Vertex price target raised to $330 from $290 at Goldman Sachs » 07:1307/0207/02/20
Goldman Sachs analyst…
Goldman Sachs analyst Salveen Richter raised the firm's price target on Vertex Pharmaceuticals (VRTX) to $330 from $290 and keeps a Conviction Buy rating on the shares. The stock's 31% rally year-to-date is due to earnings execution on the cystic fibrosis franchise through lead revenue driver Trikafta, Richter tells investors in a research note. At current share levels, the analyst sees Vertex's cystic fibrosis opportunity as "largely appreciated." As such, Richter is turning to the company's "multi-modality pipeline as the next lever for growth." Following the positive Crispr Therapeutics (CRSP) partnered Phase 1/2 CTX001 CRISPR/Cas9 gene editing data in transfusion-dependent beta thalassemia and severe sickle cell disease, as well as a "supporting" physician survey, Richter introduced the program into the model at a 60% probability of success. Further, the analyst continues to see "significant opportunity" in Vertex's Phase 2 alpha-1 antitrypsin deficiency disorder program.
Vertex reimbursement in England 'a remarkable positive,' says Jefferies » 08:3507/0107/01/20
After Vertex announced a…
After Vertex announced a reimbursement agreement with England to include the new triple pill Kaftrio, Jefferies analyst Michael Yee called the news "a remarkable positive and first time we've seen England move this fast," noting that the agreement came several months ahead of the expected triple pill approval in the EU and the fact that the U.K. is typically one of the more challenging countries for reimbursement. Yee, who calls this pact an incremental positive, keeps a Buy rating and $295 price target on Vertex shares.
Vertex announces reimbursement agreement with NHS England » 08:4106/3006/30/20
Vertex Pharmaceuticals announced that it has expanded its reimbursement agreement with NHS England for Vertex's cystic fibrosis medicines to include KAFTRIO, in a combination regimen with KALYDECO 150 mg, ahead of the medicine's anticipated approval by the European Commission. Vertex said in a release, "The new expanded agreement includes reimbursed access to Vertex's currently licensed medicines - KALYDECO, ORKAMBI and SYMKEVI, as well as the triple combination therapy if approved - and any future additional licensed indications for all of these medicines."
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Piper says EU approval can help drive next leg of CF growth at Vertex » 11:0906/2606/26/20
Piper Sandler analyst…
Piper Sandler analyst Edward Tenthoff noted that the CHMP of the European Medicines Agency has issued a positive opinion for the Kaftrio triple therapy, adding that he is confident in European approval for the combo. EU approval could add 10,000 het-min patients who never had therapy and drive the next leg of cystic fibrosis growth for Vertex, said Tenthoff, who reiterates an Overweight rating and $302 price target on the shares.
Vertex announces CHMP positive opinion for KAFTRIO » 08:2606/2606/26/20
Vertex Pharmaceuticals announced that the European Medicines Agency's, or EMA, Committee for Medicinal Products for Human Use, or CHMP, adopted a positive opinion for KAFTRIO in a combination regimen with KALYDECO 150 mg to treat people with cystic fibrosis, or CF, ages 12 and older with one F508del mutation and one minimal function mutation, or F/MF, or two F508del mutations, or F/F, in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene. Vertex said in a release, "If approved, up to 10,000 people in Europe with CF ages 12 and older who have one F508del mutation and one minimal function mutation would be newly eligible for a medicine that targets the disease- causing protein defect. Additionally, people 12 years of age and older who have two F508del mutations and who are currently eligible for one of Vertex's other EMA-approved cystic fibrosis medicines would be eligible for the new triple combination regimen. The CHMP positive opinion was based on the results of two global Phase 3 studies in people with CF: a 24-week placebo-controlled study in people ages 12 years and older with one F508del mutation and one minimal function mutation and a 4-week head-to-head study of the triple combination therapy in comparison to tezacaftor/ivacaftor in people ages 12 years and older with two F508del mutations. Both Phase 3 studies showed statistically significant and clinically meaningful improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), which was the primary endpoint, and in all key secondary endpoints. In both studies, the ivacaftor/tezacaftor/elexacaftor plus ivacaftor combination regimen was generally well tolerated."
Vertex price target raised to $330 from $285 at SunTrust » 09:1606/2406/24/20
SunTrust analyst Robyn…
SunTrust analyst Robyn Karnauskas raised the firm's price target on Vertex to $330 from $285 and keeps a Buy rating on the shares. The analyst is updating her model to reflect the company's Trikafta launch metrics, along with additional color from the management on the launch and its conversion dynamics in the U.S. Cystic fibrosis market in the U.S. is approaching a plateau, but there is potential for continued growth in international markets from approval and country-based reimbursement agreements for Trikafta, the analyst tells investors in a research note.
Solid Biosciences appoints Ian Smith as chairman of board » 07:0406/1806/18/20
Solid Biosciences (SLDB)…
Solid Biosciences (SLDB) announced the appointment of board member Ian Smith as chairman of the board. Smith succeeds Andrey Zarur, Ph.D., who served as chairman of the board since 2013 and is one of the co-founders of Solid Biosciences. The appointment is effective June 16, 2020. Smith most recently serving as EVP and COO of Vertex Pharmaceuticals (VRTX).
Crispr update suggests 'potential curative effect' of CTX001, says Piper Sandler » 09:1906/1206/12/20
Piper Sandler analyst…
Piper Sandler analyst Edward Tenthoff noted that Crispr Therapeutics (CRSP) and partner Vertex (VRTX) updated Phase I/II CTX001 data on two transfusion-dependent beta-thalassemia patients and one sickle cell disease patient at the virtual EHA conference. The update shows continued durable response, suggesting the "potential curative effect of CTX001," according to Tenthoff, who reiterates his Overweight rating and $104 price target on Crispr shares following the release of the data.
Crispr Therapeutics, Vertex announce new CTX001 data from CLIMB trials » 06:2506/1206/12/20
Crispr Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX) announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia, or TDT, and severe sickle cell disease, or SCD, and highlighted recent progress in the CTX001 development program. These data were presented during an oral presentation at the European Hematology Association, or EHA. Data presented at EHA demonstrate clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated. CRISPR Therapeutics and Vertex announced initial data for the first TDT patient in November of 2019. Patient 1 with TDT has the ss0/IVS-I-110 genotype, which is associated with a severe phenotype similar to ss0/ss0, and had a transfusion requirement of 34 units of packed red blood cells per year before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. After CTX001 infusion, two serious adverse events, or SAEs, occurred, neither of which the principal investigator, or PI, considered related to CTX001: pneumonia in the presence of neutropenia, and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. New data presented show that at 15 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL, and F-cells of 100%. Bone marrow allelic editing was 78.1% at 6 months and 76.1% at one year. Patient 2 with TDT has the ss0/IVS-II-745 genotype and had a transfusion requirement of 61 units of packed red blood cells per year before enrolling in the clinical trial. The patient achieved neutrophil engraftment 36 days after CTX001 infusion and platelet engraftment 34 days after infusion. After CTX001 infusion, two SAEs occurred, neither of which the PI considered related to CTX001: pneumonia and an upper respiratory tract infection; both subsequently resolved. At 5 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL, and F-cells of 99.4%. Data presented at EHA reflect longer-duration follow-up data for the first patient with SCD treated with CTX001. Crispr Therapeutics and Vertex announced initial data for this first SCD patient in November of 2019.Patient 1 with SCD experienced seven vaso-occlusive crises , or VOCs, and five packed red blood cell transfusions per year before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. After CTX001 infusion, three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis and abdominal pain; all subsequently resolved. New data presented today show that at 9 months after CTX001 infusion, the patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin, and F-cells of 99.7%. Bone marrow allelic editing was 81.4% at 6 months. Figure 2 presents the hemoglobin data over time for this patient. CLIMB-111 for TDT has dosed a total of 5 patients, and all patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of the first two patients. Additionally, CLIMB-111 has been expanded to allow enrollment of beta0/beta0 patients and is in the process of being expanded to allow enrollment of pediatric patients ages 12 years or older. CLIMB-121 for SCD has dosed a total of 2 patients and both patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of these first two patients. The initial safety profile in these trials appears to be consistent with myeloablative busulfan conditioning and an autologous hematopoietic stem cell transplant. In March, clinical trial sites in the U.S. and Europe temporarily paused their elective hematopoietic stem cell transplant programs due to the COVID-19 pandemic, and as a result, Crispr and Vertex temporarily paused conditioning and dosing in these trials. Enrollment, mobilization and drug product manufacturing in each trial remains ongoing. The companies are now in the process of re-initiating dosing with CTX001 at certain clinical trial sites. The CLIMB-111 and CLIMB-121 clinical trials are ongoing, and patients will be followed for 2 years following CTX001 infusion. The companies expect to provide additional data in the second half of 2020.
Vertex price target raised to $325 from $305 at Citi » 05:5906/1206/12/20
Citi analyst Mohit Bansal…
Citi analyst Mohit Bansal raised the firm's price target on Vertex to $325 from $305 and keeps a Buy rating on the shares, calling it his top pick in large cap pharmaceuticals given the "trifecta" of a growing base business, long patent life for its base and a robust pipeline. The company's pipeline for focal segmental glomerulosclerosis and opportunities in Type1 Diabetes, along with its CRISPR programs, are some of the longer-term opportunities Bansal highlighted in his note about its pipeline diversification.