The FDA approved givosiran (Givlaari, Alnylam) on 11/20/2019 for the treatment of adults with acute hepatic porphyria. WAC for givosiran is $39,000 per vial with an estimated annual cost after discount of $442,000.
The FDA approved cenobamate (Xcopri, SK Life Science) on 11/21/2019 for the treatment of partial-onset seizures in adult patients with epilepsy. The FDA reports the most common ADR in the trials were somnolence, dizziness, fatigue, and diplopia. Cenobamate may cause a reduction in the QT interval > 20 milliseconds, so the FDA advises avoiding the drug in patients with Familial Short QT syndrome.
The FDA has designated arimoclomol a Breakthrough Therapy for Niemann-Pick Disease Type C.
Alkermes submitted an NDA for olanzapine/samidorphan (ALKS 3831) for the treatment of schizophrenia and for the treatment of bipolar I disorder.
The FDA has granted sarizotan a Rare Pediatric Disease designation for the treatment of Rett syndrome.
BioMarin submitted an MAA for valoctocogene roxaparvovec to treat Hemophilia A.
Takeda plans to file an NDA for its Dengue vaccine by the end of 2020.
Announced Research Updates
Merck and Bayer announced that in a 5,050 patient, Phase III VICTORIA trial, vericiguat added to current heart failure therapies increased the time to the occurrence of cardiovascular death or heart failure hospitalization compared to placebo in patients with worsening chronic heart failure with reduced ejection fraction (HFrEF) following a decompensation event, defined as heart failure with hospitalization or receiving an intravenous diuretic for heart failure without hospitalization.
UCB announced that in the 16-week, 435 patient, Phase III, BE READY trial, more patients treated with bimekizumab achieved PASI 90 and IGA 0/1 than placebo in patients with moderate-to-severe chronic plaque psoriasis.
Myovant announced that in the 48-week, 934 patient, Phase III, HERO trial, 96.7% of patients treated with relugolix achieved sustained testosterone suppression to castrate levels (< 50 ng/dL) compared to 88.8% with leuprolide in men with advanced prostate cancer.
The Medicines Company announced that in the 18-month, 482 patient, Phase III, ORION-9 trial, the placebo adjusted reduction in LDL with inclisiran was 50% in patients with heterozygous familial hypercholesterolemia with elevated LDL despite maximally tolerated statins.
Published Research Updates
In the 26-week, 400 patient, Phase III, FUEL trial, treatment with udenafil did not increase oxygen consumption at peak exercise compared to placebo in adolescents who had undergone Fontan surgery.
In the 1,355 migraine patient, Phase III, ACHIEVE II trial, the percentage of patients pain free at two hours was 20.7% of those treated with ubrogepant 25 mg, 21.8% with 50 mg and 14.3% with placebo. The most bothersome symptoms at two hours were eliminated in 38.9% treated with ubrogepant 50 mg compared to 27.4% with placebo. The lower 25 mg dose of ubrogepant did not reach a statistical difference compared to placebo for freedom of the most bothersome symptoms.
In a 142 patient, Phase II trial, treatment with buparlisib provided limited efficacy in patients with resistant solid or hematologic malignancies with PI3K pathway activation.
According to NIH’s National Cancer Institute, Prostate cancer is the third most common cancer in the US as of February 2019 sorted by estimated new cases. There were 174,650 estimated new cases which were estimated to result in 31,620 deaths.
There are eight investigational drugs targeting Prostate Cancer in the Pharmaceutical Pipeline Tracker. Seven are in Phase III trials while the remaining drug is in multiple Phase II Trials. None of the drugs have PDUFA Dates at this time and the FDA has not assigned any Priority Designations to any of the eight drugs. Of the eight drugs Genentech/Array BioPharma's ipatasertib is available in the EU while Steba Biotech's padeliporfin (Tookad) is approved in the EU.
The FDA approved cefiderocol (Fetroja, Shionogi) on 11/15/2019 for the treatment of adult patients with complicated urinary tract infections (cUTI), including kidney infections caused by susceptible Gram-negative microorganisms, who have limited or no alternative treatment options. The package insert for cefiderocol will include a warning regarding higher all-cause mortality rate observed in cefiderocol-treated patients compared to those treated with other antibiotics in a trial in critically ill patients with multidrug-resistant Gram-negative bacterial infections.
The FDA approved crizanlizumab (Adakveo, Novartis) on 11/15/2019 to reduce the frequency of vaso-occlusive crisis in patients aged 16 years or older with sickle cell disease. WAC for crizanlizumab is $2,357 per vial. With an average dose of three to four vials per month, WAC would be $7,071, or $9,428, per month.
The FDA approved zanubrutinib (Brukinsa, BeiGene) on 11/13/2019 for the treatment of adults with mantle cell lymphoma who have received at least one prior therapy. WAC for a 30-day supply of zanubrutinib has been set at $12,935.
The FDA designated aztreonam/avibactam as a Qualified Infectious Disease Product (QIDP) and gave the combination Fast Track status for the treatment of complicated intra-abdominal infections (cIAI), complicated urinary tract infections (cUTI), and hospital-acquired bacterial pneumonia (HABP)/ventilator-associated bacterial pneumonia (VABP).
The EU approved Merck’s ebola vaccine.
The EMA’s CHMP recommended approval of osilodrostat for the treatment of Cushing syndrome.
Solid Biosciences’ SGT-001 had temporary holds on the Phase I/II IGNITE DMD trial in 2017 and 2018. The November 2017 hold was due to manufacturing problems and the January 2018 hold was due to a patientdeveloping thrombocytopenia and anemia. Both holds were resolved and the trial resumed in June 2018. The company added monitoring for complement activation and IV steroids to the protocol and made eculizumab available to treat complement activation if it developed. In November 2019, the FDA put a hold on the IGNITE DMD trial when a patient developed complement activation leading to thrombocytopenia, anemia, acute kidney injury, and cardio-pulmonary insufficiency. There were no cytokine-related nor coagulopathy-related abnormalities observed in the patient. The patient had received the highest dose of SGT-001, which is four times higher than the patient in the previous trial.
The FDA designated mavorixafor a Breakthrough Therapy for the treatment of WHIM syndrome.
The FDA granted a priority review to selumetinib to treat children with neurofibromatosis type 1 who've developed plexiform neurofibromas that cannot be treated with surgery.
CytoDyn has initiated an expanded access program for use of leronlimab in patients with triple-negative breast cancer who have exhausted all approved treatment options and do not qualify for a clinical trial. Details of the program can be found at: https://www.cytodyn.com/expanded-access
Announced Research Updates
Roche announced that in the 12-month, 180 patient, Phase III SUNFISH trial, treatment with risdiplam improved motor function as measured by the Motor Function Measure 32 (MFM32) score in patients with Type 2 or 3 spinal muscular atrophy.
Reata announced that in the 48-week, 157 patient, Phase III CARDINAL trial, treatment with bardoxolone resulted in an increase in the mean eGFR of 4.72 mL/min/1.73 sq m compared to a decrease of 4.78 mL/min/1.73 sq m with placebo in patients with Alport syndrome. After a 4-week wash-out period, the bardoxolone had a decline from baseline of 0.96 mL/min/1.73 sq m compared to a 6.11 mL/min/1.73 sq m decrease with placebo.
Akebia announced that in a 52-week, 304 Japanese patient, Phase III, open label trial, vadadustat was non-inferior to darbepoetin alfa in mean Hb (11.51 g/dL vs 11.58 g/dL) in patients with non-dialysis dependent CKD. In a 52-week, 323 Japanese patient, Phase III, trial, vadadustat was non-inferior to darbepoetin alfa in mean Hb (10.39 g/dL vs 10.62 g/dL) in patients with hemodialysis-dependent CKD.
AZ announced that in the 12-month, 373 patient, Phase III, TULIP 2 trial, 47.8% of patients treated with anifrolumab had a decrease in disease activity compared to 31.5% with placebo in patients with moderate-to-severe autoantibody-positive systemic lupus erythematosus.
UCB announced that in a 48-week, 265 patient, Phase IIb trial, 35.5%–64.0% of patients treated with bimekizumab achieved ASAS40 in patients with ankylosing spondylitis.
Mallinckrodt announced that in the 35-month, 300 patient, Phase III CONFIRM trial, 29.1% of patients treated with terlipressin achieved HRS-1 reversal (renal function improvement, avoidance of dialysis, short-term survival) compared to 15.8% with albumin monotherapy in patients with HRS-1.
Eiger announced interim data from 19 patients enrolled in the 24-week, 26 patient, Phase IIa, open-label, LIFT trial;18 /19 patients treated with the combination of lonafarnib, ritonavir and pegylated interferon lambda achieved a > 2 log IU/mL decline in HDV RNA with a median decline of 3.4 log IU/mL in patients with a chronic infection of the hepatitis delta virus.
Gilead and Galapagos announced that an extension of the Phase II DARWIN 1 & 2 trials monitored 739 patients for 156 weeks in the Phase II, DARWIN 3, open label trial, where AR20 was achieved by 89.7% of patients that received filgotinib monotherapy and 87.2% of patents that received filgotinib and MTX.
AnaptysBio announced that in a 20 patient, Phase IIa trial, 11/15 patients treated with etokimab were able to tolerate a 275 mg dose of peanut protein two weeks after a single injection compared to none (0/5) in the placebo group in patients with peanut allergy and a clinical history of anaphylaxis. At 45 days, 4/7 patients passed the peanut protein challenge compared to none with placebo.
BioMarin announced results from a 10 patient cohort enrolled in a 54 month Phase II, open label, achondroplasia trial, where treatment with vosoritide 15 µg/kg/day resulted in a mean increase of 9 cm compared to an age and gender matched natural history achondroplasia dataset (N=619).
ResTORbio announced that in the 16-week, 1,024 patient, Phase III, PROTECTOR 1 trial, treatment with dactolisib during winter cold and flu season did not decrease respiratory tract infections (RTI) compared to placebo (odds of experiencing an RTI 0.46 vs 0.44) in patients 65 or older, excluding current smokers and individuals with chronic obstructive pulmonary disease. Development of dactolisib for the prevention of respiratory tract infections in older patients was discontinued after the failure of the Phase III PROTECTOR 1 trial. Dactolisib remains in development with or without everolimus in the treatment of Parkinson’s disease with a Phase 1b/2a trial that is expected to be completed in mid-2020.
The Medicines Company announced that in the 18-month, 1,561 patient, Phase III, ORION-10 trial, the placebo adjusted reduction in LDL with inclisiran was 58% in patients with atherosclerotic cardiovascular disease or risk equivalents and elevated LDL despite maximum tolerated dose of statin with or without ezetimibe. In the 18-month, 1,617 patient, Phase III, ORION-11 trial, major adverse cardiovascular events (MACE) events were 47% lower with inclisiran than the placebo, but in the 18-month, 1,561 patient, Phase III, ORION-10 trial MACE events were slightly higher with inclisiran (4.1% vs 3.3%).
Published Research Updates
In the 12-week, 779 patient Phase III, CLEAR Wisdom trial, patients treated with bempedoic acid lowered LDL 15.1% compared to 2.4% with placebo in patients with atherosclerotic cardiovascular disease, heterozygous familial hypercholesterolemia, or both and LDL of 70 mg/dL or > despite maximally tolerated lipid-lowering therapy.
In the 12-week, 156 patient, Phase II, OASIS trial, treatment with etrasimod 2 mg improved the Mayo Clinic Score (a composite of rectal bleeding, stool frequency, and endoscopic findings) 1 point more than placebo, but the 1 mg dose was not significantly different from placbo in patients with moderate to severe ulcerative colitis.
In the 11.6-month, 307 patient Phase III, open label, ICARIA-MM trial, isatuximab added to pomalidomide and low-dose dexamethasone resulted in progression free survival of 11.5 months compared to 6.5 months with pomalidomide and dexamethasone alone in patients with relapsed/refractory multiple myeloma.
In a 31-month, 50 patient, Phase II trial, treatment with Lu-PSMA-617 resulted in a decrease in PSA values of at least 50% in 64% of patients and median overall survival was 13.3 months in men with metastatic castration-resistant prostate cancer.
Three new drugs are in development for the treatment of acute migraine. While many patients receive relief with a triptan, not all patients can tolerate this class of drugs or are helped by them. The triptans also have a contraindication in patients with cardiovascular disease, peripheral vascular disease, cerebrovascular disease, and uncontrolled hypertension because of the potential for ischemia. For these reasons new drugs are being developed. The investigational gepants (ubrogepant and rimegepant) and the recently approved lasmiditan (a ditan). The gepants (ubrogepant and rimegepant) and lasmiditan do not cause vasoconstriction and may be an alternative when patients do not tolerate triptans or cannot use a triptan due to cardiovascular disease.
ICER released a draft review of lasmiditan, ubrogepant and rimegepant in November 2019. ICER found the drugs to be comparable to each other in efficacy, but not as efficacious as triptans. Lasmiditan, ubrogepant and rimegepant would provide a benefit for patients with cardiovascular disease that have a contraindication to triptans, were not helped by triptans or do not tolerate them. Since triptans are available as generics, ICER concludes that triptans would provide a greater benefit at a lower cost than lasmiditan, ubrogepant or rimegepant. To reach a threshold of $150,000 per quality-adjusted life year ICER estimated an annual cost of $1,850 for lasmiditan, $1,780 for rimegepant and $1,740 for ubrogepant.
With the lack of data to support efficacy equivalence to triptans and a higher cost than generic triptans, the role of lasmiditan, ubrogepant and rimegepant may be for the treatment of patients that require treatment beyond an NSAID or acetaminophen and are not eligible for treatment with a triptan. There is the possibility of lower prices with these drugs by Spring 2020 with PDUFA dates for ubrogepant in December 2019 and rimegepant in March 2020.
CGRP is a neuropeptide that has both cerebral arteriolar dilating and pain modulation properties.
Decreasing CGRP inhibits vasodilation without inducing vasoconstriction. The gepants are small molecule orally administered CGRP antagonists. Merck abandoned work on two early CGRP Antagonists, telcagepant (MK-0974) and MK-3207 due to hepatotoxicity concerns, so hepatotoxicity continues to be monitored.
The FDA approved luspatercept (Reblozyl, Celgene, Acceleron) on 11/8/2019 for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions.
The FDA is due to make a decision on Shionogi’s cefiderocol this week with a PDUFA date on 11/14/2019.
In 2013 the FDA rejected Aveo’s tivozanib 507 patient, Phase III, TIVO-1 trial as a pivotal renal cancer efficacy trial, due to inconsistencies in the overall survival and progression free survival results. In November 2019 the FDA expressed concern over the interim results presented from the TIVO-3 trial and felt it did not demonstrate improved overall survival and that final analysis may provide worse results. Aveo is planning to narrow its NDA for tivozanib to relapsed/refractory renal cell cancer, but the FDA will require approval of the new statistical analysis and a presentation of results to an advisory committee. Aveo could lose approval in Europe depending on the final results from the TIVO-3 trial.
The FDA accepted the NDA for Cassiopea’s clascoterone cream 1% for the treatment of acne and set a PDUFA date of Aug. 27, 2020.
Announced Research Updates
Halozyme announced that in a 500-patient, Phase III trial, treatment with PEGPH20 in combination with gemcitabine and nab-paclitaxel did not improve overall survival compared to gemcitabine and nab-paclitaxel alone (11.2 months vs 11.5 months) in patients with Stage IV previously untreated pancreatic ductal adenocarcinoma. Halozyme announced the discontinuation of the development of PEGPH20 after the failed Phase III trial. Halozyme is restructuring the company to focus on their ENHANZE drug delivery technology.
Scynexis announced that in the 376 patient, Phase III, VANISH-303 trial, treatment with ibrexafungerp resulted in clinical cure at day 10 in 50.5% of patients compared to placebo in female patients with vulvovaginal candidiasis.
Green Valley announced that in a 9-month, 818 patient, Phase III trial, patients treated with oligomannate had a decrease in their ADAS-Cog12 Score of 2.54 points less than placebo (2.7 vs 0.16) in Chinese patients with mild to moderate Alzhemer's disease. China granted conditional approval, with monitoring for safety for oligomannate to treat mild to moderate Alzheimer's disease and improve cognitive function. An editorial comment on the Chinese Phase III trial questioned if the improvement seen was accurate since few details on the trial are given and modulating neuroinflammation has not been shown to improve cognition in Alzheimer’s patients in earlier trials.
AB Science SA announced that in a 36-week, 355 patient, Phase III trial, treatment with masitinib reduced the severe asthma exacerbation rate compared to placebo in patients with severe asthma uncontrolled by oral corticosteroids.
ObsEva announced that in the 10-week, 778 patient, Phase III IMPLANT4 trial, treatment with nolasiban did not improve the pregnancy rate compared to placebo (40.5% vs 39.1%) in women undergoing in vitro fertilization. ObsEva has discontinued development of nolasiban for in vitro fertilization.
MorphoSys announced that in a 48-week, 75 patient, Phase II trial, 96% of bimagrumab treated patients had lost at least 5% of their total body fat, and 77% had lost at least 15% of their total body fat in patients with obesity and type 2 diabetes.
Exelixis announced that in the 52-week, 455 patient, Phase III, ESAX-DN trial, treatment with esaxerenone reduced the urinary albumin-to-creatinine ratio 58.3% compared to an 8.3% decrease with placebo in patients with type 2 diabetes with microalbuminuria who were receiving an ACEI or ARB.
Published Research Updates
In the 36.5-month, 16,071 patient, Phase III, LOFT trial, 3.7% of patients treated with odanacatib developed a vertebral fracture compared to 7.8% that received placebo in postmenopausal women with osteoporosis. An extension trial that included 8,257 patients from the LOFT trial, 4.9% of patients treated with odanacatib developed a vertebral fracture compared to 9.6% that received placebo after a total of 47.6 months. After 47.6 months the composite of cardiovascular death, myocardial infarction, or stroke occurred was 5% with odanacatib compared to 4.3% with placebo and stroke occurred in 2.3% of odanacatib patients compared to 1.7% with placebo in 8,257 postmenopausal women with osteoporosis enrolled in the LOFT and LOFT extension studies. Merck discontinued development of odanacatib due to the increased risk of cardiovascular events, specifically stroke.
In the 12-week, 378 patient Phase III, KALM-1 trial, 51.9% of patients treated with difelikefalin had a 3-point or > improvement in the weekly mean of the 24-hour Worst Itching Intensity Numeric Rating Scale (WI-NRS) compared to 30.9% with placebo in hemodialysis patients with moderate-to-severe chronic kidney disease-associated pruritus.
The FDA approved the combination of elexacaftor, ivacaftor, and tezacaftor (Trikafta, Vertex Pharmaceuticals) on October 21 for the treatment of patients age 12 years or older with cystic fibrosis (CF) and at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. The combination was approved almost 6 months early (3/19/2020 PDUFA date).
The FDA approved lasmiditan (Reyvow, Lilly) on 10/11/2019 for the treatment of acute migraines, with or without aura, in adult patients. Due to the occurrence of dizziness and sedation from CNS depression, patients are warned of potential impairment while taking lasmiditan and advised not to drive, operate machinery or take with alcohol or other CNS depressants for at least eight hours after taking the drug, even if they feel well enough to do so. The FDA announced that during clinical trials the most common ADR with lasmiditan were dizziness, fatigue, paresthesia and sedation.
The FDA approved brolucizumab (Beovu, Novartis) on 10/8/2019 for the treatment of wet age-related macular degeneration (AMD). Brolucizumab is administered by intravitreal Injection every three months. WAC was set at $1,850 per dose, the same as aflibercept (Eylea), which is given more frequently. After the loading dose, analysts estimate the annual brolucizumab WAC at $16,000 for both eyes compared to $24,000 for aflibercept and and $40,000 for ranibizumab (Lucentis).
The FDA approved afamelanotide (Scenesse, Clinuvel) on 10/8/2019 to treat phototoxic reactions in patients with erythropoietic protoporphyria.
The FDA approved trifarotene cream (Aklief, Galderma) on October 4 for the treatment of acne vulgaris.
The FDA accepted the NDA for rimegepant, for the treatment and prevention of migraines and set a PDUFA date in March 2020.
The FDA accepted the NDA for triheptanoin, for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) and set a PDUFA target date of 7/31/2020.
The FDA accepted the BLA for Trastuzumab deruxtecan, for the treatment HER2-positive metastatic breast cancer and set a PDUFA date in March 2020.
The FDA accepted the BLA for Viaskin Peanut for the treatment of peanut-allergic children and set a PDUFA date of 8/5/2020.
Ascendis plans to file a BLA for TransCon hGH in the first half of 2020 and an MAA in in the second half of 2020 to treat pediatric growth hormone deficiency. TransCon hGH was designated an Orphan drug by the EMA.
The EMA has granted teplizumab PRIME status for the prevention or delay of clinical type 1 diabetes
The FDA Antimicrobial Drugs Advisory Committee voted 14-2 to recommend approval of cefiderocol for the treatment of complicated urinary tract infections.
The FDA awarded a Rare Pediatric Disease designation to sepofarsen for the treatment of Leber’s congenital amaurosis 10.
CHMP advised the EMA not to approve quizartinib due to a lack of evidence to support a survival benefit.
The FDA has granted Fast Track status to imetelstat for the treatment of adult patients with relapsed or refractory myelofibrosis, including whose disease has relapsed after or is refractory to janus kinase inhibitor treatment.
The FDA approved diroximel fumarate (Vumerity, Biogen/Alkermes) on 10/30/2019 for the treatment of relapsing forms of multiple sclerosis. Diroximel fumarate is an oral prodrug of monomethyl fumarate, which allows the 505(b)(2) pathway based on Biogen’s dimethyl fumarate (Tecfidera) to be used. Biogen licensing diroximel fumarate from Alkermes.
Chugai announced that in October 2019 the FDA accepted the BLA and the EMA accepted the MAA of satralizumab for the treatment of neuromyelitis optica spectrum disorder.
Announced Research Updates
Actinium announced interim data from 75 patients in the 150 patient, Phase III, SIERRA trial, where 100% of patients treated with Iomab-B achieved successful engraftment of allogeneic hematopoietic stem cell transplant compared to 18% that received salvage chemotherapy in patients with active, relapsed or refractory acute myeloid leukemia.
Athenex announced that in two 57-day, Phase III trials, treatment of actinic keratosis with tirbanibulin resulted in complete clearance in 44% and 54% of the patients compared to 5% and 13% for vehicle treated groups. One-year follow-up found recurrence rates of 74% and 72% with tirbanibulin. Athenex plans to submit an NDA for tirbanibulin in 1Q21 and an MAA in 2Q21.
AZ announced preliminary results from the 1,000 patient, Phase III, open-label, POSEIDON trial of patients with metastatic non-small cell lung cancer with PD-L1 expression levels, but not mutation in the epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase (ALK) gene. Progression free survival was improved with both durvalumab, tremelimumab and platinum-based chemotherapy and durvalumab and platinum-based chemotherapy compared to platinum-based chemotherapy alone as first line treatment
AZ announced that in a 26 patient, Phase II, open-label trial, treatment with selumetinib resulted in a partial response in 71% of patients with patients with neurofibromatosis type 1.
TG Therapeutics announced that in the 112 patient, open-label, Phase IIb, UNITY-NHL trial, treatment with umbralisib achieved the predetermined target overall response rate of 40% to 50% in patients with follicular lymphoma who have received at least 2 prior lines of therapy including an anti-CD20 monoclonal antibody and an alkylating agent.
Cyclerion announce that in a 12-week, 156 patient, Phase II trial, treatment with praliciguat did not improve the urine albumin to creatinine ratio compared to placebo in patients with type 2 diabetes and diabetic nephropathy. Cyclerion also announce that in the 12-week, 196 patient, Phase II, CAPACITY trial, treatment with praliciguat did not improve cardiopulmonary exercise testing compared to placebo in heart failure patients with preserved ejection fraction. Cyclerion is discontinuing development of praliciguat for the treatment of heart failure with preserved ejection fraction and plans to license the drug to another company for development as a treatment for diabetic nephropathy.
Published Research Updates
In a 3 year, 3,289 patient, Phase IIb trial, immunization with M72/AS01E resulted in 0.3 active pulmonary tuberculosis cases per 100 person-years compared to 0.6 cases per 100 person-years with placebo in African patients with withlatent Mycobacterium tuberculosis infection. M72/AS01E's efficacy was 49.7%. 70% of the patient population had already received the BCG vaccine.
The 26-week interim data from a 52-week, 3 patient, Phase IIb trial, found that 26-weeks after a single infusion of etranacogene dezaparvovec resulted in a mean increase in Factor IX (FIX) activity of 47% of normal range (individual results: 33%, 51%, 57%) and no patient required a Factor IX infusion, experienced a bleeding event or required immunosuppression.
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