Weekly Update for the Week Ending December 14, 2019

Regulatory Update
 
The FDA approved golodirsen (Vyondys 53, Sarepta Therapeutics) on 12/12/2019 for the treatment of Duchenne muscular dystrophy amenable to exon 53 skipping. We took a look at this surprise reversal of the August 2019 rejection of the drug in a post last week.
 
The FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee voted 12-0 to recommend approval of Horizon Therapeutics’ teprotumumab in December 2019 for the treatment of Thyroid Eye Disease.
 
The FDA lifted the clinical trial hold for Abeona Therapeutics’ EB-101 targeting recessive dystrophic epidermolysis bullosa
 
The FDA designated Dermira/Roche’s lebrikizumab a Fast Track treatment for moderate-to-severe atopic dermatitis.
 
The FDA Cardiovascular and Renal Drugs Advisory Committee voted 11-2 against recommending approval of Correvio’s vernakalant due to safety concerns over hypotension, arrhythmias, and conduction disturbance.
 
Biocryst submitted an NDA for BCX7353 in December 2019 with a hereditary angioedema indication.
 
Seattle Genetics plans to file an NDA and MAA for tucatinib in 1Q20.
 
Announced Research Updates

1. Bluebird Bio announced that in the 5-year, 23 patient, Phase I/II, open-label Northstar Trial, treatment with elivaldogene tavalentivec resulted in transfusion independence (Hemoglobin of at least 9 g/dL and no transfusions for more than 12 months) in 8 out of 10 beta-thalassemia patients without the beta 0/beta 0 genotype and 3 out of 8 who did. 
   
2. Iterum Therapeutics announced that in the 668 patient, Phase III SURE 3 trial, IV sulopenem followed by oral sulopenem/probenecid did not meet non-inferiority compared to to IV ertapenem followed by oral ciprofloxacin/metronidazole or amoxicillin-clavulanate in the treatment of complicated intra-abdominal infections.
   
3. Leo Pharma announced the results from three Phase III trials (ECZTRA 1, 2 and 3) involving 1,976 patients with moderate-to-severe atopic dermatitis who were candidates for systemic therapy. In the trials more patients treated with tralokinumab with or without topical corticosteroids achieved an Investigator Global Assessment (IGA) score of clear (0) or almost clear (1) skin at week 16 and at least a 75% or > improvement in their Eczema Area and Severity Index (EASI) score at week 16 compared to placebo. The FDA has given tralokinumab and orphan drug priority designation.
   
4. MacroGenics announced interim data from the 563 patient, Phase III, SOPHIA trial, where margetuximab plus one of four chemotherapy agents (capecitabine, eribulin, gemcitabine or vinorelbine) resulted in overall survival of 21.6 months compared to 19.8 months with trastuzumab and one of four chemotherapy agents in previously treated HER-2 positive metastatic breast cancer patients who were receiving standard chemotherapy. There was no difference in progression-free survival between the two drugs.
   
5. Mustang Bio announced updated interim data from 11 XSCID infants that completed 23.6-months in a Phase I/II, open-label trial, suggested that treatment with MB-107 lead to normal for age levels of NK cells in 3-4 months in 9 patients.
   
6. Cara announced that in a 12-week, Phase II trial, treatment with oral difelikefalin 1 mg improved the daily 24-hour Worst Itching Intensity Numeric Rating Scale (WI-NRS) score compared to placebo (-4.4 vs -3.3) in patients with CKD-associated pruritus.
   

Published Research Updates

1. In the 24-hour, 768 patient, Phase III, open-label ATHENA trial, treatment with Travena’s oliceridine reduced the NRS pain score by 2.2 points at 30 mins from a baseline score of 6.3 in surgical (94%) and non-surgical patients with moderate to severe acute pain.
   
2. In the 184 patient, Phase II, open-label, DESTINY-Breast01 trial, treatment with AstraZeneca/ Daiichi Sankyo’strastuzumab deruxtecan resulted in an objective response rate of 60.9% of patients with HER2 positive metastatic breast cancer previously treated with trastuzumab emtansine.
   
3. In a 21-week, 38-patient, Phase II trial, 46% of patients treated with Argenx’ efgartigimod added to standard of care therapy improved their platelet count to 50x109/L during two or more visits compared to 25% in the placebo cohort in patients with primary immune thrombocytopenia (ITP).
   
4. A 52-week, 172 patient, open-label extension of a 14-week efficacy trial examined the safety for Daiichi Sankyo’s mirogabalin and found the most common adverse effect to be nasopharyngitis, diabetic retinopathy, peripheral edema, somnolence, diarrhea, increased weight, and dizziness in Asian patients with diabetic peripheral neuropathic pain.
   
5. ​In the 1-year, 480 patient, Phase II, HER2CLIMB trial, treatment Seattle Genetics tucatinib in combination with trastuzumab and capecitabine resulted in progression-free survival in 33.1% of patients compared to 12.3% with trastuzumab and capecitabine alone in patients with locally advanced unresectable or metastatic HER2-positive breast cancer who were previously treated with trastuzumab, pertuzumab and trastuzumab emtansine.