BioPharm Updates:
Spark Therapeutics ONCE delivers promising initial hemophilia data; JNJ receives thumbs down for safety for rheumatoid arthritis candidate
Spark Therapeutics (NASDAQ:ONCE) announced positive preliminary data from its Phase 1/2 trial for SPK-8011 for the treatment of hemophilia A. The company noted:
During this time, there has been a steady and consistent rise in factor VIII activity levels that have now stabilized at levels of 11 percent and 14 percent of normal, respectively. Shares closed up 20% to $79.72. The company announced after-hours that it has commenced an underwritten public offering of $300m in shares of its common stock.
The Arthritis Advisory Committee voted 12-1 opposing that the safety profile of sirukumab (Johnson & Johnson (NYSE:JNJ) is adequate to support approval for the treatment of adult patients with moderately to severely active rheumatoid arthritis. Further commentary about the outcome can be found at Endpoints News.
Illumina, Inc. (NASDAQ: ILMN) shares closed up 15% following its report of strong 2Q earnings. The company noted revenue of $662 million, a 10% increase compared to $600 million in 2Q 2016. For fiscal 2017, it expects revenue to grow by 12%.
Agile Therapeutics, Inc. (Nasdaq:AGRX) announced that it intends to offer and sell shares of its common stock in an underwritten public offering. Shares are down 6% to $4.29 in the after-hours session.
Theravance Biopharma, Inc. (NASDAQ: TBPH) announced what it referred to as “positive results” from its Phase 2b study of velusetrag (TD-5108) for the treatment of patients with diabetic and idiopathic gastroparesis. However, deep down in its press release it noted that the primary endpoint was not met.
The primary endpoint analysis included a pre-specified analysis of each dose against placebo to report nominal p-values…patients in the 15 and 30 mg velusetrag study arms did not demonstrate nominally statistically significant improvements in gastroparesis symptoms versus placebo, possibly due to an increased frequency in gastrointestinal side effects at these doses that may have been caused by rapid emptying of the stomach. The lack of dose response resulted in a lack of statistical significance across the three doses when adjusted for multiplicity.
Other Major price movers (stocks priced > $1.00, volume > 100k):
Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume INCLUDING sub-$1 stocks.
ADVANCERS:
ADMA Biologics Inc (NASDAQ:ADMA): $3.47; +9%.
Corcept Therapeutics Incorporated (NASDAQ:CORT): $13.38; +9%.
Global Blood Therapeutics Inc (NASDAQ:GBT): $27.20; +8%.
Cerus Corporation (NASDAQ:CERS): $2.39; +8%.
ChemoCentryx Inc (NASDAQ:CCXI): $10.69; +7%.
DECLINERS:
Novan Inc (NASDAQ:NOVN): $4.54; -17%.
Lantheus Holdings Inc (NASDAQ:LNTH): $15.50; -17%.
AcelRx Pharmaceuticals Inc (NASDAQ:ACRX): $2.95; -13%.
Immune Pharmaceuticals Inc (NASDAQ:IMNP): $2.04; -7%.
Asterias Biotherapeutics Inc (NYSEMKT:AST): $3.30; -7%.
Pipeline updates below:
DRUG STAGE CATALYST
TICKER: ABBV
Imbruvica
Second-line Chronic graft-versus-host disease (GVHD)
APPROVED Approval announced August 2, 2017.
TICKER: AMRN
Vascepa REDUCE-IT outcomes trial
High Triglycerides With Mixed Dyslipidemia
– Interim analysis announced September 12, 2016 - trial to proceed. Second interim analysis due 3Q 2017. Final data due 2Q or 3Q 2018.
TICKER: AZN
Acalabrutinib
Relapsed or Refractory Mantle Cell Lymphoma
PDUFA PRIORITY REVIEW Priority Review announced August 2, 2017. Assume 6-month review rendering a PDUFA date on or around February 1, 2018.
TICKER: BCRX
BCX7353 - ZENITH-1
Angioedema attacks in patients with hereditary angioedema (HAE)
PHASE 2 Phase 2 initiation announced August 2, 2017.
TICKER: CRMD
Neutrolin - LOCK-IT 100
Hemodialysis patients with central venous catheters
PHASE 3 Phase 3 data due end of 2018 with interim analysis due 4Q 2017.
TICKER: CTIC
PIXUVRI - PIX306 Trial
B-cell non-Hodgkin lymphoma
PHASE 3 Phase 3 data due 1H 2018. Noted August 2, 2017 that enrollment has completed.
TICKER: JNJ
Sirukumab
Rheumatoid arthritis
PDUFA BLA filing announced September 23, 2016. PDUFA estimate September 23, 2017. Advisory Committee Meeting August 2, 2017 voted 12-1 against that safety data is adequate to support approval. 13-0 in favor of efficacy.
TICKER: KITE
Axicabtagene ciloleucel - KTE-C19 (ZUMA-5)
Indolent B-Cell Non-Hodgkin Lymphoma
PHASE 2 Phase 2 commencement of dosing announced August 2, 2017.
TICKER: MDGX
AmnioFix Injectable
Plantar Fasciitis
PHASE 3 Phase 3 trial to be initiated 3Q 2017.
TICKER: MEIP
Pracinostat in combination with Vidaza
Elderly patients with newly diagnosed acute myeloid leukemia (AML)
PHASE 3 Phase 3 dosing has commenced - noted August 2, 2017.
TICKER: ONCE
SPK-8011
Hemophilia A
PHASE 1/2 Phase 1/2 promising initial data released from first two patients - August 2, 2017. 11% and 14% Factor VIII.
TICKER: PCRX
EXPAREL
Single-dose injection femoral nerve block for total knee arthroplasty surgery
PHASE 3 Phase 3 data released July 25, 2017. Upper extremity met primary endpoint, lower extremity trial missed endpoint. sNDA filing due later in 2017.
TICKER: TBPH
Velusetrag (TD-5108)
Gastroparesis
PHASE 2B Phase 2b data released August 2, 2017. Primary endpoint not met.
Spark Therapeutics ONCE delivers promising initial hemophilia data; JNJ receives thumbs down for safety for rheumatoid arthritis candidate
Spark Therapeutics (NASDAQ:ONCE) announced positive preliminary data from its Phase 1/2 trial for SPK-8011 for the treatment of hemophilia A. The company noted:
During this time, there has been a steady and consistent rise in factor VIII activity levels that have now stabilized at levels of 11 percent and 14 percent of normal, respectively. Shares closed up 20% to $79.72. The company announced after-hours that it has commenced an underwritten public offering of $300m in shares of its common stock.
The Arthritis Advisory Committee voted 12-1 opposing that the safety profile of sirukumab (Johnson & Johnson (NYSE:JNJ) is adequate to support approval for the treatment of adult patients with moderately to severely active rheumatoid arthritis. Further commentary about the outcome can be found at Endpoints News.
Illumina, Inc. (NASDAQ: ILMN) shares closed up 15% following its report of strong 2Q earnings. The company noted revenue of $662 million, a 10% increase compared to $600 million in 2Q 2016. For fiscal 2017, it expects revenue to grow by 12%.
Agile Therapeutics, Inc. (Nasdaq:AGRX) announced that it intends to offer and sell shares of its common stock in an underwritten public offering. Shares are down 6% to $4.29 in the after-hours session.
Theravance Biopharma, Inc. (NASDAQ: TBPH) announced what it referred to as “positive results” from its Phase 2b study of velusetrag (TD-5108) for the treatment of patients with diabetic and idiopathic gastroparesis. However, deep down in its press release it noted that the primary endpoint was not met.
The primary endpoint analysis included a pre-specified analysis of each dose against placebo to report nominal p-values…patients in the 15 and 30 mg velusetrag study arms did not demonstrate nominally statistically significant improvements in gastroparesis symptoms versus placebo, possibly due to an increased frequency in gastrointestinal side effects at these doses that may have been caused by rapid emptying of the stomach. The lack of dose response resulted in a lack of statistical significance across the three doses when adjusted for multiplicity.
Other Major price movers (stocks priced > $1.00, volume > 100k):
Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume INCLUDING sub-$1 stocks.
ADVANCERS:
ADMA Biologics Inc (NASDAQ:ADMA): $3.47; +9%.
Corcept Therapeutics Incorporated (NASDAQ:CORT): $13.38; +9%.
Global Blood Therapeutics Inc (NASDAQ:GBT): $27.20; +8%.
Cerus Corporation (NASDAQ:CERS): $2.39; +8%.
ChemoCentryx Inc (NASDAQ:CCXI): $10.69; +7%.
DECLINERS:
Novan Inc (NASDAQ:NOVN): $4.54; -17%.
Lantheus Holdings Inc (NASDAQ:LNTH): $15.50; -17%.
AcelRx Pharmaceuticals Inc (NASDAQ:ACRX): $2.95; -13%.
Immune Pharmaceuticals Inc (NASDAQ:IMNP): $2.04; -7%.
Asterias Biotherapeutics Inc (NYSEMKT:AST): $3.30; -7%.
Pipeline updates below:
DRUG STAGE CATALYST
TICKER: ABBV
Imbruvica
Second-line Chronic graft-versus-host disease (GVHD)
APPROVED Approval announced August 2, 2017.
TICKER: AMRN
Vascepa REDUCE-IT outcomes trial
High Triglycerides With Mixed Dyslipidemia
– Interim analysis announced September 12, 2016 - trial to proceed. Second interim analysis due 3Q 2017. Final data due 2Q or 3Q 2018.
TICKER: AZN
Acalabrutinib
Relapsed or Refractory Mantle Cell Lymphoma
PDUFA PRIORITY REVIEW Priority Review announced August 2, 2017. Assume 6-month review rendering a PDUFA date on or around February 1, 2018.
TICKER: BCRX
BCX7353 - ZENITH-1
Angioedema attacks in patients with hereditary angioedema (HAE)
PHASE 2 Phase 2 initiation announced August 2, 2017.
TICKER: CRMD
Neutrolin - LOCK-IT 100
Hemodialysis patients with central venous catheters
PHASE 3 Phase 3 data due end of 2018 with interim analysis due 4Q 2017.
TICKER: CTIC
PIXUVRI - PIX306 Trial
B-cell non-Hodgkin lymphoma
PHASE 3 Phase 3 data due 1H 2018. Noted August 2, 2017 that enrollment has completed.
TICKER: JNJ
Sirukumab
Rheumatoid arthritis
PDUFA BLA filing announced September 23, 2016. PDUFA estimate September 23, 2017. Advisory Committee Meeting August 2, 2017 voted 12-1 against that safety data is adequate to support approval. 13-0 in favor of efficacy.
TICKER: KITE
Axicabtagene ciloleucel - KTE-C19 (ZUMA-5)
Indolent B-Cell Non-Hodgkin Lymphoma
PHASE 2 Phase 2 commencement of dosing announced August 2, 2017.
TICKER: MDGX
AmnioFix Injectable
Plantar Fasciitis
PHASE 3 Phase 3 trial to be initiated 3Q 2017.
TICKER: MEIP
Pracinostat in combination with Vidaza
Elderly patients with newly diagnosed acute myeloid leukemia (AML)
PHASE 3 Phase 3 dosing has commenced - noted August 2, 2017.
TICKER: ONCE
SPK-8011
Hemophilia A
PHASE 1/2 Phase 1/2 promising initial data released from first two patients - August 2, 2017. 11% and 14% Factor VIII.
TICKER: PCRX
EXPAREL
Single-dose injection femoral nerve block for total knee arthroplasty surgery
PHASE 3 Phase 3 data released July 25, 2017. Upper extremity met primary endpoint, lower extremity trial missed endpoint. sNDA filing due later in 2017.
TICKER: TBPH
Velusetrag (TD-5108)
Gastroparesis
PHASE 2B Phase 2b data released August 2, 2017. Primary endpoint not met.
1
'Everything works out in the end,,,If it hasn't worked out,,it's not the end'
https://twitter.com/twitter/statuses/957208055766241280
https://twitter.com/twitter/statuses/957208055766241280
