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GENFIT to receive US$20M milestone after Ipsen’s Iqirvo® exceeds the US$200M threshold in its first full year of net sales

On February 12, 2026 GENFIT (Euronext: GNFT), a biopharmaceutical company dedicated to improving the lives of patients with rare and life-threatening liver diseases, reported that the strong commercial performance of Iqirvo in its first full year on the primary biliary cholangitis (PBC) market has triggered a US$20M milestone payment from Ipsen under the companies’ licensing agreement.

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The commercial success of Iqirvo in 2025 underscores the strength of our partnership with Ipsen and exceeded our initial expectations with first-year net sales reaching US$208M. This performance activated a US$20M milestone payment ahead of plan, reinforcing GENFIT’s financial position. This momentum also led to the receipt of an additional €30M tranche under GENFIT’s royalty‑financing agreement with HCRx, enhancing financial flexibility in a non-dilutive way.

In parallel, Ipsen confirmed the initiation of the first and only global Phase 3 clinical trial in primary sclerosing cholangitis (PSC), addressing a significant unmet medical need, as no approved therapies currently exist for this severe and progressive disease. PSC represents a substantial untapped market opportunity, comparable in size to second line PBC. Should Iqirvo ultimately receive regulatory approval for this indication, GENFIT would be eligible for additional milestone payments as well as additional double‑digit royalties.

Beyond Iqirvo, GENFIT continues to advance its oncology program. The ongoing Phase 1b study in cholangiocarcinoma (CCA), evaluating GNS561 in combination with a MEK inhibitor, is progressing in line with expectations. As communicated in December 2025, early positive activity was observed in the first evaluable patients at Week 6. Enrollment for a the next escalated-dose cohort has now been completed, with no dose-limiting toxicities (DLTs) reported to date. Recruitment for the following one will now proceed, consistent with the predefined dose‑escalation process. Multi-cohort readouts on safety, tolerability and activity along with the determination of the recommended Phase 2 doses remain expected by the end of the first half of 2026.

Pascal Prigent, CEO of GENFIT declared: "We are pleased with Ipsen’s continued success with Iqirvo. Net sales have surpassed US$200M in 2025 and this remarkable performance has allowed us to get the first commercial milestone a year earlier than anticipated. We believe that this commercial trajectory has the potential to be transformative for our business model. We are also pleased with the progress of our GNS561 program, with a new cohort fully recruited. This program might bring significant hope for patients with a devastating disease, and we are eagerly awaiting new data. As we are also anticipating the first ACLF patients to be included in a phase 2 evaluating G1090N in the second half of the year we believe 2026 will be a landmark year for GENFIT."

GENFIT will publish its 4Q25 revenue and cash position on February 26, 2026.

(Press release, Genfit, FEB 12, 2026, https://ir.genfit.com/news-releases/news-release-details/genfit-receive-us20m-milestone-after-ipsens-iqirvor-exceeds [SID1234662638])

CRISPR Therapeutics Provides Business Update and Reports Fourth Quarter and Full Year 2025 Financial Results

On February 12, 2026 CRISPR Therapeutics (Nasdaq: CRSP) reported financial results for the fourth quarter and full year ended December 31, 2025.

"As we close out the fourth quarter, CRISPR Therapeutics continues to make steady progress across a broad and increasingly mature pipeline," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "We made meaningful advances across multiple clinical and preclinical programs, including encouraging data from zugo-cel in autoimmune disease and oncology, continued global uptake of CASGEVY, and important developments across our in vivo liver editing portfolio, and momentum in our siRNA collaboration with Sirius Therapeutics. At the same time, we continue to strengthen our platform capabilities to support long-term value creation. Together, these developments reflect continued execution across the portfolio."

Recent Highlights and Outlook

Hemoglobinopathies and CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY is approved in the U.S., EU, Great Britain, Canada, Switzerland, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain, Qatar, the United Arab Emirates (UAE), and Kuwait for patients 12 years and older with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).
CASGEVY generated fourth quarter 2025 revenue of $54 million and full year 2025 revenue of $116 million, reflecting continued growth in treated patients. 64 patients received infusions of CASGEVY during the year, including 30 in the fourth quarter. Globally, 147 people with SCD or TDT initiated the treatment process with their first cell collection during the year.
Patient initiations and first cell collections increased nearly three-fold in 2025 compared to 2024, with continued momentum going into 2026.
Access to CASGEVY continued to expand across key markets. As of year-end, approximately 90% of patients in the U.S. have reimbursed access to CASGEVY. CASGEVY is also reimbursed in the U.K., Italy, Austria, Denmark, Luxembourg, KSA, the Kingdom of Bahrain, the UAE, and Kuwait. Most recently, in January, Vertex secured reimbursed access for eligible patients with SCD in Scotland, consistent with the prior reimbursement agreement reached in 2025 for patients with TDT.
At the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2025, positive pediatric data from the pivotal studies in children ages 5-11 years with SCD or TDT were presented and featured in the "Best of ASH (Free ASH Whitepaper)" program. Global regulatory submissions in this age group are expected to begin in the first half of 2026. The U.S. Food and Drug Administration (FDA) awarded a Commissioner’s National Priority Voucher for this pediatric submission, supporting an expedited review once submissions are complete.
CRISPR Therapeutics continues to advance its in vivo hematopoietic stem cell editing approach utilizing lipid nanoparticle (LNP)-mediated delivery through preclinical studies. This initiative has the potential to expand the addressable patient populations for SCD and TDT.

In Vivo Liver Editing

CRISPR Therapeutics continues to advance a diversified portfolio of in vivo gene editing programs leveraging its proprietary LNP delivery platform.

The Company continues to advance CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), in Phase 1b clinical trials, prioritizing development in severe hypertriglyceridemia (sHTG) and refractory hypercholesterolemia. The Company expects to provide an update in the second half of 2026.
Building on insights from CTX320, the Company is advancing its next-generation LPA program, CTX321. CTX321 incorporates an updated guide RNA that demonstrates approximately two-fold greater potency in preclinical testing while utilizing the same LNP delivery system. CTX321 is currently in IND/CTA-enabling studies, with an Lp(a) program update expected in 2026.
CRISPR Therapeutics continues to advance several preclinical in vivo gene editing candidates, including:

CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), is the first investigational candidate to emerge from the Company’s SyNTase editing platform. The Company expects to initiate a clinical trial for CTX460 in mid-2026.
CTX340, targeting angiotensinogen (AGT) for refractory hypertension, is currently in IND/CTA-enabling studies. The Company expects to initiate a clinical trial for CTX340 in the first half of 2026.

siRNA-based Programs

CRISPR Therapeutics’ small interfering RNA (siRNA)-based portfolio includes clinical-stage programs in cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics.

CTX611 (SRSD107), a long-acting siRNA targeting Factor XI (FXI), is in an ongoing Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). The Company expects to provide an update in the second half of 2026.
The program has the potential to target a range of thromboembolic and clotting-related indications and represents a multi-billion-dollar market opportunity, including atrial fibrillation (AF), venous thromboembolism (VTE), ischemic stroke, cancer-associated thrombosis (CAT), thrombosis in chronic kidney disease (CKD), peripheral vascular disease (PVD), chronic coronary artery disease (CAD). CRISPR Therapeutics is expected to lead global Phase 3 development of CTX611 with Sirius Therapeutics responsible for development activities in greater China.
CRISPR Therapeutics has the option to nominate up to two siRNA targets for research and development and expects to provide an update in 2026. For each target, CRISPR Therapeutics will fund research and retain opt-in rights to lead clinical development and commercialization. Sirius will be eligible to receive milestone payments and contingent payments, as well as tiered royalties.

Autoimmune Disease and Immuno-Oncology

Zugocabtagene geleucel (zugo-cel; formerly CTX112) continues to advance in both autoimmune disease and hematologic malignancies.

In autoimmune disease, Phase 1 clinical trials of zugo-cel are ongoing across multiple indications, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis and a second Phase 1 trial in immune thrombocytopenia purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA), with continued progress across studies. The Company expects to provide updates in the second half of 2026.
In immuno-oncology, the Phase 1/2 clinical trial of zugo-cel in B-cell malignancies is ongoing. The Company expects to provide updates in the second half of 2026. Zugo-cel is also being evaluated in combination with pirtobrutinib in aggressive B-cell lymphomas under an existing collaboration with Lilly.
CRISPR Therapeutics continues to leverage its proprietary lipid nanoparticle (LNP) delivery platform, mRNA, and conjugation capabilities to advance its in vivo CAR-T platform with potential applications in autoimmune disease and oncology.
CRISPR Therapeutics’ autoimmune disease and immuno-oncology platforms are supported by a wholly-owned, GMP manufacturing facility located in Framingham, Massachusetts, which provides end-to-end production capabilities for its cell therapy portfolio and supports both clinical and future commercial supply.

Regenerative Medicine

CRISPR Therapeutics continues to advance its regenerative medicine portfolio, including its efforts in diabetes. The Company is advancing CTX213, a deviceless beta cell replacement product candidate consisting of unencapsulated precursor islet cells derived from edited induced pluripotent stem cells (iPSCs), for the treatment of Type 1 diabetes. CTX213 has demonstrated compelling preclinical efficacy via direct administration and is progressing towards the clinic. The Company expects to provide additional updates as development progresses.

Upcoming Events

The Company will present at the upcoming events:

Citi’s 2026 Virtual Oncology Leadership Summit

Date: Wednesday, February 18, 2026

Time: 10:45 a.m. ET

TD Cowen 46th Annual Health Care Conference

Date: Monday, March 2, 2026

Time: 11:10 a.m. ET

Leerink Partners Global Healthcare Conference

Date: Wednesday, March 11, 2026

Time: 1:00 p.m. ET

Fourth Quarter and Full Year 2025 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $1,975.8 million as of December 31, 2025, compared to $1,903.8 million as of December 31, 2024. The increase in cash was primarily driven by proceeds from the issuance of common shares, option exercise activity and interest income, offset by operating expenses.
R&D Expenses: R&D expenses were $83.5 million for the fourth quarter of 2025, compared to $71.7 million for the fourth quarter of 2024. The increase in R&D expense was primarily driven by an increase in licensing fees.
G&A Expenses: General and administrative expenses were $18.4 million for the fourth quarter of 2025, compared to $18.1 million for the fourth quarter of 2024.
Collaboration Expense: Collaboration expense, net, was $53.7 million for the fourth quarter of 2025, compared to $10.4 million for the same period in 2024. In 2024, we exercised our option to defer specified costs under the CASGEVY program in excess of the deferral limit of $110.3 million under the amended collaboration agreement with Vertex. The year-over-year increase in collaboration expense reflects the absence of a comparable deferral in 2025.
Net Loss: Net loss was $130.6 million for the fourth quarter of 2025, compared to a net loss of $37.3 million for the fourth quarter of 2024.

About CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world.

(Press release, CRISPR Therapeutics, FEB 12, 2026, View Source [SID1234662637])

CORMEDIX TO PARTICIPATE IN UPCOMING INVESTOR CONFERENCES

On February 12, 2026 CorMedix Therapeutics (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for life-threatening diseases and conditions, reported that senior management will be presenting and participating in investor meetings at upcoming investor conferences taking place in March.

Leerink Partners Global Healthcare Conference
Date: March 10, 2026
Time: 1:00pm Eastern
Format: Fireside Chat
Webcast: Link to webcast

Citizens Life Sciences Conference
Date: March 11, 2026
Time: 9:35am Eastern
Format: Fireside Chat
Webcast: Link to webcast

Barclays 28th Annual Global Healthcare Conference
Date: March 12, 2026
Format: One-on-one investor meetings

(Press release, CorMedix, FEB 12, 2026, View Source [SID1234662636])

AdvanCell and GlycoNet-supported startup 48Hour Discovery announce collaboration and exclusive licensing agreement

On February 12, 2026 AdvanCell, a clinical-stage radiopharmaceutical company developing innovative targeted alpha therapies for cancer, and 48Hour Discovery, a peptide discovery platform company and GlycoNet-supported startup, reported a collaboration and exclusive licensing agreement to develop a novel peptide-based Lead-212 (²¹²Pb) radioligand therapy program for oncology.

The collaboration is focused on the development of a purpose-built ²¹²Pb-targeted radioligand therapy, including a peptide ligand optimized for delivery of AdvanCell’s lead-212 (²¹²Pb) alpha-emitting payload. While centered on a 212Pb targeted RLT, the work also serves as a proof point for the broader applicability of 48Hour Discovery’s peptide discovery engine in radioligand therapy (RLT) development, demonstrating its ability to generate ligands compatible with targeted alpha delivery.

Precision Alpha Therapy, Enabled by Rapid Peptide Discovery
Lead-212 is a potent alpha-emitting radionuclide that enables localized, high-energy radiation delivery while minimizing damage to surrounding healthy tissue. AdvanCell has built an integrated platform around ²¹²Pb spanning isotope supply, manufacturing, and clinical development.

48Hour Discovery’s platform—combining large phage display libraries, high-throughput screening, and AI and chemical optimization—has been designed to rapidly generate peptides with high affinity and specificity. This program highlights the platform’s capacity to meet the stringent requirements of RLT development, including selectivity, PK and biodistribution properties, stability and payload compatibility.

"This collaboration reflects the strength of our ²¹²Pb platform and our ambition to translate scientific innovation into novel targeted alpha therapies that can meaningfully change outcomes for patients," said Philina Lee, PhD, CEO of AdvanCell. "It also demonstrates how innovative discovery technologies can be applied to accelerate the development of highly targeted alpha therapies."

Rick Finnegan, CEO of 48Hour Discovery, added:
"We see this program as an important validation of our discovery engine in the radioligand therapy space. It underscores the broader utility of our platform for generating ligands suited to demanding oncology applications. Importantly, this agreement is one of several partnerships we have entered into recently with companies developing radioligand therapies, and together they highlight the versatility of our platform across this rapidly growing modality."

(Press release, GlycoNet, FEB 12, 2026, View Source;utm_medium=rss&utm_campaign=advancell-and-glyconet-supported-startup-48hour-discovery-announce-collaboration-and-exclusive-licensing-agreement [SID1234662635])

BioMarin Announces Closing of Private Offering of Senior Notes

On February 12, 2026 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) ("BioMarin") reported that it closed its previously announced offering of $850 million of 5.500% senior unsecured notes due 2034 (the "Notes"). The issue price of the Notes is 100.000%.

BioMarin intends to use the net proceeds from the offering of the Notes, together with borrowings under a new $2 billion senior secured term loan "B" facility (the "Term Loan B Facility") and $800 million senior secured term loan "A" facility (the "Term Loan A Facility" and, together with the Term Loan B Facility, the "Term Facilities") and cash on hand, to fund the consideration payable in connection with the pending acquisition (the "Acquisition") of Amicus Therapeutics, Inc. ("Amicus") and related fees and expenses in connection with the Acquisition and borrowings under the Term Facilities and the offering of the Notes. In addition to the Term Facilities, BioMarin expects to enter into a $600 million senior secured revolving credit facility connection with the Acquisition (the "New Revolving Facility" and, together with the Term Facilities, the "New Senior Secured Credit Facilities"). BioMarin may borrow up to $150 million under the New Revolving Facility to pay such fees and expenses.

Gross proceeds from the offering of the Notes were deposited into an escrow account at the closing of the offering, pending consummation of the Acquisition. In the event that the Acquisition is not completed on or prior to December 19, 2026, or upon the occurrence of certain other events, BioMarin will be required to redeem all of the Notes at a redemption price equal to 100% of the initial issue price of the Notes plus accrued and unpaid interest from the date of issuance, or the most recent date to which interest has been paid or provided for, to but excluding the special mandatory redemption date.

The Notes are jointly and severally guaranteed by certain of BioMarin’s subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities, including, after the closing of the Acquisition, Amicus and certain of its subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities.

The indenture governing the Notes contains customary covenants that, among other things, restrict, with certain exceptions, the ability of each of BioMarin and its subsidiaries to incur additional debt, pay dividends, make certain other restricted payments, incur debt secured by liens, dispose of assets, engage in consolidations and mergers or sell or transfer all or substantially all of its assets.

The Notes have not been, and will not be, registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other securities laws and may not be offered or sold in the United States absent an effective registration statement or an applicable exemption from the registration requirements of or in a transaction not subject to the Securities Act and any state or other applicable securities laws. Accordingly, the Notes were offered and sold only to a limited number of persons who were either (1) reasonably believed to be "qualified institutional buyers" as defined in Rule 144A under the Securities Act or (2) non-U.S. persons outside the United States pursuant to Regulation S under the Securities Act. The Notes are subject to restrictions on transferability and resale and may not be transferred or resold except in compliance with the registration requirements of the Securities Act or pursuant to an exemption therefrom and in compliance with any state or other applicable securities laws.

This press release is for information purposes only and is not intended to and does not constitute, or form part of, an offer, invitation or the solicitation of an offer or invitation to purchase, otherwise acquire, subscribe for, sell or otherwise dispose of any securities, or the solicitation of any vote or approval in any jurisdiction, pursuant to the proposed transaction or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law.

(Press release, BioMarin, FEB 12, 2026, View Source [SID1234662634])