Main Reading Room
(Library 1 ―  MRR)

First approval ... 2

Exdensur (depemokimab) approved by U.S. FDA for treatment of severe eosinophilic asthma (GSK, December 16, 2025)

• The U.S. FDA approved GSK’s Exdensur (depemokimab), the first and only ultra–long-acting biologic with twice-yearly dosing, as an add-on maintenance therapy for adults and adolescents (≥12 years) with severe asthma characterized by an eosinophilic phenotype. Approval was based on the Phase III SWIFT-1 and SWIFT-2 trials, where depemokimab demonstrated sustained efficacy, reducing annualized asthma exacerbations by 58% and 48% versus placebo over 52 weeks, in addition to showing lower rates of exacerbations requiring hospitalization or emergency care. Approximately two million Americans live with severe asthma, and around half continue to experience frequent exacerbations, underscoring the need for novel, durable treatment options such as Exdensur.

Blujepa (gepotidacin) approved by U.S. FDA as an oral option for uncomplicated urogenital gonorrhea (GSK, December 11, 2025)

• The U.S. FDA has approved gepotidacin (Blujepa) as an oral treatment option for adults and adolescents aged 12 years and older (≥45 kg) with limited or no alternative options for uncomplicated urogenital gonorrhea caused by susceptible Neisseria gonorrhoeae. This approval offers patients an alternative to the current standard injectable therapy. Gepotidacin is the first new class of antibiotic approved for gonorrhea in more than 30 years. Gonorrhea remains a major public health concern and a World Health Organization priority pathogen, with over 600,000 U.S. cases reported in 2023 and continued need for new treatment options. Gepotidacin had previously received FDA approval earlier in 2025 as an oral therapy for uncomplicated urinary tract infections in females.

Additional approval ... 11

Sanofi’s Wayrilz Approved in the EU as the First BTK Inhibitor for Immune Thrombocytopenia (December 23, 2025)

• The European Commission approved Sanofi’s Wayrilz (rilzabrutinib), a novel oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, for adult patients with immune thrombocytopenia (ITP) who are refractory to other treatments. Wayrilz acts through multi-immune modulation to address underlying immune dysregulation in ITP, beyond platelet restoration alone. Approval was based on the Phase 3 LUNA 3 trial, which demonstrated rapid and durable platelet responses and improvements in ITP symptoms. 

Wegovy® Pill Approved in the U.S. as First Oral GLP-1 Therapy for Weight Management (Novo Nordisk, December 22, 2025)

• The U.S. FDA has approved the once-daily oral Wegovy® pill (semaglutide 25 mg) as the first oral GLP-1 receptor agonist for weight management, indicated to reduce excess body weight, support long-term weight maintenance, and lower the risk of major adverse cardiovascular events. Approval was supported by results from the OASIS and SELECT programs, including the OASIS 4 trial, where oral semaglutide achieved 16.6% mean weight loss—comparable to injectable Wegovy® 2.4 mg—with one-third of participants achieving ≥20% weight loss. The safety and tolerability profile was consistent with prior semaglutide studies. Novo Nordisk plans to launch the Wegovy® pill in the U.S. in early January 2026.

Lunsumio VELO™ (mosunetuzumab) Approved by U.S. FDA as a New Subcutaneous Option for Relapsed or Refractory Follicular Lymphoma (CD20, Roche, December 22, 2025)

• The U.S. FDA approved Roche’s CD20xCD3 bispecific antibody Lunsumio VELO™ as a subcutaneous formulation for adults with relapsed or refractory follicular lymphoma after at least two prior systemic therapies. Delivered in about one minute instead of the previous 2–4 hour infusion, the new formulation significantly shortens administration time while offering a treatment approach that can better align with clinical needs and patient preferences. Approval was granted under the FDA’s accelerated approval pathway, supported by compelling complete response rates from the Phase I/II GO29781 study in this difficult-to-treat third-line or later setting; continued approval may depend on confirmatory trial data.

U.S. FDA Approves JASCAYD® (nerandomilast) for the Treatment of Progressive Pulmonary Fibrosis in Adults (Boehringer Ingelheim, December 19, 2025)

• The U.S. FDA approved Boehringer Ingelheim’s JASCAYD® (nerandomilast) tablets for adult patients with progressive pulmonary fibrosis (PPF), marking the first and only approved preferential PDE4B inhibitor with immunomodulatory and antifibrotic effects for this indication. The decision was supported by the Phase III FIBRONEER™-ILD trial, the largest PPF program to date, which demonstrated that nerandomilast significantly slowed lung function decline versus placebo, with comparable permanent discontinuation rates. PPF affects up to 100,000 people in the U.S. and up to 5.6 million globally and is associated with underlying interstitial lung diseases such as autoimmune ILD and hypersensitivity pneumonitis. This is the second FDA-approved indication for JASCAYD, following idiopathic pulmonary fibrosis.

AstraZeneca announced that Saphnelo (anifrolumab) has been approved in the European Union for subcutaneous self-administration as a pre-filled pen for adult patients with systemic lupus erythematosus (SLE) receiving standard therapy (AstraZeneca, December 16, 2025). 

• The approval is supported by positive Phase 3 TULIP-SC trial results, in which subcutaneous administration demonstrated statistically significant and clinically meaningful reductions in disease activity versus placebo while maintaining a safety profile consistent with previous findings. This convenient SC option has the potential to expand access while providing comparable clinical benefit to the IV formulation. 

AstraZeneca and Daiichi Sankyo announced that Enhertu (trastuzumab deruxtecan) in combination with pertuzumab has been approved in the United States as the first new 1st-line treatment in a decade for adults with unresectable or metastatic HER2-positive breast cancer (December 15, 2025). 

• The approval, granted under Priority Review and following Breakthrough Therapy Designation, is based on the Phase 3 DESTINY-Breast09 trial, in which Enhertu plus pertuzumab reduced the risk of disease progression or death by 44% versus THP (taxane + trastuzumab + pertuzumab), with a median progression-free survival of 40.7 months compared with 26.9 months for THP. The PFS benefit was consistent across patient subgroups. 

EMA Issues Positive Opinion for Higher 7.2 mg Dose of Wegovy® to Support Greater Weight Loss in Obesity (GLP-1, Novo, December 12, 2025)

• The European Medicines Agency’s CHMP issued a positive opinion recommending approval of a higher Wegovy® (semaglutide 7.2 mg) dose, moving it closer to availability in the EU. In the STEP UP clinical program, adults with obesity (without diabetes) receiving Wegovy® 7.2 mg achieved an average 20.7% weight loss at 72 weeks, with approximately one in three patients reaching ≥25% weight reduction. The higher dose maintained a safety and tolerability profile consistent with the already approved 2.4 mg dose and preserved muscle function while primarily reducing fat mass. The clinical program also reinforced Wegovy’s established benefits in obesity-related complications, including cardiovascular risk reduction and improvements in osteoarthritis-related knee pain.

GSK’s RSV vaccine Arexvy receives positive CHMP opinion for use in all adults aged 18 years and older (December 12, 2025)

• GSK announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the indication of its adjuvanted recombinant RSV vaccine, Arexvy, to include all adults aged 18 years and older. A final decision from the European Commission is expected in February 2026. If approved, the expanded indication will make Arexvy available for broad adult protection against RSV across the EU. Arexvy is already approved in Europe for adults aged 60 and older, and for adults aged 50–59 at increased risk of RSV disease. RSV leads to an estimated 158,000 adult hospitalizations annually in the EU, highlighting the importance of expanded vaccination options.

AKEEGA® Approved by U.S. FDA as First Precision Therapy for BRCA2-Mutated Metastatic Castration-Sensitive Prostate Cancer, Showing 54% Reduction in Disease Progression vs Standard of Care (PARP, Johnson & Johnson, December 12, 2025)

• The U.S. FDA approved the supplemental New Drug Application for AKEEGA® (niraparib and abiraterone acetate dual-action tablet) plus prednisone for the treatment of patients with BRCA2-mutated metastatic castration-sensitive prostate cancer (mCSPC), establishing the first precision medicine combination therapy approved for this population. Approval was supported by the Phase 3 AMPLITUDE trial, in which AKEEGA® plus prednisone and androgen deprivation therapy (ADT) reduced the risk of radiographic progression or death by 54% versus placebo/abiraterone acetate plus prednisone and ADT, and prolonged time to symptomatic progression by 59%.

Breyanzi® (lisocabtagene maraleucel) approved by the U.S. FDA as the first and only CAR T cell therapy for adults with relapsed or refractory marginal zone lymphoma (MZL) (Bristol Myers Squibb, December 4, 2025).

• The approval is based on the MZL cohort of the Phase 2 TRANSCEND FL study, where Breyanzi demonstrated a 95.5% overall response rate and a 62.1% complete response rate after at least two prior lines of systemic therapy, with durable responses and a safety profile consistent with prior experience. Breyanzi is now the only FDA-approved CAR T therapy indicated across five cancer types, the broadest label among CD19-directed CAR T therapies.

Jaypirca® Receives Expanded U.S. FDA Approval for Relapsed or Refractory CLL/SLL Previously Treated with a Covalent BTK Inhibitor (Eli Lilly, December 3, 2025)

• The U.S. FDA approved an expanded indication for Jaypirca® (pirtobrutinib), the first and only non-covalent BTK inhibitor, for adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who were previously treated with a covalent BTK inhibitor. The approval is supported by the Phase 3 BRUIN CLL-321 trial, the only randomized study in CLL/SLL in which all patients had prior covalent BTK exposure. This expansion significantly broadens access to Jaypirca and aligns with NCCN guideline-endorsed populations, while converting its previous accelerated approval to full approval.

Filing & other regulatory ... 7

Sanofi Provides Update on Tolebrutinib Regulatory Submission in Non-Relapsing Secondary Progressive Multiple Sclerosis (Setback, BTK, December 24, 2025)

• Sanofi announced that the U.S. Food and Drug Administration issued a Complete Response Letter (CRL) for the new drug application for tolebrutinib in adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS). This decision represents a significant deviation from the FDA’s previously communicated interactions, and Sanofi expressed disappointment, reaffirming its commitment to working with the agency to identify a possible regulatory path forward. Tolebrutinib previously received Breakthrough Therapy Designation and holds provisional approval in the UAE; regulatory reviews continue in the EU and other regions. Sanofi also confirmed that ongoing IFRS impairment testing related to the asset will have no impact on 2025 business net income or earnings guidance.

Enhertu granted Breakthrough Therapy Designation in the US as post-neoadjuvant therapy for patients with HER2-positive early breast cancer (AstraZeneca, Daiichi Sankyo, 22 December 2025)

• AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been granted Breakthrough Therapy Designation (BTD) in the US for adult patients with HER2-positive early breast cancer with residual invasive disease in the breast and/or axillary lymph nodes after neoadjuvant treatment and high risk of disease recurrence. The FDA granted this BTD based on results from the DESTINY-Breast05 Phase III trial presented in a Presidential Symposium at the 2025 European Society for Medical Oncology (ESMO) Congress and subsequently published in The New England Journal of Medicine.

Novo Nordisk Submits NDA to U.S. FDA for CagriSema, the First Once-Weekly GLP-1 and Amylin Analogue Combination for Weight Management (December 18, 2025)

• Novo Nordisk has submitted a New Drug Application to the U.S. FDA for CagriSema, a once-weekly fixed-dose injectable combination of the amylin analogue cagrilintide (2.4 mg) and the GLP-1 receptor agonist semaglutide (2.4 mg) for adults with obesity or overweight with at least one weight-related comorbidity. In the Phase 3 REDEFINE 1 trial, CagriSema achieved an average 23% body-weight reduction when evaluating the treatment effect assuming all patients remained on therapy, demonstrating substantial weight-loss potential through complementary mechanisms of action. If approved, CagriSema would become the first combination therapy of a GLP-1 receptor agonist and an amylin analogue for chronic weight management.

DATROWAY® EU Application Validated for First-Line Treatment of Metastatic Triple-Negative Breast Cancer Not Eligible for Immunotherapy (TROP2, Daiichi Sankyo / AstraZeneca, December 18, 2025)

• The European Medicines Agency validated the Type II Variation application for DATROWAY® (datopotamab deruxtecan) as a first-line monotherapy for adults with unresectable or metastatic triple-negative breast cancer (TNBC) who are not candidates for PD-1/PD-L1 inhibitor therapy. The submission is based on results from the Phase 3 TROPION-Breast02 trial, in which DATROWAY demonstrated a statistically significant and clinically meaningful overall survival benefit versus chemotherapy—the first and only therapy to do so in this population. If approved, DATROWAY could become a new standard of care.

U.S. Food and Drug Administration (FDA) Grants Priority Review to Bristol Myers Squibb's Application for Opdivo® (nivolumab) Plus Chemotherapy Combination for Classical Hodgkin Lymphoma. (December 11, 2025)

• The sBLA is for the first-line treatment of adults and adolescents (≥12 years) with previously untreated Stage III or IV classical Hodgkin lymphoma (cHL) in combination with doxorubicin, vinblastine and dacarbazine (AVD). The submission is supported by results from the Phase 3 SWOG S1826 trial, which evaluated Opdivo + AVD in newly diagnosed advanced-stage cHL and positions the combination as a potential new standard of care aimed at improving early and durable treatment outcomes.

TECVAYLI®＋DARZALEX FASPRO® Receives U.S. FDA Breakthrough Therapy Designation as a Potential Second-Line Standard of Care in Relapsed/Refractory Multiple Myeloma (BCMA, Johnson & Johnson, December 9, 2025)

• New data from the Phase 3 MajesTEC-3 trial showed that the investigational combination of TECVAYLI® (teclistamab) plus DARZALEX FASPRO® (daratumumab and hyaluronidase) achieved an 83% reduction in the risk of disease progression or death compared with standard treatment regimens in patients with relapsed/refractory multiple myeloma (RRMM) after nearly three years of follow-up (HR 0.17; 95% CI 0.12–0.23; p<0.0001). Overall survival was also significantly improved, and 91% of patients who were progression-free at six months remained progression-free at three years. 

AstraZeneca announced that the U.S. Food and Drug Administration has accepted the New Drug Application (NDA) for baxdrostat under Priority Review for the treatment of adults with hard-to-control (uncontrolled or treatment-resistant) hypertension as an add-on therapy when current antihypertensive medicines are insufficient ( December 2, 2025)

• The submission is supported by results from the Phase 3 BaxHTN trial, presented at ESC 2025 and published in the New England Journal of Medicine, which demonstrated statistically significant and clinically meaningful reductions in systolic blood pressure. At 12 weeks, baxdrostat achieved placebo-adjusted reductions of 9.8 mmHg (2 mg) and 8.7 mmHg (1 mg), with consistent effects in both uncontrolled and treatment-resistant hypertension subgroups. If approved, baxdrostat could become the first aldosterone synthase inhibitor to receive regulatory approval. 

Pivotal studies ... 12

DESTINY-Endometrial02 Phase 3 Trial of ENHERTU® Launched as Adjuvant Therapy in HER2-Expressing Endometrial Cancer (Daiichi Sankyo / AstraZeneca, December 22, 2025)

• Daiichi Sankyo and AstraZeneca announced initiation of the Phase 3 DESTINY-Endometrial02 tr, with the first patient dosed. The study is evaluating ENHERTU® (trastuzumab deruxtecan) with or without radiotherapy versus standard chemotherapy with or without radiotherapy as adjuvant treatment following surgery in patients with HER2-expressing (IHC 3+/2+) endometrial cancer. The trial is being conducted in collaboration with GOG Foundation and ENGOT, with GINECO as the lead ENGOT group, aiming to determine whether ENHERTU can improve postoperative outcomes in this high-risk population.

Otsuka Initiates Global Phase 3 Trial of Repinatrabit for Phenylketonuria (December 19, 2025)

• Otsuka announced the start of a global Phase 3 clinical trial (PheORD) evaluating repinatrabit (JNT-517), an investigational oral small-molecule therapy for phenylketonuria (PKU). The randomized study will assess efficacy, safety, and tolerability of twice-daily repinatrabit in patients with PKU. The therapy has received U.S. FDA orphan drug and rare pediatric disease designations. PKU, a rare genetic disorder affecting approximately 1 in 24,000 individuals worldwide, leads to elevated phenylalanine levels that can cause severe neurodevelopmental impairment if untreated. Despite dietary therapy being the current standard of care, many patients face challenges maintaining adequate control, underscoring the need for additional treatment options.

Zasocitinib Shows Strong Phase 3 Efficacy, Delivering Clear Skin with Once-Daily Oral Therapy in Moderate-to-Severe Plaque Psoriasis (TYK2, Takeda, December 18, 2025)

• Takeda announced positive topline results from two pivotal Phase 3 trials of zasocitinib (TAK-279), a highly selective oral TYK2 inhibitor, in adults with moderate-to-severe plaque psoriasis. The studies met all primary and ranked secondary endpoints. By week 16, more than half of patients achieved PASI 90 (clear or almost clear skin), and approximately 30% achieved PASI 100 (complete clearance), with improvements observed as early as week 4 and increasing through week 24. Zasocitinib demonstrated superiority versus placebo and apremilast, and was generally well tolerated with a safety profile consistent with previous trials. These results highlight the potential of once-daily oral zasocitinib to provide a highly effective, convenient treatment option and help catalyze a new era in psoriasis care.

Orforglipron Enables Maintenance of Weight Loss After Switching from Injectable Incretins to Oral GLP-1 Therapy in First-of-its-Kind Phase 3 Trial (Eli Lilly, Chugai, December 18, 2025)

• In the Phase 3 ATTAIN-MAINTAIN study, orforglipron, an investigational once-daily oral GLP-1 receptor agonist, met the primary and all key secondary endpoints by maintaining weight loss over 52 weeks in participants who previously achieved significant weight reduction on Wegovy or Zepbound. Participants switching from Wegovy maintained nearly all previously achieved weight loss (average difference 0.9 kg), while those switching from Zepbound maintained weight loss with an average difference of 5.0 kg. These findings support orforglipron as a potential oral maintenance therapy following injectable incretin treatment, and Eli Lilly has submitted the therapy to the U.S. FDA for obesity treatment approval.

KEYTRUDA®＋Padcev® Demonstrate Significant Survival and Pathologic Complete Response Benefits in Cisplatin-Eligible Muscle-Invasive Bladder Cancer When Used Perioperatively (Nectin-4, Merck, Pfizer, Astellas, December 17, 2025)

• Merck reported positive topline results from the Phase 3 KEYNOTE-B15 (EV-304) trial in cisplatin-eligible patients with muscle-invasive bladder cancer (MIBC). The combination of KEYTRUDA® (pembrolizumab) and Padcev® (enfortumab vedotin-ejfv), administered before and after surgery, delivered statistically significant and clinically meaningful improvements in event-free survival, overall survival, and pathologic complete response rates compared with standard neoadjuvant chemotherapy plus surgery. This marks the first and only perioperative regimen combining immunotherapy and an antibody–drug conjugate to extend survival in this patient population. KEYTRUDA plus Padcev is also approved in the U.S. for the treatment of adult patients with MIBC who are ineligible for cisplatin-based chemotherapy.

Gilead’s Investigational Single-Tablet Regimen of Bictegravir + Lenacapavir Achieves Primary Endpoint in Phase 3 ARTISTRY-2 Trial (HIV, December 15, 2025)

• Gilead announced positive Phase 3 ARTISTRY-2 results showing that its investigational once-daily single-tablet regimen combining bictegravir and lenacapavir (BIC/LEN) was statistically non-inferior to BIKTARVY® in virologically suppressed adults with HIV. The primary endpoint—proportion of participants with HIV-1 RNA ≥50 copies/mL at Week 48—met the prespecified non-inferiority criterion. The regimen was generally well tolerated with no new safety issues identified. Together with supportive data from ARTISTRY-1, these findings will form the basis of global regulatory submissions as Gilead aims to expand simplified treatment options for maintaining durable viral suppression.

Sanofi Provides Update on Tolebrutinib in Primary Progressive Multiple Sclerosis (Setback, BTK, December 15, 2025)

• Sanofi announced results from the Phase 3 PERSEUS study in primary progressive multiple sclerosis (PPMS), reporting that tolebrutinib did not meet the primary endpoint of delaying time to six-month composite confirmed disability progression compared with placebo. Based on these findings, the company will not pursue regulatory registration for PPMS. Preliminary analysis indicated that the safety profile remained consistent with prior studies, although drug-induced liver injury (DILI) remains an identified risk requiring careful monitoring and management. Full efficacy and safety data will be presented at an upcoming medical congress, and Sanofi reaffirmed its continued commitment to advancing treatment options for progressive forms of multiple sclerosis.

Retatrutide Achieves Up to 28.7% Weight Loss and Significant Osteoarthritis Pain Reduction in Phase 3 TRIUMPH-4 Trial (GLP-1, Eli Lilly, December 11, 2025)

• In the Phase 3 TRIUMPH-4 study, Eli Lilly’s investigational triple-agonist retatrutide (GIP/GLP-1/glucagon receptor agonist) achieved unprecedented dual benefits in adults with obesity/overweight and knee osteoarthritis. At 68 weeks, participants receiving the 12 mg dose experienced an average 28.7% body weight reduction (71.2 lbs) and up to a 75.8% improvement in knee pain, as measured by WOMAC pain scores, along with significant improvements in physical function. More than one in eight patients became completely free of knee pain by study end. Retatrutide met all primary and key secondary endpoints, reinforcing its potential as a transformative therapy. Seven additional Phase 3 trials in obesity and type 2 diabetes are expected to read out in 2026.

Roche’s giredestrant Reduced Risk of Invasive Disease Recurrence or Death by 30% in ER-Positive Early-Stage Breast Cancer (December 10, 2025)

• Roche reported positive Phase 3 lidERA trial results showing that the investigational oral selective estrogen receptor degrader (SERD) giredestrant significantly reduced the risk of invasive disease recurrence or death by 30% versus standard endocrine therapy in patients with ER-positive, HER2-negative early-stage breast cancer. At three years, invasive disease-free survival reached 92.4% with giredestrant compared with 89.6% with standard therapy, with consistent benefit across key subgroups. Overall survival data are still maturing but show a favorable trend. As the first oral SERD to demonstrate superior iDFS in the adjuvant setting, giredestrant represents the most meaningful advance in endocrine therapy for early breast cancer in over two decades and may become a new standard of care.

TUKYSA Added to First-Line Maintenance Therapy Extends Progression-Free Survival by Over 8 Months in HER2+ Metastatic Breast Cancer (Pfizer, December 10, 2025)

• The Phase 3 HER2CLIMB-05 study demonstrated that adding TUKYSA® (tucatinib) to trastuzumab and pertuzumab as investigational first-line maintenance therapy after chemotherapy significantly reduced the risk of disease progression or death by 36% versus trastuzumab and pertuzumab alone (HR 0.641; p<0.0001). Median progression-free survival reached 24.9 months versus 16.3 months, extending PFS by 8.6 months, with consistent benefit across predefined subgroups including hormone receptor status, de novo vs. recurrent disease, and patients with or without brain metastases. Overall survival analysis remains immature but shows a favorable trend. The combination exhibited a manageable safety profile.

Novartis Ianalumab Significantly Prolongs Disease Control in Immune Thrombocytopenia with Only Four Monthly Doses (December 9, 2025)

• Phase 3 VAYHIT2 results showed that ianalumab (9 mg/kg) plus eltrombopag extended time to treatment failure by 45%, allowing patients to maintain disease control 2.8 times longer than placebo plus eltrombopag (13.0 vs 4.7 months). Additionally, 62% of patients achieved sustained platelet response at six months compared with 39% on placebo combination. Administered as just four once-monthly IV doses, ianalumab has the potential to provide durable disease control and reduce the need for chronic treatment in ITP. Novartis plans regulatory submission combining these data with ongoing first-line VAYHIT1 trial results in 2027.

Jaypirca® Significantly Reduces Risk of Disease Progression or Death by 80% vs. Chemoimmunotherapy in Treatment-Naïve CLL/SLL (BTK, Eli Lilly, December 9, 2025)

• Phase 3 BRUIN CLL-313 showed that Jaypirca® (pirtobrutinib), a non-covalent BTK inhibitor, significantly improved progression-free survival versus bendamustine + rituximab in previously untreated CLL/SLL without del(17p), reducing the risk of progression or death by 80% (HR 0.20; p<0.0001). Benefits were consistent across subgroups, including high-risk features such as TP53 mutations and unmutated IGHV. Overall survival trends favored Jaypirca despite crossover, with final OS analysis planned.

Jaypirca® Demonstrates Non-Inferiority—and Numerically Higher Response—vs. Imbruvica in First Head-to-Head Phase 3 Trial in CLL/SLL (BTK, Eli Lilly, December 7, 2025)

• In the Phase 3 BRUIN CLL-314 study, Jaypirca® (pirtobrutinib), a reversible BTK inhibitor, met its primary endpoint of non-inferiority to Imbruvica® (ibrutinib) for overall response rate (ORR) in treatment-naïve or BTK inhibitor-naïve CLL/SLL, achieving a numerically higher ORR (87.0% vs. 78.5%). Progression-free survival remains immature but trended in favor of Jaypirca, including a 43% reduction in risk of progression or death in the overall population and a 76% reduction in treatment-naïve patients, the subgroup with longest follow-up. These results were published in the Journal of Clinical Oncology and presented at ASH 2025.

Rusfertide Demonstrates Durable Hematocrit Control and Sustained Clinical Benefit in Polycythemia Vera in Long-Term Phase 3 Data (Takeda, December 6, 2025)

• Protagonist Therapeutics and Takeda reported new 52-week results from the Phase 3 VERIFY study in patients with polycythemia vera (PV), showing durable efficacy and safety of rusfertide. Continuous treatment maintained hematocrit control and reduced the need for therapeutic phlebotomy, with 61.9% of patients remaining free from phlebotomy eligibility through Week 52. Patients who crossed over from placebo to rusfertide after 32 weeks achieved comparable benefit, with 77.9% achieving absence of phlebotomy eligibility between Weeks 40–52. No new safety concerns emerged. Supporting long-term outcomes, four-year data from the combined REVIVE and THRIVE studies demonstrated a 13-fold reduction in annual phlebotomy rates versus baseline, reinforcing rusfertide’s potential as a meaningful addition to PV management.

Earlier Use of CARVYKTI® Shows Durable Treatment-Free Remission at 2.5 Years in Relapsed/Refractory Multiple Myeloma (CAR-T/BCMA, Johnson & Johnson, December 6, 2025)

• Updated Phase 3 CARTITUDE-4 data showed that 80% of as-treated standard-risk RRMM patients who received CARVYKTI® as early as second line remained progression-free and required no further therapy at 2.5 years. Additional translational analyses suggested that earlier intervention may be associated with better immune fitness and longer progression-free survival. These findings reinforce CARVYKTI®’s potential to deliver durable, treatment-free disease control.

Bristol Myers Squibb to Continue Phase 3 ADEPT-2 Study in Psychosis Associated with Alzheimer’s Disease Following DMC Recommendation (Cobenfy, BMS, December 3, 2025)

• Bristol Myers Squibb announced it will continue enrollment in the Phase 3 ADEPT-2 study evaluating Cobenfy for psychosis associated with Alzheimer’s disease. During a blinded internal review, the company identified execution irregularities at a small number of clinical trial sites and decided, prior to database lock, to exclude data from those sites. In agreement with the U.S. FDA, an interim independent efficacy and safety analysis was conducted and reviewed by the Data Monitoring Committee (DMC), which subsequently recommended continuing the study with additional patient enrollment. BMS remains blinded to the study data. Cobenfy, already approved for adult schizophrenia, has potential to become the first muscarinic receptor–targeting therapy for agitation and psychosis in Alzheimer’s disease.

AbbVie announced Phase 3 ECLIPSE trial results showing that atogepant (AQUIPTA®)  60 mg demonstrated superiority over placebo for the acute treatment of migraine with or without aura (December 1, 2025). 

• The study met its primary endpoint, with 24.3% of patients achieving pain freedom at two hours after treating the first migraine attack compared to 13.1% with placebo, and also achieved statistical significance across the majority of key secondary endpoints, including freedom from the most bothersome symptom at two hours. The safety profile over 24 weeks was consistent with previous atogepant preventive studies, with no new safety signals observed. AbbVie has submitted an application to the European Medicines Agency seeking approval for atogepant as an acute migraine treatment in adults across Europe.

Other R&D ... 18

Phase 2b DUPLEX-AD Study of JNJ-5939 in Moderate to Severe Atopic Dermatitis Did Not Meet Efficacy Threshold; Trial Stopped Early Following Interim Analysis (Setback, Johnson & Johnson, December 26, 2025)

• The Phase 2b DUPLEX-AD proof-of-concept study evaluating JNJ-95475939 (JNJ-5939) in patients with moderate to severe atopic dermatitis was discontinued early following a planned interim analysis. The study did not meet the predefined high efficacy criteria required to continue clinical development. JNJ-5939 was reported to be well tolerated in the study.

AstraZeneca reported that the Phase 3 LATIFY trial evaluating ceralasertib in combination with Imfinzi (durvalumab) did not meet its primary endpoint of improving overall survival compared with standard-of-care docetaxel in patients with previously treated locally advanced or metastatic non-small cell lung cancer without actionable genomic alterations (Setback, December 22, 2025). 

• The trial enrolled patients whose disease had progressed following prior immunotherapy and platinum-based chemotherapy. The combination was generally well tolerated, with a safety profile consistent with the known profiles of each agent and no new safety concerns identified. Full data will be presented at an upcoming medical meeting. 

QALSODY® demonstrates long-term benefit in SOD1-ALS in Phase 3 VALOR and open-label extension study results published in JAMA Neurology（RNAi, ALS, Biogen, December 22, 2025）

• Long-term data (over 3.5 years) showed that early initiation of QALSODY was associated with slower decline in clinical function, breathing, and muscle strength. Sustained reductions in neurofilament, a marker of neurodegeneration, further supported biological impact, and some patients regained previously lost function.

Updated Phase 3 EMBER-3 Data Reinforce Efficacy of Inluriyo™ (imlunestrant) Monotherapy and Combination with Verzenio® in ER+, HER2– Advanced Breast Cancer (Eli Lilly, December 12, 2025)

• Updated results from the Phase 3 EMBER-3 trial showed that Inluriyo™ (imlunestrant), an oral estrogen receptor antagonist, demonstrated clinically meaningful outcomes in ER+, HER2– advanced or metastatic breast cancer. As monotherapy, imlunestrant reduced the risk of disease progression or death by 38% and showed an 11.4-month numerical improvement in median overall survival versus endocrine therapy in patients with ESR1-mutated disease. In the overall study population, the combination of imlunestrant plus Verzenio® (abemaciclib) achieved a median progression-free survival of 10.9 months, showed a favorable trend in overall survival, and extended time to chemotherapy by more than one year. 

BioNTech and Bristol Myers Squibb reported the first interim data from a global randomized Phase 2 trial of pumitamig (BNT327/BMS-986545), a PD-L1 × VEGF-A bispecific antibody, in combination with chemotherapy in patients with locally advanced or metastatic triple-negative breast cancer (TNBC), regardless of PD-L1 status (December 9, 2025).

• The investigational regimen demonstrated encouraging antitumor activity and a manageable safety profile in both first-line and second-line settings. Detailed results will be presented at the San Antonio Breast Cancer Symposium (SABCS) 2025.

One in Four Metastatic Breast Cancer Patients Treated with Kisqali® Remain Progression-Free Beyond Four Years (Novartis, December 9, 2025)

• A pooled post-hoc analysis of first-line patients from the MONALEESA Phase III trials showed that 25% of individuals with HR+/HER2- advanced breast cancer remained progression-free for ≥4 years on Kisqali® (ribociclib) plus endocrine therapy. Median progression-free survival reached 6.8 years, with benefit seen regardless of age, BMI, menopausal status, or negative prognostic factors such as liver metastases or ≥3 metastatic sites. Kisqali remains the only CDK4/6 inhibitor to demonstrate statistically significant overall survival benefit across all Phase III trials in metastatic breast cancer. Additional five-year NATALEE sub-analysis data also reinforced sustained distant disease-free survival benefit in early breast cancer.

Astellas to Present New XOSPATA™ (gilteritinib) Data Across the FLT3m+ AML Disease Continuum at ASH 2025 (FLT3, December 8, 2025)

• Astellas announced that multiple new analyses of XOSPATA™ (gilteritinib) in FLT3 mutation-positive acute myeloid leukemia (FLT3m+ AML) will be presented at the 2025 ASH Annual Meeting, covering relapsed/refractory, newly diagnosed, and post-transplant maintenance settings. Presentations include a pooled post-hoc analysis from the Phase 3 ADMIRAL and COMMODORE studies assessing outcomes with post-transplant gilteritinib resumption in R/R FLT3m+ AML, as well as findings from the Phase 3 MORPHO maintenance trial and Phase 1/2 VICEROY trial in newly diagnosed disease. These data collectively explore how treatment timing, sequencing, and combination strategies may influence long-term outcomes and support continued optimization of therapy across the FLT3m+ AML continuum.

INCA033989 demonstrates rapid spleen volume reduction, symptom improvement and anemia benefit in CALR-mutated myelofibrosis, supporting disease-modifying potential（Incyte, December 7, 2025）

• Phase 1 studies showed rapid and robust spleen volume and symptom reductions, and anemia improvement with INCA033989 monotherapy and in combination with ruxolitinib. Favorable safety was observed with no dose-limiting toxicities and unreached maximum tolerated dose; decreases in mutCALR allele burden further suggest potential disease-modifying activity.

Lynozyfic™ (linvoseltamab) Monotherapy Demonstrates Strong Efficacy in Newly Diagnosed Multiple Myeloma, Supporting Potential Frontline Role (BCMA, Regeneron, December 7, 2025)

• Regeneron reported encouraging Phase 1/2 results from the LINKER-MM4 trial evaluating Lynozyfic™ (linvoseltamab), a BCMA×CD3 bispecific antibody, as monotherapy in newly diagnosed multiple myeloma (NDMM). Across all dose cohorts (50 mg, 100 mg, 200 mg), ≥70% of patients achieved very good partial response or better despite limited follow-up, with responses expected to deepen over time. Notably, 95% of evaluable VGPR+ patients achieved minimal residual disease (MRD) negativity, comparable to outcomes usually seen with intensive multi-drug regimens. These findings suggest Lynozyfic monotherapy may serve as a foundational or simplified frontline treatment option for both transplant-eligible and transplant-ineligible patients. The data were presented at the American Society of Hematology (ASH) 2025 Annual Meeting.

Yescarta® Shows Consistent Efficacy, Safety and Quality-of-Life Benefit Across Broad Relapsed/Refractory LBCL Populations in New Analysis (CAR-T, Gilead, December 7, 2025)

• Kite (a Gilead company) reported new pooled analyses from the Phase 3 ZUMA-7 and Phase 2 ALYCANTE studies showing that second-line CAR-T therapy with Yescarta® (axicabtagene ciloleucel) delivers durable efficacy, manageable safety and quality-of-life benefits in patients with relapsed/refractory large B-cell lymphoma (R/R LBCL), including those ineligible for high-dose chemotherapy and autologous stem cell transplant. The findings support earlier use of Yescarta as a potentially curative, single-infusion therapy and further reinforce CAR-T treatment as a new standard of care in the second-line setting.

CASGEVY® shows transformative efficacy and consistent safety in children aged 5–11 years with severe sickle cell disease and transfusion-dependent beta thalassemia（Gene editing, Vertex, December 6, 2025）

• New pivotal study results presented at ASH demonstrated meaningful clinical benefit of CASGEVY in children 5–11 years, consistent with durable efficacy and safety previously observed in patients aged 12 years and older. Vertex plans global regulatory submissions for the pediatric indication in the first half of 2026.

Lundbeck Reports Positive Phase 2 Long-Term Data for Bexicaserin in Rare Childhood-Onset Epilepsies at AES 2025 (Serotonin, Lundbeck, December 6, 2025)

• Lundbeck announced new long-term Phase 2 data showing that bexicaserin (LP352), an investigational therapy for seizures associated with developmental and epileptic encephalopathies (DEEs), sustained clinically meaningful seizure reduction for up to two years after treatment initiation. Durable benefits were observed across multiple DEE subtypes with a favorable safety and tolerability profile. DEEs are rare, severe childhood-onset epilepsies characterized by drug-resistant, unpredictable seizures and developmental impairment, underscoring the need for new treatment options.

Kite’s next-generation bicistronic CAR T therapies KITE-753 and KITE-363 show promising Phase 1 efficacy and safety in relapsed/refractory B-cell lymphoma (Gilead, December 6, 2025)

• Kite reported encouraging Phase 1 results for its investigational bicistronic CAR T-cell therapies KITE-753 and KITE-363 in relapsed／refractory large B-cell lymphoma. Both candidates target CD19 and CD20 and incorporate dual co-stimulatory domains (CD28 and 4-1BB) using the KITE DuoCore™ construct, designed to enhance durability, safety and resistance to antigen escape. Among CAR-naïve patients treated with KITE-753 at dose level 3, 79% achieved complete response, with robust CAR T expansion despite a relatively low cell dose. Safety was favorable with no dose-limiting toxicities and no Grade ≥3 CRS or ICANS at dose level 3. Results support continued development of these next-generation CAR T platforms aimed at improving efficacy, tolerability and accessibility of cell therapy.

Kite’s investigational CAR T therapy anitocabtagene autoleucel (anito-cel) demonstrated deep, durable responses in relapsed or refractory multiple myeloma in the pivotal Phase 2 iMMagine-1 study (Gilead, December 6, 2025)

• Updated data from the Phase 2 iMMagine-1 study showed anito-cel achieved a 96% overall response rate and 74% stringent complete/complete response rate at a median 15.9-month follow-up, with 95% of evaluable patients reaching minimal residual disease negativity. Progression-free survival rates were 82.1% at 12 months and 61.7% at 24 months, while overall survival rates reached 94% at 12 months and 83% at 24 months, with medians not yet reached. Responses deepened over time and appeared rapid in many patients despite high-risk and heavily pretreated characteristics. Safety remained predictable and manageable with no delayed neurotoxicity signals such as Parkinsonism or immune effector cell–associated enterocolitis reported across Phase 1/2 programs. These results support an anticipated U.S. launch in 2026, pending regulatory review.

HYMPAVZI® (marstacimab) Demonstrates 93% Reduction in Bleeds vs. On-Demand Therapy in Phase 3 BASIS Trial for Hemophilia A or B With Inhibitors (TFPI, Pfizer, December 6, 2025)

• Phase 3 BASIS trial data presented at the American Society of Hematology (ASH) 2025 Meeting and published in Blood showed that HYMPAVZI® (marstacimab) achieved a 93% reduction in treated annualized bleeding rate compared with prior on-demand bypassing therapy in adults and adolescents with hemophilia A or B with inhibitors. The therapy is administered once weekly via subcutaneous injection, offering a simplified regimen without the need for treatment-related laboratory monitoring. These findings reinforce HYMPAVZI as a superior prophylactic approach over on-demand treatment for this high-risk hemophilia population.

Zorevunersen shows durable seizure reduction and functional improvement in Dravet syndrome, supporting disease-modifying potential（RNAi, Biogen, December 5, 2025）

• Phase 1/2a and OLE results demonstrated sustained reductions in seizures, increased seizure-free days, and improvements in cognition, behavior and quality of life on top of standard anti-seizure medicines. Propensity-score weighted comparison with natural history data also showed consistent benefit, aligned with ongoing Phase 3 EMPEROR outcomes.

Eisai Presents New Data on the Continued and Expanding Benefit of LEQEMBI® (lecanemab-irmb) in Early Alzheimer’s Disease at CTAD 2025 (Biogen, December 4, 2025)

• Eisai and Biogen presented new findings at CTAD 2025 showing that continued maintenance treatment with LEQEMBI® (lecanemab) may substantially delay Alzheimer’s disease progression. Long-term modeling suggested that, in patients with early Alzheimer’s disease and low amyloid burden who began treatment at the mild cognitive impairment stage, disease progression to moderate Alzheimer’s could potentially be delayed by up to 8.3 years. Additional safety and efficacy data were also shared on the subcutaneous formulation: already approved in the U.S. for maintenance therapy, it is currently under U.S. regulatory review for initiation treatment and has been filed in Japan.

Bexicaserin (LP352) to present comprehensive new dataset for rare childhood-onset epilepsies at the 2025 American Epilepsy Society (AES) Annual Meeting, highlighting early and sustained seizure reduction in patients with developmental and epileptic encephalopathies (DEEs)（Lundbeck, December 2, 2025）

• New analyses from the Phase 1b/2a PACIFIC trial and its open-label extension indicate early seizure frequency reductions as soon as two weeks after initiation, sustained over the long term. Bexicaserin is being developed to treat seizures associated with Developmental and Epileptic Encephalopathies (DEEs), a group of severe childhood-onset epilepsies often resistant to conventional antiseizure medications. The data further highlight the significant unmet medical need in DEEs, where few approved therapies exist for most subtypes.

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