75 per share, rounded to the nearest $0.05.

(e)
The disclosure under the section entitled " —Opinions of ITI’s Financial Advisors – Opinion of Jefferies LLC" is hereby amended and supplemented by adding the following double underlined language to a new rightmost column of the table beginning on page 56 of the Proxy Statement:

Date Announced
Target

Acquiror Enterprise Value /
Peak Sales Multiples
December 2023 Karuna Therapeutics, Inc. Bristol-Myers Squibb Company 1.6x
July 2023 Reata Pharmaceuticals, Inc. Biogen Inc. 2.4x
April 2023 IVERIC bio, Inc. Astellas Pharma Inc. 1.4x
August 2022 Global Blood Therapeutics, Inc. Pfizer Inc. 1.8x

May 2022 Biohaven Pharmaceutical Holding Company Ltd. Pfizer Inc. 1.8x
February 2021 GW Pharmaceuticals plc Jazz Pharmaceuticals Public
Limited Company 2.4x
October 2020 MyoKardia, Inc. Bristol-Myers Squibb Company 1.7x
November 2019 The Medicines Company Novartis AG 1.5x

(f)
The disclosure under the section entitled " —Opinions of ITI’s Financial Advisors – Opinion of Jefferies LLC" is hereby amended and supplemented by deleting the bulleted list of selected companies below the third paragraph on page 57 of the Proxy Statement, and replacing it with the following chart:

Company Enterprise Value / 2027 Revenue Multiples
Acadia Pharmaceuticals Inc. 1.8x
Alkermes plc. 2.9x
Amicus Therapeutics, Inc. 3.1x
Apellis Pharmaceuticals, Inc. 3.6x
Axsome Therapeutics, Inc. 3.0x
Neurocrine Biosciences, Inc. 3.6x
Sarepta Therapeutics, Inc. 3.1x
TG Therapeutics, Inc. 4.7x

(g)
The disclosure under the section entitled " —Opinions of ITI’s Financial Advisors – Opinion of Jefferies LLC" is hereby amended and supplemented by adding the following double underlined language to the second paragraph on page 58 of the Proxy Statement:

Jefferies performed a discounted cash flow analysis of ITI by calculating the estimated present value of the stand-alone risk-adjusted unlevered free cash flows, as described further in the section of this proxy statement captioned " —Certain Financial Projections", that ITI was forecasted to generate during the fiscal years ending December 31, 2025 through December 31, 2047 based on the Projections. Jefferies also calculated a range of terminal values for ITI by adjusting ITI’s fiscal year 2047 risk-adjusted unlevered free cash flow based on the Projections to account for normalized levels of working capital and research and development expenses, and applying a selected range of perpetuity growth rates of 0.0% to 3.0% (which range Jefferies was instructed to use by ITI’s management). The net present value as of December 31, 2024 of the risk-adjusted unlevered free cash flows and terminal value of ITI described in the preceding sentences were then calculated using the mid-year convention and a selected discount rate range of 10.25% to 12.25%, based on Jefferies’ estimate of ITI’s weighted average cost of capital. This resulted in a range of implied enterprise values for ITI. Jefferies then added ITI’s estimated net cash as of December 31, 2024 of approximately $1 billion, and the present value of ITI’s estimated federal net operating loss carryforwards of $468 million and research and development credits of $51 million, each as of December 31, 2024, and future losses (in each case as provided by ITI’s management), to calculate a range of implied equity values. Jefferies divided the result by the number of fully diluted outstanding shares of our common stock as of January 9, 2025 (determined using the treasury stock method and taking into account approximately 106.2 million shares of ITI’s common stock, approximately 3.1 million outstanding in-the-money options with a weighted average exercise price of $31.23, approximately 2.0 million restricted stock units, and approximately 0.2 million performance stock units (including assumptions regarding the vesting of such performance stock units)), as provided by ITI’s management, to calculate a range of implied per share equity values for ITI. This analysis indicated a reference range of implied per share equity values of $102.90 to $127.70 per share, as compared to the Merger Consideration of $132.00 per share.

(h)
The disclosure under the section entitled " —Opinions of ITI’s Financial Advisors – Opinion of Jefferies LLC" is hereby amended and supplemented by adding the following double underlined language to the second bullet under the third full paragraph on page 58 of the Proxy Statement:

•
the publicly available stock price targets for our shares of common stock by 14 research analysts, which indicated low and high stock price targets ranging from $87.00 per share to $135.00 per share (with a median price target of $101.00 per share), as compared to the Merger Consideration of $132.00 per share.

Additional Information and Where to Find It

On February 18, 2025, the Company filed the Proxy Statement with the SEC in connection with the proposed acquisition of the Company by Parent. Beginning on February 18, 2025, the Proxy Statement and proxy card were mailed to the Company’s stockholders of record as of February 13, 2025. Other documents regarding the transaction may be filed with the SEC. This Current Report on Form 8-K is not a substitute for the Proxy Statement or any other document that the Company may file with the SEC and send to its stockholders in connection with the transaction. Before making any voting decision, the Company’s stockholders are urged to read all relevant documents filed or to be filed with the SEC, including the Proxy Statement, as well as any amendments or supplements to those documents, when they become available, because they contain or will contain important information about the Company and the proposed acquisition.

The Company’s stockholders are or will be able to obtain a free copy of the Proxy Statement, as well as other filings containing information about Parent and the Company, free of charge, at the SEC’s website (www.sec.gov). Copies of the Proxy Statement and other documents filed by the Company with the SEC may be obtained from the Company, free of charge, by contacting the Company through its website at View Source

Participants in the Solicitation

Parent and the Company and certain of their respective directors and executive officers, under SEC rules, may be deemed to be "participants" in the solicitation of proxies from stockholders of the Company in connection with the proposed transaction. Information about Parent’s directors and executive officers is available in Parent’s Annual Report on Form 10-K for the year ended December 29, 2024, which was filed with the SEC on February 13, 2025, and Parent’s definitive proxy statement for its 2024 annual meeting of stockholders, which was filed with the SEC on March 13, 2024. Information about the Company’s directors and executive officers is available in the Proxy Statement, which was filed with the SEC on February 18, 2025. To the extent holdings of Parent’s or the Company’s securities by their respective directors or executive officers have changed since the amounts set forth in such proxy statements, such changes have been or will be reflected on Initial Statements of Beneficial Ownership on Form 3 or Statements of Change in Ownership on Form 4 filed with the SEC. Investors and stockholders of the Company are or will be able to obtain these documents free of charge from the SEC’s website at www.sec.gov, from Parent on Parent’s website at www.jnj.com, from the Company on the Company’s website at www.intracellulartherapies.com or on request from Parent or the Company.

On February 18, 2025 Cartesian Therapeutics, Inc. (NASDAQ: RNAC) (the "Company"), a clinical-stage biotechnology company pioneering mRNA cell therapies for autoimmune diseases, reported that its management expects to participate in the following investor conferences in February and March (Press release, Cartesian Therapeutics, FEB 18, 2025, View Source [SID1234650345]):

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!

A fireside chat at the H.C. Wainright & Co. 3rd Annual Cell Therapy Virtual Conference at 1:00 p.m. ET on Tuesday, February 25, 2025
A presentation at the TD Cowen 45th Annual Health Care Conference at 1:50 p.m. ET on Monday, March 3, 2025 in Boston, MA
A fireside chat at the Leerink Global Healthcare Conference at 8:40 a.m. ET on Monday, March 10, 2025 in Miami, FL
A live webcast of the presentation and fireside chats is expected to be accessible in the Events section of the Company’s website at www.cartesiantherapeutics.com, where an archived replay of the events will also be available for a limited time.