Update for February 9, 2021

Regulatory Update
 
The FDA approved tepotinib (Tepmetko, EMD Serono) on 2/3/2021, for the treatment of metastatic non-small cell lung cancer harboring METex14 skipping alterations. Tepotinib will compete with capmatinib, which is also approved for the treatment of NSCLC harboring METex14 skipping alterations. Tepotinib is dosed 450 mg once daily. Capmatinib is dosed 400 mg twice a day. WAC for a month’s supply of capmatinib is $20,290. Both drugs have been evaluated as a treatment for NSCLC harboring METex14 skipping alterations. In the 152 patient, VISION trial, treatment with tepotinib resulted in an overall response rate of 46%. In the 364 patient GEOMETRY mono-1 trial, treatment with capmatinib resulted in an overall response rate of 41% in the pre-treated group and 68% in the treatment-naive group.
 
The FDA approved lisocabtagene maraleucel (Breyanzi, Bristol Myers Squibb), on 2/5/2021, for the treatment of relapsed or refractory large B-cell lymphoma, including diffuse large B-cell lymphoma, high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B. Lisocabtagene maraleucel was approved with a Black Warning regarding Cytokine Release Syndrome and Neurologic Toxicities. Lisocabtagene maraleucel is the third approved CAR-T cell therapy. Marketed CAR-T cell therapies include tisagenlecleucel (Kymriah, Novartis), approved in 2017 for the treatment of acute lymphoblastic leukemia and in 2018 for large B-cell lymphoma. The other CAR-T cell therapy is Gilead’s axicabtagene ciloleucel (Yescarta), which is approved to treat relapsed or refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and DLBCL arising from follicular lymphoma. Both drugs have the same Black Box Warning as lisocabtagene maraleucel. Tisagenlecleucel and axicabtagene ciloleucel have a WAC of $373,000 per dose, while BMS has set WAC for lisocabtagene maraleucel at $410,300 per dose.
 
The FDA approved umbralisib (Ukoniq, TG Therapeutics), on 2/5/2021, for the treatment of relapsed or refractory marginal zone lymphoma or refractory follicular lymphoma.
 
Announced Research Updates
 
BMS announced that in the 16-week, 1,020 patient, Phase III, POETYK PSO-2 trial, more patients treated with deucravacitinib achieved PASI 75 and a Physician's Global Assessment (sPGA) score of 0 to 1 compared to apremilast or placebo in patients with moderate to severe psoriasis. 
 
Mustang Bio plans to initiate a 10 patient, Phase III trial in 2021 to evaluate MB-107 in the treatment of XSCID. Results of the trail will be combined with 15 patents treated with MB-107 in earlier studies. The combined results will be compared to a historical group of XSCID patients treated with hematopoietic stem cell transplantation.
 
PTC announced that in a 40-week, 20 patient, Phase II trial, treatment with ataluren resulted in an insignificant increase of dystrophin levels in patients with Duchenne muscular dystrophy.
 
AZ announced that in the 823 patient, Phase III, open-label, KESTREL trial, neither durvalumab monotherapy nor durvalumab plus tremelimumab improved overall survival compared to cetuximab, fluorouracil, cisplatin or carboplatin in patients with recurrent or metastatic head and neck squamous cell cancer. 
 
Published Research Updates
 
A long-term extension of 1,820 patients who completed the SIRROUND-D and SIRROUND-T Phase III trials, found that a reduction in rheumatoid arthritis symptoms and control of disease with sirukumab were maintained. No new safety conversion were found. The most common adverse events were infections.
 
In an extension of the Phase II DARWIN 1 & 2 trials, 739 patients continued for a total of four or more years in the Phase II, DARWIN 3, open label trial. The ACR20/50/70 responses were 89.3%/69.6%/49.1% with filgotinib plus MTX and 91.8%/69.4%/44.4% with filgotinib alone.
 
In a 12-week, 300 patient, Phase IIb trial, reproxalap reduced signs and symptoms of dry eye disease compared to placebo in patients with dry eye disease. 
 
In a 120 patient, Phase II trial, treatment with pevonedistat and azacitidine resulted in overall survival of 21.8 months compared to 19 months with azacitidine alone in patients with higher-risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) or low-blast acute myeloid leukemia (LB-AML) who had not previously received a hypomethylating agent.
 
In a 16-week, 3,021 patient, Phase III trial, treatment with tanezumab 5mg administered subcutaneously every 8 weeks significantly improved the WOMAC Pain and Physical Function score, but not in the Patient's Global Assessment of Osteoarthritis score compared to naproxen, celecoxib or diclofenac in patients with moderate-to-severe osteoarthritis of the hip or knee. Tanezumab 2.5mg did not differ from placebo in either of the primary endpoints.
 
In the 16-week, 567 patient, Phase III, BE VIVID trial, 85% of patients treated with bimekizumab achieved a 90% Psoriasis Area and Severity Index (PASI 90) compared to 50% with ustekinumab and 5% with placebo in patients with moderate-to-severe chronic plaque psoriasis. An Investigator Global Assessment (IGA) score of clear or almost clear (IGA 0/1) was achieved by 84% of bimekizumab patients compared to 53% with ustekinumab and 5% with placebo. In the 16-week, 435 patient, Phase III, BE READY trial, 91% of patients treated with bimekizumab achieved PASI 90 and 93% achieved IGA of 0/1 compared to 1% and 1% with placebo in patients with moderate-to-severe chronic plaque psoriasis.