Biopharm News: Portola PTLA surges 47% on FDA A
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Posted On: 06/23/2017 9:52:20 PM
Biopharm News:
Portola PTLA surges 47% on FDA Approval; AVEO soars 72% on positive European recommendation
Portola Pharmaceuticals Inc (NASDAQ
TLA) surged to close up 47% to $56.06 following its intra-day FDA approval announcement for Bevyxxa (betrixaban) for the treatment of venous thromboembolism (VTE), or blood clots. Friday’s surge was largely due to uncertainty whether the FDA would issue an approval or not, as Portola’s Phase 3 trial missed its initial key endpoint.
AVEO Oncology (NASDAQ:AVEO) shares soared to close up 72% to $1.25 following news that the Committee for Medicinal Products for Human Use (CHMP) of European Medicines Agency (EMA), has recommended FOTIVDA (tivozanib) for approval as a treatment for patients with advanced renal cell carcinoma (RCC). A final decision is due in about 67 days. Its U.S. pivotal trial in third-line RCC, the TIVO-3 trial, is due to report futility analysis in mid-2017, with top-line data due 1Q 2018.
Cara Therapeutics, Inc. (Nasdaq:CARA) shares closed up 14% to $22.65 following news that the FDA has granted Breakthrough Therapy designation to I.V. CR845 for the treatment of moderate-to-severe uremic pruritus (UP) in chronic kidney disease (CKD) patients undergoing hemodialysis. The company is also awaiting data next week from its Phase 2b trial of CR845 in patients with Osteoarthritis (OA).
Stemline Therapeutics, Inc. (Nasdaq: STML) announced updated data from Stages 1 and 2 from its ongoing pivotal Phase 2 trial of SL-401 in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). The data showed an overall response rate (ORR) was 84%, with a complete response (CR) rate of 59%. Shares closed up 7% to $9.10. Further data are due 2H 2017 with a BLA filing due 4Q 2017 or 1Q 2018.
bluebird bio, Inc. (Nasdaq: BLUE) shares dipped to close down 5.5% to $112.00. The company announced early interim data from the first 3 patients in its ongoing Northstar-2 (HGB-207) Phase 3 trial of LentiGlobin for the treatment of transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes. The company noted promising data from the first patient treated that saw normal haemoglobin levels six months after treatment (13.3 g/dL). The second patient showed the level of hemoglobin after three months was improving but less than the first patient, which likely caused the pullback in its share price Friday together with profit-taking, given that even with Friday's retracement the stock has climbed 48% during the first three weeks of June alone.
Ocular Therapeutix Inc (NASDAQ:OCUL) shares closed down 12% to $10.15 following news that its current CEO, Dr. Amar Sawhney, will step down from his position on or before September 30, 2017.
Major price movers (stocks priced > $1.00, volume > 100k):
ADVANCERS:
Ascendis Pharma A/S (NASDAQ:ASND): $26.36; +14%.
Immune Design Corp (NASDAQ:IMDZ): $9.65; +14%.
Idera Pharmaceuticals Inc (NASDAQ:IDRA): $1.93; +12%.
GlycoMimetics Inc (NASDAQ:GLYC): $13.41; +11%.
Repligen Corporation (NASDAQ:RGEN): $44.20; +10%.
DECLINERS:
Invivo Therapeutics (NASDAQ:NVIV): $2.85; -22%.
Titan Pharmaceuticals, Inc. (NASDAQ:TTNP): $2.15; -10%.
AcelRx Pharmaceuticals Inc (NASDAQ:ACRX): $2.15; -10%.
Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR): $1.55; -10%.
Infinity Pharmaceuticals Inc. (NASDAQ:INFI): $1.81; -10%.
Pipeline updates below:
DRUG STAGE CATALYST
TICKER: BLCM
BPX-501 - BP-004
Adjunct T-cell therapy administered after allogeneic HSCT - (hematopoietic stem cell transplantation) - cancer
PHASE 1/2 Phase 1/2 update June 23, 2017.
TICKER: BLUE
LentiGlobin - HGB-207 Northstar-2
non-β0/β0 transfusion-dependent thalassemia (TDT)
PHASE 3 Noted on December 14, 2016 that Phase 3 dosing has commenced. Early data released at EHA June 23, 2017.
TICKER: BLUE
HGB-205
Transfusion-Dependent β-Thalassemia (TDT) and Severe Sickle Cell Disease (SCD)
PHASE 1/2 Phase 1/2 data presented at EHA June 23, 2017.
TICKER: GBT
GBT440
Sickle cell disease in adolescents
PHASE 1/2 Phase 2a initiated June 2016. Preliminary data released at European Hematology Association (EHA) - June 23, 2017.
TICKER: KPTI
Selinexor - SADAL
Cancer - Diffuse Large B-Cell Lymphoma (DLBCL)
PHASE 2B Phase 2b additional interim data released June 23, 2017 at EHA. Final top-line data due 2H 2018.
TICKER: NVO
Semaglutide (NN9536)
Obesity
PHASE 2 Phase 2 data released June 23, 2017 with Phase 3 trial to commence 2018.
TICKER: ONCE
SPK-9001
Hemophilia B
PHASE 1/2 Phase 1/2 data at ASH 2016. Update released April 6, 2017 with further update due July 10, 2017 at International Society on Thrombosis and Haemostasis (ISTH).
TICKER: PTI
PTI-428
Cystic fibrosis
PHASE 1 Phase 1 preliminary data from multiple ascending dose (MAD) cohorts due by the end of June 2017.
TICKER: PTLA
Bevyxxa (betrixaban)
Venous thromboembolism (VTE) Prevention
APPROVED FDA Approval noted June 23, 2017.
TICKER: QURE
AMT-060
hemophilia B
PHASE 1/2 Phase 1/2 long term updated data to be presented at International Society of Thrombosis and Hemostasis (ISTH) meeting, July 10, 2017. Pivotal trial to be initiated in 2018.
TICKER: RARE
KRN23 Burosumab
X-Linked Hypophosphatemia (XLH) - adult
PHASE 3 Phase 3 data released April 18, 2017 - primary endpoint met. BLA fiing due 2H 2017 for both adult and pediatric patients. Phase 3 pediatric data not required for BLA.
TICKER: STML
SL-401
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) cancer
PHASE 2 Phase 2 enrollment completed - noted March 23, 2017. Update on patients enrolled in Stages 1 and 2 at EHA June 23, 2017 with top-line data from Stage 3 due 2H 2017 with BLA filing due 4Q 2017 or 1Q 2018.
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Portola PTLA surges 47% on FDA Approval; AVEO soars 72% on positive European recommendation
Portola Pharmaceuticals Inc (NASDAQ
TLA) surged to close up 47% to $56.06 following its intra-day FDA approval announcement for Bevyxxa (betrixaban) for the treatment of venous thromboembolism (VTE), or blood clots. Friday’s surge was largely due to uncertainty whether the FDA would issue an approval or not, as Portola’s Phase 3 trial missed its initial key endpoint. AVEO Oncology (NASDAQ:AVEO) shares soared to close up 72% to $1.25 following news that the Committee for Medicinal Products for Human Use (CHMP) of European Medicines Agency (EMA), has recommended FOTIVDA (tivozanib) for approval as a treatment for patients with advanced renal cell carcinoma (RCC). A final decision is due in about 67 days. Its U.S. pivotal trial in third-line RCC, the TIVO-3 trial, is due to report futility analysis in mid-2017, with top-line data due 1Q 2018.
Cara Therapeutics, Inc. (Nasdaq:CARA) shares closed up 14% to $22.65 following news that the FDA has granted Breakthrough Therapy designation to I.V. CR845 for the treatment of moderate-to-severe uremic pruritus (UP) in chronic kidney disease (CKD) patients undergoing hemodialysis. The company is also awaiting data next week from its Phase 2b trial of CR845 in patients with Osteoarthritis (OA).
Stemline Therapeutics, Inc. (Nasdaq: STML) announced updated data from Stages 1 and 2 from its ongoing pivotal Phase 2 trial of SL-401 in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). The data showed an overall response rate (ORR) was 84%, with a complete response (CR) rate of 59%. Shares closed up 7% to $9.10. Further data are due 2H 2017 with a BLA filing due 4Q 2017 or 1Q 2018.
bluebird bio, Inc. (Nasdaq: BLUE) shares dipped to close down 5.5% to $112.00. The company announced early interim data from the first 3 patients in its ongoing Northstar-2 (HGB-207) Phase 3 trial of LentiGlobin for the treatment of transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes. The company noted promising data from the first patient treated that saw normal haemoglobin levels six months after treatment (13.3 g/dL). The second patient showed the level of hemoglobin after three months was improving but less than the first patient, which likely caused the pullback in its share price Friday together with profit-taking, given that even with Friday's retracement the stock has climbed 48% during the first three weeks of June alone.
Ocular Therapeutix Inc (NASDAQ:OCUL) shares closed down 12% to $10.15 following news that its current CEO, Dr. Amar Sawhney, will step down from his position on or before September 30, 2017.
Major price movers (stocks priced > $1.00, volume > 100k):
ADVANCERS:
Ascendis Pharma A/S (NASDAQ:ASND): $26.36; +14%.
Immune Design Corp (NASDAQ:IMDZ): $9.65; +14%.
Idera Pharmaceuticals Inc (NASDAQ:IDRA): $1.93; +12%.
GlycoMimetics Inc (NASDAQ:GLYC): $13.41; +11%.
Repligen Corporation (NASDAQ:RGEN): $44.20; +10%.
DECLINERS:
Invivo Therapeutics (NASDAQ:NVIV): $2.85; -22%.
Titan Pharmaceuticals, Inc. (NASDAQ:TTNP): $2.15; -10%.
AcelRx Pharmaceuticals Inc (NASDAQ:ACRX): $2.15; -10%.
Arrowhead Pharmaceuticals Inc (NASDAQ:ARWR): $1.55; -10%.
Infinity Pharmaceuticals Inc. (NASDAQ:INFI): $1.81; -10%.
Pipeline updates below:
DRUG STAGE CATALYST
TICKER: BLCM
BPX-501 - BP-004
Adjunct T-cell therapy administered after allogeneic HSCT - (hematopoietic stem cell transplantation) - cancer
PHASE 1/2 Phase 1/2 update June 23, 2017.
TICKER: BLUE
LentiGlobin - HGB-207 Northstar-2
non-β0/β0 transfusion-dependent thalassemia (TDT)
PHASE 3 Noted on December 14, 2016 that Phase 3 dosing has commenced. Early data released at EHA June 23, 2017.
TICKER: BLUE
HGB-205
Transfusion-Dependent β-Thalassemia (TDT) and Severe Sickle Cell Disease (SCD)
PHASE 1/2 Phase 1/2 data presented at EHA June 23, 2017.
TICKER: GBT
GBT440
Sickle cell disease in adolescents
PHASE 1/2 Phase 2a initiated June 2016. Preliminary data released at European Hematology Association (EHA) - June 23, 2017.
TICKER: KPTI
Selinexor - SADAL
Cancer - Diffuse Large B-Cell Lymphoma (DLBCL)
PHASE 2B Phase 2b additional interim data released June 23, 2017 at EHA. Final top-line data due 2H 2018.
TICKER: NVO
Semaglutide (NN9536)
Obesity
PHASE 2 Phase 2 data released June 23, 2017 with Phase 3 trial to commence 2018.
TICKER: ONCE
SPK-9001
Hemophilia B
PHASE 1/2 Phase 1/2 data at ASH 2016. Update released April 6, 2017 with further update due July 10, 2017 at International Society on Thrombosis and Haemostasis (ISTH).
TICKER: PTI
PTI-428
Cystic fibrosis
PHASE 1 Phase 1 preliminary data from multiple ascending dose (MAD) cohorts due by the end of June 2017.
TICKER: PTLA
Bevyxxa (betrixaban)
Venous thromboembolism (VTE) Prevention
APPROVED FDA Approval noted June 23, 2017.
TICKER: QURE
AMT-060
hemophilia B
PHASE 1/2 Phase 1/2 long term updated data to be presented at International Society of Thrombosis and Hemostasis (ISTH) meeting, July 10, 2017. Pivotal trial to be initiated in 2018.
TICKER: RARE
KRN23 Burosumab
X-Linked Hypophosphatemia (XLH) - adult
PHASE 3 Phase 3 data released April 18, 2017 - primary endpoint met. BLA fiing due 2H 2017 for both adult and pediatric patients. Phase 3 pediatric data not required for BLA.
TICKER: STML
SL-401
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) cancer
PHASE 2 Phase 2 enrollment completed - noted March 23, 2017. Update on patients enrolled in Stages 1 and 2 at EHA June 23, 2017 with top-line data from Stage 3 due 2H 2017 with BLA filing due 4Q 2017 or 1Q 2018.
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