Table of Contents
Leo Pharma Announces Positive Results of Phase III Hand Eczema Clinical Trial
LEO Pharma A/S, a global leader in medical dermatology, announced that the DELTA 2 trial yielded positive results. DELTA 2 is the second of two pivotal phase III clinical trials involving delgocitinib cream, an investigational topical pan-Janus kinase (JAK) inhibitor, for the treatment of adults with moderate to severe chronic hand eczema (CHE).
The trial’s primary endpoint was met with a statistically significant improvement in CHE after 16 weeks of treatment with delgocitinib cream versus cream vehicle, and the treatment was well tolerated. When compared to subjects treated with cream vehicle, patients treated with delgocitinib cream had a significantly higher proportion of their CHE signs and symptoms cleared early in the treatment period. DELTA 2 is the second phase III trial to achieve all key secondary endpoints and confirm the positive results of DELTA 1.
“It’s incredibly exciting to see the level of consistency that our DELTA 2 results show, especially in light of the positive DELTA 1 results announced late last year,” said Jörg Möller, Executive Vice President, Global Research & Development, LEO Pharma. “We know that CHE can have a significant negative impact on a patient’s quality of life, physical functioning, and ability to work. These findings move us one step closer to establishing delgocitinib as a best-in-class innovative topical treatment for patients suffering from this difficult-to-treat disease.”
Subjects who completed 16 weeks of treatment in DELTA 1 or DELTA 2 trials were invited to participate in the DELTA 3 extension trial to assess the long-term effects of delgocitinib cream. 2 The data set will be further analyzed to determine the full potential of delgocitinib cream in the treatment of adults with moderate to severe CHE. DELTA 2 detailed results will be submitted for scientific presentation and publication at a later date.
FDA Approves GSK’s PD-1 inhibitor Jemperli
GSK plc announced that the US Food and Drug Administration (FDA) had granted full approval to Jemperli (dostarlimab-gxly) for the treatment of adult patients with mismatch repair-deficient (dMMR) recurrent or advanced endometrial cancer, as determined by a US FDA-approved test, who have progressed on or after a prior platinum-containing regimen and are not candidates for curative surgery or radiation.
“This US regulatory action confirms our confidence in Jemperli as an important treatment option for patients with dMMR recurrent or advanced endometrial cancer,” said Hesham Abdullah, Senior Vice President, Global Head of Oncology Development at GSK. We continue to harness Jemperli’s potential as the foundation for our immuno-oncology development programs to address unmet patient needs, including earlier lines of endometrial cancer and other solid tumors.”
Jemperli was granted accelerated approval in April 2021 for the treatment of adult patients with dMMR recurrent or advanced endometrial cancer that had progressed on or after prior treatment with a platinum-containing regimen. This approval is based on additional data from the ongoing GARNET trial’s A1 expansion cohort, which is a phase I, multicenter, open-label, single-arm study of Jemperli monotherapy in patients with advanced or recurrent solid tumors. Cohort A1 looked at Jemperli’s efficacy in 141 patients with dMMR advanced or recurrent endometrial cancer who had progressed on or after prior treatment with a platinum-containing regimen.
Overall response rate (ORR) and duration of response (DOR) were the primary efficacy outcome measures, as determined by a blinded independent central review using RECIST v1.1. The confirmed ORR was 45.4% (95% CI: 37.0, 54.0), with a complete response rate of 15.6% and a partial response rate of 29.8%. The median DOR (measured from the time of first response) was not reached (range: 1.2+, 52.8+ months), with 85.9% of patients having a duration of 12 months and 54.7% having a duration of 24 months. The median duration of response was 27.9 months.
Treatment-related adverse events for cohort A1 were consistent with previous analyses. Fatigue/asthenia, anemia, rash, nausea, diarrhea, constipation, and vomiting were the most common adverse reactions (20%). Anemia, increased transaminases, urinary tract infection, fatigue/asthenia, and diarrhea were the most common Grade 3 or 4 adverse reactions (2%).
Regenerative Medicine Advanced Therapy (RMAT) and FDA Fast Track Designations to IASO Bio’s CT103A
On Feb. 12, 2023, IASO Biotherapeutics (IASO Bio), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, announced that the U.S. Food and Drug Administration (FDA) had granted both Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track (FT) designation to its investigational new drug BCMA CAR-T CT103A (Equecabtagene Autoleucel) for the treatment of relapsed/refractory Multiple Myeloma (RRMM). IASO Bio and Innovent Biologics, Inc. (1801.HK) are jointly developing equecabtagene autoleucel for the treatment of RRMM in mainland China.
IASO Bio’s Equecabtagene autoleucel (CT103A) is a BCMA chimeric antigen receptor autologous T cell injection, a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3ζ activation domains. Based on strict selection and screening, utilizing a proprietary in-house optimization platform, and integrated in-house manufacturing process improvement, the construct of CT103A is potent and shows prolonged persistency in patients.
The NMPA accepted the New Drug Application for equecabtagene autoleucel for the treatment of relapsed/refractory multiple myeloma (RRMM). Earlier in February 2021, Equecabtagene autoleucel also received Breakthrough Therapy Designation by the NMPA, and later on, in February 2022, it received Orphan Drug Designation (ODD). Similarly, in December 2022, U.S. FDA granted IND approval to Equecabtagene autoleucel. Moreover, as per the update from the IASO Bio, in addition to multiple myeloma, the NMPA has approved the IND application of equecabtagene autoleucel for the new expanded indication of Neuromyelitis Optica Spectrum Disorder (NMOSD).
Multiple myeloma is a cancer of plasma cells. In general, when plasma cells become cancerous and grow out of control, this is called multiple myeloma. It is the second most common blood cancer diagnosis, after non-Hodgkin lymphoma, in the United States. MM is slightly more prevalent in males compared to females. Myeloma incidence is strongly related to age, with the highest incidence rates being in older people.
As per DelveInsight, in the year 2020, the total number of incident cases of Multiple Myeloma was 91,693 cases in the 8MM (i.e., the United States, EU4 (Spain, Germany, France, and Italy), the UK, Japan, and China. In the United States, there were 32,270 cases of Multiple Myeloma (the highest number among the 8MM) in 2020. China reported the second-highest incident cases of Multiple myeloma, i.e., 21,116, which accounted for nearly 23% of the total 8MM cases in the year 2020. In relapsed/refractory MM (RRMM), cancer returns after treatment or after a period of remission. Refractory multiple myeloma refers to cancer that is not responding to standard therapy. There are many potential mechanisms that may impact and promote treatment resistance. The development of emerging therapies like CT103A, and others, holds immense potential to improve the relapsed/refractory MM (RRMM) treatment scenario.
Jubilant Therapeutics Receives Orphan Drug Designation for its Drug, JBI-778, to Treat Glioblastoma Multiforme (GBM)
On February 13, 2023, Jubilant Therapeutics Inc. announced that the United States Food and Drug Administration (US FDA) had granted Orphan Drug Designation (ODD) for JBI-778 for the treatment of Glioblastoma Multiforme (GBM). Jubilant Therapeutics’s JBI-778 is an oral, brain penetrant, and substrate-competitive protein arginine methyl transferase 5 (PRMT5) inhibitor for treating tumors with brain metastases and primary brain tumors, including high-grade glioma.
The drug has a unique mechanism of action compared to existing PRMT5 inhibitors by being substrate-competitive and S-adenosylmethionine (SAM) cooperative, combined with a high brain exposure that enables targeting of both primary brain tumors and CNS metastasis.
Syed Kazmi, Chief Executive Officer of Jubilant Therapeutics Inc., has said, “JBI-778’s differentiated profile compared to other PRMT5 inhibitors in development addresses safety issues of first-generation PRMT5 inhibitors. It also enables a balanced exposure in the brain and plasma – for the treatment of GBM and brain metastases along with the systemic disease. We have identified a novel synthetic lethality approach for prospective patient selection in target indications. JBI-778 has already been cleared by the US FDA to initiate clinical trials, which we anticipate to start during the second half of 2023.”
As per DelveInsight, the total diagnosed incident population of Glioblastoma in the 7MM was 32,546 cases in 2021 and is projected to increase in the coming years. Among the EU-4, the UK countries, Germany accounted for the highest number of Glioblastoma cases, followed by France, whereas Spain accounted for the lowest number of cases in 2021. There are several emerging therapies under development to treat Glioblastoma Multiforme. The approval and the launch of emerging therapies, including JBI-778, among others, hold immense potential to improve the Glioblastoma Multiforme treatment scenario in the coming years. JBI-778 has a unique opportunity to address the unmet needs of patients with an enhanced therapeutic index.
Roche Announces Positive New Phase III Data Vabysmo in RVO
Roche declared positive new data from two global phase III studies, BALATON and COMINO, evaluating Vabysmo (faricimab) in macular edema due to branch and central retinal vein occlusion at 24 weeks. According to the studies, Vabysmo treatment produced early and long-lasting vision improvements and met the primary outcome of non-inferior visual acuity gains compared to treatment with aflibercept.
Reduced central subfield thickness, a measure of retinal fluid drying from baseline, was another indication of vabysmo’s effectiveness. Vabysmo’s safety profile matched that of earlier studies. The Bascom Palmer Eye Institute in Florida, United States, is hosting Angiogenesis, Exudation, and Degeneration 2023, where the results will be virtually presented on February 11.
Around 70 million people worldwide are affected by retinal vein occlusion, diabetic macular edema, and neovascular or wet age-related macular degeneration, which are three of the main causes of vision loss. Data from the BALATON and COMINO studies will be submitted to health agencies around the world, including the European Medicines Agency and the US Food and Drug Administration, for clearance to treat macular edema brought on by RVO. This would be Vabysmo’s third indication; it is already permitted in more than 50 countries to treat diabetic macular edema and non-AMD macular edema.
The effectiveness and safety of vabysmo in treating nAMD and DME have been proven by two-year data from four major, global studies with more than 3,000 participants. With phase III trials showing treatment intervals of up to four months for persons with these problems, Vabysmo is the first bispecific antibody approved for the eye. It targets and blocks angiopoietin-2 and vascular endothelial growth factor-A, two signalling pathways associated with a number of retinal disorders that can cause blindness. To treat these conditions, more than 450,000 Vabysmo doses have been given out globally.
FDA Accepts NDA for Aldeyra’s reproxalap for Dry Eye Disease
Aldeyra Therapeutics declared that the U.S. Food and Drug Administration had accepted the New Drug Application for topical ocular reproxalap, a first-in-class investigational new drug candidate, for the treatment of the signs and symptoms of dry eye disease. The FDA assigned a Prescription Drug User Fee Act date of November 23, 2023. The FDA noted that no potential filing review issues have been identified and that an advisory committee meeting is not currently planned.
The NDA is supported by previously reported safety and efficacy results from five appropriate and well-controlled clinical trials that included information for the Schirmer test, Schirmer test 10 mm responder analysis, ocular dryness symptom score, and ocular redness. The NDA contains information on action starting minutes after medication administration and lasting up to 12 weeks, crossover and parallel-group clinical trial designs, and evaluation in dry eye chamber challenge and real-world situations. More than 2,000 participants have received topical ocular reproxalap studies, and there have been no clinically relevant safety issues noted. Mild and temporary instillation site irritation is the most often reported adverse event in clinical trials.