Fri, Oct. 14, 8:03 AM
- The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopts a positive opinion recommending approval of Intercept Pharmaceuticals' (NASDAQ:ICPT) obeticholic acid (OCA), branded as Ocaliva, for the treatment of primary biliary cholangitis (PBC), a chronic liver disease formerly known as primary biliary cirrhosis that is characterized by the progressive destruction of the bile ducts.
- OCA, in combination with UDCA (ursodeoxycholic acid), was approved in the U.S. in May for the treatment of PBC. UDCA is the only approved treatment for PBC in Europe, but the company says that a large proportion of patients receiving UDCA continue to experience elevations in a liver enzyme called alkaline phosphatase (ALP), a biomarker of liver stress.
- UDCA (tablet formulation) is the property of privately held Asklepion Pharmaceuticals. Retrophin (NASDAQ:RTRX) acquired the rights to the liquid formulation in June.
- OCA is an agonist of the farnesoid X receptor, a nuclear receptor expressed in the liver and intestine that is a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways.
- A final decision from the European Commission usually takes ~60 days.
- ICPT is ahead 3% premarket but only on 685 shares.
Wed, Sep. 7, 12:43 PM
Wed, Sep. 7, 11:00 AM
Wed, Sep. 7, 9:17 AM
Wed, Sep. 7, 8:22 AM
- Micro cap Retrophin (NASDAQ:RTRX) is up 29% premarket on robust volume in response to its announcement of positive results in a Phase 2 clinical trial, DUET, evaluating sparsentan (RE-021) for the treatment of focal segmental glomerulosclerosis, a rare kidney disorder that leads to end-stage renal disease.
- The study met its primary endpoint of a statistically valid reduction (more than two-fold) in proteinuria (protein in urine) compared to irbesartan [Sanofi's (NYSE:SNY) Avapro] after an eight-week treatment period. Specifically, patients treated with 200, 400 and 800 mg/day of sparsentan (n=64) showed a mean reduction of proteinuria from baseline of 44.8% after eight weeks of treatment compared to 18% for those (n=32) receiving 300 mg/day of irbesartan (p=0.006). The 400 mg and 800 mg sparsentan cohorts also showed superiority to 300 mg of irbesartan, 47.4% vs. 19.0% (p=0.011).
- The individual sparsentan cohorts, however, failed to show statistically valid superiority to irbesartan although clear signals of relative improvement were observed.
- The company plans to present the data at an upcoming medical conference or submit it for publication.
- Management will host a conference call this morning at 8:30 am ET to discuss the data.
Thu, Aug. 18, 8:46 AM
- Raptor Pharmaceuticals (NASDAQ:RPTP) is ahead 16% premarket on robust volume on the buzz that several companies are eyeing the Novato, CA firm, including Shire plc (NASDAQ:SHPG) who alleged has already bid $800M and Retrophin (NASDAQ:RTRX), founded by biotech bad boy Martin Shkreli.
- Raptor focuses its development efforts in rare diseases. Its pipeline includes programs in Huntington's disease, cancer, cystic fibrosis and mitochondrial diseases.
- It currently markets two products: PROCYSBI (cysteamine bitartrate) delayed-release capsules for nephropathic cystinosis and QUINSAIR (aerosolized form of levofloxacin) for the management of chronic pulmonary infections.
Thu, Aug. 4, 4:30 PM
Wed, Aug. 3, 5:35 PM
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Mon, Jun. 20, 5:01 PM
- Retrophin (NASDAQ:RTRX) secures the rights to a liquid formulation of ursodeoxycholic acid (UDCA) from privately held Asklepion Pharmaceuticals for the potential treatment of primary biliary cholangitis (PBC).
- Under the terms of the license agreement, Asklepion will receive an upfront payment of $0.5M and and up to $23.5M in sales-based milestones. It is eligible to receive up to $35.0M in milestones related to additional indications. Retrophin will assume all future program costs and will pay Asklepion tiered royalties on net sales.
- Retrophin says it will file a New Drug Application (NDA) for the PBC indication in 2017.
- UDCA, or ursadiol, is a naturally occurring hydrophilic bile acid derived from cholesterol. A liquid formulation would provide an attractive dosing alternative to patients who have difficulty swallowing pills.
- In late May, the FDA approved Intercept Pharmaceuticals' (NASDAQ:ICPT) Ocaliva (obeticholic acid), in combination with UDCA, for the treatment of PBC. Both are orally administered in tablet form.
Sun, Jun. 5, 8:28 AM
- Prosecutors have filed new charges accusing Martin Shkreli of engaging in more illegal maneuvers at his old drug company Retrophin (NASDAQ:RTRX) by using employees and consultants to conceal control of his stock.
- The new eight-count indictment, adding a conspiracy charge, sheds more light on the e-mail communications between Shkreli and his former corporate lawyer, Evan Greebel, who is accused of covering up securities fraud.
- A court hearing on the case is scheduled for tomorrow.
Tue, May 3, 4:05 PM
- Retrophin (NASDAQ:RTRX): Q1 EPS of $0.14 beats by $0.36.
- Revenue of $29M (+67.0% Y/Y) misses by $2M.
Mon, May 2, 5:35 PM
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Fri, Apr. 15, 11:46 AM
- Celator Pharmaceuticals (CPXX +4.6%) initiated with Buy rating and $24 (63% upside) price target by Needham.
- Retrophin (RTRX -1.7%) initiated with Outperform rating and $25 (67%upside) price target by BMO Capital.
- Fate Therapeutics (FATE -3.2%) initiated with Outperform rating and $4 (85% upside) price target by BMO Capital.
- Tobira Therapeutics (TBRA +4.4%) initiated with Buy rating and $22 price target by H.C. Wainwright.
- Wright Medical Group (WMGI +2.4%) initiated with Buy rating and $23 (21% upside) price target by Bank of America.
- Cleveland Research rates UnitedHealth Group (UNH -0.9%) a Buy with a $155 (22% upside) price target; Anthem (ANTM -0.5%) a Buy with a $170 (20% upside) price target and Aetna (AET -0.4%) Neutral with a $120 (10% upside) price target.
- Glaukos (GKOS +0.3%) initiated with Hold rating by Stifel.
- Baxter International (BAX -0.3%) upgraded to Overweight from Neutral by Piper Jaffray. Price target raised to $58 (36% upside) from $39.
- Baird upgrades Exactech (EXAC +0.7%) to Outperform from Neutral and raises the price target to $26 (18% upside) from $21. Stryker (SYK -0.9%) downgraded to Neutral from Outperform and raises the price target to $116 (6% upside) from $111.
- Tenet Healthcare (THC -2.5%) upgraded to Positive from Neutral by Susquehanna.
- Clovis Oncology (CLVS -1.5%) downgraded to Neutral from Overweight by JP Morgan. Price target lowered to $15 (7% upside) from $42.
- AstraZeneca (AZN +0.3%) downgraded to Sell from Neutral by Goldman Sachs. Price target lowered to 3,700p (11% downside risk) from 4,400p.
- Intercept Pharmaceuticals (ICPT +0.2%) downgraded to Underweight from Equal Weight by Morgan Stanley. Price target lowered to $80 (48% downside risk) from $100.
Thu, Feb. 25, 4:05 PM
- Retrophin (NASDAQ:RTRX): Q4 EPS of $0.07 beats by $0.29.
- Revenue of $30.4M (+117.0% Y/Y) beats by $1M.
Wed, Feb. 24, 5:35 PM
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Wed, Feb. 24, 11:23 AM
- The European Commission designates Retrophin's (RTRX -2%) RE-024 an Orphan Drug for the treatment of pantothenate kinase-associated neurodegeneration ((PKAN)), a rare and life-threatening genetic disorder caused by mutations in the PANK2 gene that results in the accumulation of iron in the brain. There are no approved treatments for the condition.
- The PANK2 gene encodes a crucial protein that phosphorylates (introduces a phosphate group) vitamin B5 (pantothenate) to phosphopantothenate. The disruption of this metabolic pathway leads to decreased levels of Coenzyme A (CoA), which plays a key role in many cellular functions.
- RE-024 is a small molecule that serves as phosphopantothenate replacement therapy for PKAN, which leads to the restoration of normal CoA levels.
- Among the benefits of Orphan Drug status in the EU is a 10-year period of market exclusivity for the indication, if approved.