Pipeline News and Updates
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We added six new drugs to the knowledgebase:
Regulatory Update
The FDA designated gedatolisib a Breakthrough Therapy for the treatment of HR+/HER2- metastatic breast cancer that has progressed during treatment with a CDK4/6 therapy and a non-steroidal aromatase inhibitor. Acadia submitted an NDA for trofinetide for the treatment of Rett syndrome. The United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) supports an expedited review of sabizabulin for the treatment in hospitalized COVID-19 patients at high risk for Acute Respiratory Distress Syndrome (ARDS). The EMA approved teclistamab for the treatment of relapsed and refractory multiple myeloma. The EMA approved eladocagene exuparvovec for the treatment of aromatic l-amino acid decarboxylase (AADC) deficiency. Announced Research Updates VBL Therapeutic announced that in the 409 patient, Phase III OVAL trial, adding ofranergene obadenovec to paclitaxel did not improve overall survival or progression-free survival compared to paclitaxel monotherapy in patients with platinum-resistant ovarian cancer. VistaGen announced that in the Phase III PALISADE-1 trial, treatment with PH94B did not decrease the Subjective Units of Distress Scale (SUDS) compared to placebo in patients with social anxiety experiencing acute anxiety. Published Research Updates The 639 patient Phase III, INTELLANCE-1 trial was discontinued after an interim analysis failed to demonstrate an improvement in overall survival with depatuxizumab in combination with radiation and temozolomide in the treatment of EGFR-amplified glioblastoma. In the 52-week, 346 patient, Phase III, ACOUSTICS trial, the annual exacerbation rate was decreased by 51% with lebrikizumab 125 mg and 40% with 37.5 mg compared to placebo in adolescents (aged 12-17 years) with uncontrolled asthma. In the 20-week, 276 patient, Phase II METEORIC-HF trial, treatment with omecamtiv mecarbi did not improve peak oxygen uptake during cardiopulmonary exercise testing compared to placebo in patients with HFrEF. In the six-week, 166 patient, Phase III FLASH trial, 16% of patients treated with one cycle of light activated topical hypericin had at least a 50% reduction in lesions compared to 4% with placebo in patients with early-stage cutaneous T-cell lymphoma. In the open-label extension of the trial, 40% of patients that received two cycles and 49% that received three cycles achieved a 50% reduction. In a 12-week, 40 patient, Phase II trial, treatment with palovarotene did not reduce heterotopic ossification flare-ups compared to placebo in patients with fibrodysplasia ossificans progressiva (FOP). In a 402 patient, Phase III trial, treatment with the oral combination of paclitaxel and encequidar resulted in an overall response rate of 36% compared to 23% with IV paclitaxel in patients with metastatic breast cancer. In the induction phase of the 50-week, 219 patient, Phase III ultraVIOLET trial, oteseconazole was non-inferior to fluconazole (93.2% vs 95.8%) in resolving an acute vulvovaginal candidiasis (VVC) infection in female patients with recurrent VVC. 185 patients entered the maintenance phase and were treated with oteseconazole or fluconazole for 11 weeks and then followed for 37 weeks. At the end of 48-weeks, 5.1% of oteseconazole patients experienced a culture-verified acute infection compared to 42.2% with fluconazole. This week we added eight new drugs to the knowledgebase:
Regulatory Update
The FDA delayed a decision for tislelizumab, until a site inspection of BeiGene’s Chinese manufacturing facility can be completed. Travel issues related to COVID-19 have prevented the FDA from inspecting the plant. Announced Research Updates Pluristem announced that in a 26-week, 240 patient, Phase III trial, treatment with PLX-PAD did not improve the Short Physical Performance Battery (SPPB) test score compared to placebo in patients undergoing hip arthroplasty following fracture. Sesen Bio voluntarily paused development of oportuzumab monatox and is seeking financial partners to help fund further development. Published Research Updates In the 16-week, 666 patient, Phase III, POETYK PSO-1 trial, 58.4% of patients treated with deucravacitinib achieved PASI 75 and 53.6% achieved a Physician's Global Assessment (sPGA) score of 0 to 1 compared to 35.1% and 32.1% with apremilast and 12.7% and 7.2% with placebo in patients with moderate to severe psoriasis. In the 112 patient cohorts of the Phase II, KRYSTAL-1 trial, treatment with adagrasib resulted in an ORR of 42.9% in patients with non-small cell lung cancer (NSCLC) harboring the KRAS G12C mutation, who had been treated with platinum-based chemotherapy and anti-programmed death 1 or programmed death ligand 1 therapy. In a 100 patient, Phase 1b trial, treatment with zandelisib resulted in an objective responses of 92% with 60 mg and 120 mg, and 83% with 180 mg in patients with relapsed or refractory B-cell malignancy. In the 20-week, 139 patient, Phase II ELEKTRA trial, treatment with soticlestat resulted in a 50% reduction in convulsive and drop seizure frequencies in Dravet syndrome patients compared to placebo. The decrease in seizures in Lennox-Gastaut syndrome patients was not significantly better than placebo. Regulatory Update
The FDA accepted the BLA for mosunetuzumab to treat relapsed or refractory follicular lymphoma in patients who have received at least two prior systemic therapies. A PDUFA date is set for 12/29/2022. The FDA accepted the NDA for lecanemab to treat Alzheimer’s disease, and set a PDUFA date for 1/6/2023. Two months after rejecting toripalimab due to a manufacturing quality process deficiency, the FDA accepted the resubmitted NDA and set a PDUFA date for 12/23/2022. An onsite inspection of the manufacturing plant will be required. The FDA placed a hold on the Phase II/III ReMEDy2 trial (NCT05065216), due to the incidence of hypotension in patients treated with DM199. Announced Research Updates Nordic Nanovector discontinued the Phase IIb Paradigm trial, evaluating lutetium (177lu) lilotomab satetraxetan to treat refractory follicular lymphoma, after an interim analysis revealed low efficacy Sarepta announced one-year functional results from 20 patients enrolled in cohort 1 of the open-label, Phase I, ENDEAVOR trial, where treatment with delandistrogene moxeparvovec improved the NSAA score compared to a historical control group of patients with DMD. There was one case of myocarditis identified in the Phase I ENDEAVOR trial, which appeared to resolve four months after treatment with IV methyl-prednisolone. Sarepta also announced four-year results from a four patient, Phase I/II trial, where delandistrogene moxeparvovec maintained a 7 point improvement in the North Star Ambulatory Assessment (NSAA) over baseline and a 9.4 point improvement compared to a historical control group in patients with Duchenne muscular dystrophy. Lysogene announced 24 month data from 16 patients enrolled in the 20 patient, Phase II/III, AAVance trial, where treatment with LYS-SAF302 resulted in a 22% decrease in heparan sulfate (HS) in the cerebrospinal fluid (CSF), a 20% decrease in HS-derived oligosaccharides in the CSF, 41% decrease in serum neurofilament light (NFL) levels and a 33% decrease in CSF NFL levels in patients with mucopolysaccharidosis Type IIIA (MPS IIIA). MacroGenics discontinued a Phase 2 trial evaluating enoblituzumab in combination with retifanlimab or tebotelimab, in the treatment of recurrent or metastatic squamous cell carcinoma of the head and neck, after seven fatalities potentially associated with hemorrhagic events were reported. Novo Nordisk announced that in the 32-week, 133 patient, Phase III, explorer7 trial, prophylaxis with concizumab resulted in an annual bleeding rate of 1.7 compared to 11.8 with no prophylaxis in patients with hemophilia A or B with inhibitors. Sanofi announced that in the 13-month, 80 patient, Phase III, ATLAS-PPX trial, prophylaxis with fitusiran resulted in an annual bleeding rate of 0 compared to 61.1% with prior factor or bypassing agent prophylaxis in patients with hemophilia A or B with or without inhibitors. Aldeyra announced that in a two-day, 50 patient, Phase II crossover trial, treatment with reproxalap decreased eye redness and increased tear production as measure with a Schirmer test in patients with dry eye disease. Published Research Updates In the 104-week, 206 patient, open-label, Phase II, BE ACTIVE 2 trial, a long-term extension of the BE ACTIVE trial, continued treatment with bimekizumab resulted in sustained improvement in pain, fatigue and function. 16-week results from the 52-week, 503 patient, Phase III, OASIS-1 trial, found that 69.3%of patients treated with mirikizumab achieved at least a 2-point improvement in their sPGA score and 64.3% achieved PASI90 compared to 6.5% and 6.5%with placebo in patients with moderate to severe plaque psoriasis. At 52-weeks, PASI90/PASI100/sPGA response was achieved with mirikizumab 250Q4W/placeboQ8W in 18.7%/9.9%/17.6%, mirikizumab 250Q4W/125Q8W in 85.6%/58.9%/85.6%, and mirikizumab 250Q4W/250Q8W in 84.6%/60.4%/82.4%. In a 90 patient cohort of an open-label, Phase I/Ib trial, treatment with mosunetuzumab resulted in a complete response (CR) rate of 60% compared to 14% in a historical control group that received copanlisib in patients with relapsed or refractory follicular lymphoma. In the 94-patient, Phase II, open-label, POD1UM-202 trial, treatment with retifanlimab resulted in an objective response rate of 13.8% in patients with locally advanced or metastatic squamous cell carcinoma of the anal canal, who progressed on, or are intolerant of, platinum-based chemotherapy. In a 33 patient, Phase II trial, treatment with eprenetapopt plus azacitidine resulted in relapse free survival of 12.5 months in post-transplant patients with TP53 mutant MDS and AML. AMX0035 (Sodium phenylbutyrate & Taurursodiol)
Santhera has worldwide marketing rights for vamorolone for DMD and all other indications. Santhera began a rolling NDA for vamorolone in the treatment of DMD in March 2022. The contract manufacturer for vamorolone, will not be ready for an FDA inspection until 4Q22, so completion of the NDA will not be until the end of the year. The FDA extended the PDUFA for teplizumab by three-months to allow more time to review labeling and if any post-marketing requirements are required. The new PDUFA date is 11/17/2022. The FDA put a hold on HIV trials using injectable lenacapavir due to concerns regarding the compatibility of vials made of borosilicate glass with lenacapavir solution. These vials could potentially lead to the formation of sub-visible glass particles. The FDA rejected the NDA for lenacapavir in March 2022 due to the problems with the vials. Gilead developed a new type of vial and the hold was lifted in May 2022. Gilead resubmitted an NDA for lenacapavir for the treatment of multi-drug resistant HIV-1 infection in June 2022. The new NDA includes data to demonstrate the compatibility of lenacapavir with an alternative vial type made from aluminosilicate glass. The FDA rejected tebipenem for the treatment of complicated urinary tract infections and requested an addition clinical trial. The EMA approved olipudase alfa to treat non-Central Nervous System manifestations of Acid Sphingomyelinase Deficiency. Announced Research Updates MeiraGTx, announced that in a 6-month, 45 patient, open-label, dose-escalation, Phase I/II trial, treatment with botaretigene sparoparvovec resulted in an improvement in low light visual mobility, patient reported outcomes in extreme light and ETDRS visual acuity in patients with X-linked retinitis pigmentosa (XLRP). Jazz Pharmaceuticals announced that in the 51-week, 68 patient, Phase III, RELEASE MSS1 trial, treatment with nabiximols oromucosal spray did not improve the Lower Limb Muscle Tone-6 (LLMT-6) score compared to placebo in patients with multiple sclerosis spasticity. Capricor announced that in an extension of the HOPE-2 trial, DMD patients received no treatment for 392 days, then were reinitiated on CAP-1002 every three months. After one-year of open label treatment, CAP-1002 continued to demonstrate improvements in the mid-level elbow PUL 1.2 score compared to placebo. Novartis announced that in the 649 patient, Phase III, RATIONALE 306 trial, treatment with tislelizumab plus chemotherapy resulted in overall survival of 17.2 months compared to 10.6 months with chemotherapy alone in patients with advanced or metastatic esophageal squamous cell carcinoma. Can-Fite BioPharma announced that in the 16-week, 528 patient, Phase III, COMFORT trial, 9.7% of patients treated with piclidenoson 3 mg achieved PASI-75 compared to 2.6% with placebo in patients with moderate-to-severe plaque psoriasis. A non-inferior difference was found in a secondary endpoint at week 32, 17% of patients treated with piclidenoson achieved PASI-75 compared to 26.2% with apremilast. DBV Technologies announced that in the 12-month, 362 patient, Phase III, EPITOP trial, 67% of patients who received Viaskin Peanut patch tolerated a peanut protein challenge compared to 33.5% with placebo in patients 1 to 3 years old with peanut allergies. Published Research Updates In the 152 patient, Phase II APPROVE trial, treatment with apatinib plus pegylated liposomal doxorubicin resulted in PFS of 5.8 months compared to 3.3 months with pegylated liposomal doxorubicin alone in patients with platinum-resistant or refractory ovarian cancer. |
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