Wed, Apr. 22, 4:04 PM
- Hedge fund investor Kyle Bass, head of Dallas, TX-based Hayman Capital Management L.P., files a challenge to a key patent covering Biogen's (BIIB +0.6%) $2.9B Tecifera (dimethyl fumarate). Specifically, he is challenging the validity of U.S. Patent No. 8,759,393 entitled, "Utilization of Dialkylfumarates."
- Mr. Bass founded the group, Coalition for Affordable Drugs, which is targeting the '393 patent via a process called inter partes review, a type of patent challenge allowed by the 2012 American Invents Act. It was designed as a way to curtail the ballooning number of lawsuits by patent trolls. According to him, the group's raison d'etre is to stop companies from over-pricing drugs based on evergreening patents.
- Other companies in the crosshairs of the Coalition for Affordable Drugs include Shire (NASDAQ:SHPG) and Acorda Therapeutics (NASDAQ:ACOR).
Tue, Apr. 21, 11:34 AM
- Sanofi's (SNY +0.4%) Genzyme unit initiates a Phase 2a clinical trial evaluating the pharmacodynamics of GZ/SAR402671, a new once-daily oral therapy for Fabry disease, a rare lysosomal storage disorder that results in the buildup of a type of fat, globotriasylceramide (GL-3), in the body's cells caused by a deficiency in a certain enzyme. It affects ~one in 50K males, predominantly, or ~10K total people worldwide.
- The primary endpoint of the nine-patient 26-week study is the reduction in accumulated GL-3 from the skin capillary endothelium. After its completion, all patients will have the option to enroll in an extension study.
- Genzyme's current offering for Fabry, Fabrazyme (agalsidase beta), replaces the deficient enzyme, alpha-galactosidase A. The FDA approved the intravenously-administered therapy in April 2003. It generated over $500M in sales for Sanofi last year. Annual therapy costs ~$200K per patient.
- Related tickers: (FOLD)(SHPG +1.8%)(PLX +1%)
Thu, Apr. 9, 12:11 PM
- Shire's (SHPG -2.8%) product candidate for cholestasis disorders, SHP625, failed to achieve the primary or secondary efficacy endpoints in a 13-week Phase 2 study (IMAGO) in Alagille syndrome, a rare, life-threatening genetic disorder characterized by liver damage caused by abnormalities in the bile ducts. The buildup of bile in the liver causes scarring (fibrosis) that prevents the liver from working properly.
- The primary endpoint was the change from baseline in serum bile acid compared to placebo. The secondary endpoint was itching (pruritus) as measured by the ItchRO instrument. Both endpoints were lower in the test group and placebo group compared to baseline. A post-hoc analysis showed a positive correlation between the percent change from baseline in serum bile acid and pruritis in the test group, but the placebo cohort was too small to assess the relationship.
- Despite the setback, the company remains committed to developing SHP625 in Alagille syndrome (AGLS). Two larger placebo-controlled trials in AGLS are ongoing. SHP625 is also being studied in progressive familial cholestasis, primary biliary cirrhosis and primary sclerosing cholangitis.
- Shire acquired the rights to SHP625 (LUM001) via its acquisition of Lumena Pharmaceuticals in May 2014.
Tue, Apr. 7, 3:58 PM
- Shire plc (SHPG +4.4%) is confident that it will prevail in a patent challenge from hedge fund Hayman Capital Management regarding its ulcerative colitis drug Lialda (mesalamine). The company won a previous challenge to its patent, which expires in 2020, in 2013 by Actavis (ACT -0.8%) unit Watson. No competitor has been able to get a generic mesalamine on the U.S. market, a fact that strengthens Shire's position.
- Generic firms aren't giving up, though. A trial versus Mylan is set to begin September 1.
- Lialda/Mezavant (brand name in Europe) generated almost $634M in sales for Shire in 2014.
Mon, Mar. 30, 1:03 PM
- According to Bloomberg, privately-held Rome-based drug firm Sigma-Tau Group is attracting interest from prospective bidders for its rare diseases business. The unit, valued as high as $1B, could be nice fit with Jazz Pharmaceuticals (JAZZ -0.3%) or Shire plc (SHPG -1%). Last year, Jazz acquired the rights to Sigma-Tau's defibrotide in the Americas for as much as $250M. No formal negotiations are underway, though, and the Cavazza family that owns the business may choose not to sell.
- Sigma-Tau, founded in 1957, generated sales of €697M ($757M) in 2013, with the rare diseases unit contributing €165M. The 220-employee unit focuses on blood diseases and cancer.
- Previously: Jazz kicks off Fast Track review process for VOD med (Dec. 12, 2014)
- Previously: Jazz secures rights to defibrotide in the Americas (July 2, 2014)
Fri, Mar. 27, 4:16 PM
- Brean Capital's Difei Yang says small cap Hyperion Therapeutics (NASDAQ:HPTX) should be on the acquisition radar considering Big Pharma's appetite for rare disease players. The company's revenues are generated by two products, Ravicti (glycerol phenylbutyrate) and Buphenyl (sodium phenylbutyrate), that treat urea cycle disorders, inherited metabolic diseases caused by a deficiency in one of the enzymes or transporters in the urea cycle. Elevated ammonia, a consequence of the failure to convert ammonia to urea, can have serious consequences, such as brain damage, coma and even death.
- Mr. Yang estimates that the company's takeout price at $60 per share. He lists Shire plc (NASDAQ:SHPG), Valeant Pharmaceuticals (NYSE:VRX), and Endo International (NASDAQ:ENDP) as potential suitors. HPTX closed today at just under $43.
- The company booked ~$114M in revenues last year but took a loss of ($6.3M).
Thu, Mar. 19, 7:41 AM
- Amicus Therapeutics (NASDAQ:FOLD) jumps 20% premarket on higher-than-normal volume in response to its announcement of accelerated schedules for its migalastat regulatory applications for the treatment of Fabry disease in the U.S. and Europe.
- Based on a Type C (late development stage) meeting with the FDA, the company intends to submit its New Drug Application (NDA) in H2. The agency agreed to review the application under its Accelerated Approval pathway. A Phase 4 (post-approval study) will be required so several protocol designs were discussed.
- Based on a meeting with the European Commission (EC), Amicus intends to submit its Marketing Authorization Application (MAA) in Q2 instead of mid-year. The company will request Accelerated Assessment of the MAA, which Rapporteurs (EC representatives assigned to provide guidance to application sponsors) indicated support for.
- Amicus will host a conference call this morning at 8:00 am ET to discuss its global regulatory strategy for migalastat monotherapy for Fabry disease.
- Related tickers: (NYSE:SNY) (NASDAQ:SHPG) (NYSEMKT:PLX)
Wed, Mar. 11, 10:56 AM
- Biotech investors, giddy over the robust demand for hepatitis C drugs like Gilead's (GILD +0.4%) Sovaldi and Harvoni, are eagerly anticipating the next wave of new therapies for non-alcoholic steatohepatitis (NASH) or "fatty liver disease." It resembles the alcoholic variety but affects people who drink little or no alcohol. According to the NIH, it affects 2 - 5% of Americans.
- Publicly-traded companies with NASH-related development programs include: Tobira Therapeutics (RGDO +2.3%), Islet Sciences (OTCQB:ISLT), Merck (MRK -0.5%), Intercept Pharmaceuticals (ICPT +3.4%), Gilead Sciences, Shire plc (SHPG -0.6%), Galectin Therapeutics (GALT), La Jolla Pharmaceutical (LJPC -1.5%), Conatus Pharmaceuticals (CNAT -1.2%), Raptor Pharmaceutical (RPTP -1.3%), Galmed Pharmaceuticals (GLMD +1.9%) and Genfit (OTCPK:GNFTF).
- TheStreet's Adam Feuerstein says that comparing clinical trial results between firms will require scrutiny because of differences in the designs of the studies, including efficacy criteria and treatment duration. In an article published today, he compares the ongoing trials of Genfit and Intercept. Genfit's efficacy endpoint of its mid-stage study is less stringent than Intercept's but it may make it more difficult to demonstrate a statistically significant benefit versus placebo. Results from Genfit's trial are expected later this month. If positive, it should rally NASH-related stocks.
Fri, Feb. 27, 9:59 AM
- Biogen Idec (BIIB -0.5%) and collaboration partner Swedish Orphan Biovitrum AB announce positive top-line results from a Phase 3 trial evaluating Alprolix (Coagulation Factor IX [Recombinant], Fc Fusion Protein) in children under age 12 with severe hemophilia B.
- Medical professionals recommend prophylactic treatment in children with severe hemophilia in order to manage and/or prevent bleeding episodes, but frequent administration schedules can be burdensome for patients and caregivers. Alprolix is administered once every 7 - 10 days for routine prophylaxis (prevention).
- In the study, children treated with Alprolix experienced an overall median annualized bleeding rate (ABR) of 1.97, while the median ABR for spontaneous joint bleeds was zero. About one third of the participants had no bleeding episodes at all. Overall, 91.7% of bleeding episodes were controlled by one or two infusions of Alprolix. None of the patients produced inhibitors (antibodies) to Alprolix in the study, which was the most significant potential complication of treatment.
- The European Medicines Agency requires the inclusion of pediatric study data in the initial Marketing Authorization Application (MAA) for a new hemophilia treatment. Interim data were used in the U.S. New Drug Application (NDA).
- Alprolix is the only approved hemophilia B therapy with prolonged circulation in the body. The FDA cleared it in March of last year. It generated $25.3M and $40.3M in sales for Biogen in Q3 and Q4, respectively.
- Related tickers: (BAX +0.3%)(OPK -0.8%)(SGMO -1.9%)(ALNY -1.9%)(ONCE +3%)(PFE -0.2%)(SHPG -1.6%)
Wed, Feb. 25, 3:28 PM
Tue, Feb. 24, 8:58 AM
- Shire plc (NASDAQ:SHPG) acquires San Diego, CA-based Meritage Pharma for an upfront payment of $70M plus milestones.
- Meritage's lead product candidate is an oral budesonide suspension for the treatment of eosinophilic esophagitis.
- Shire obtained the rights to acquire Meritage in connection with its buy of ViroPharma, which purchased the exclusive right to acquire Meritage in 2011.
- Previously: Shire extends offer for ViroPharma (Dec. 27, 2013)
Mon, Feb. 23, 12:32 PM
- Orphan drug developer PTC Therapeutics (PTCT +16.1%) is exploring selling itself amid rumors of interest from Shire plc (SHPG +1.9%) and BioMarin Pharmaceuticals (BMRN +1.8%).
- PTC's lead product candidate is Translarna (ataluren) for the treatment of patients with genetic disorders due to a nonsense mutation. It is in clinical development for Duchenne muscular dystrophy (DMD) caused by a nonsense mutation (nmDMD) and cystic fibrosis caused by a nonsense mutation (mnCF). The FDA designated it an Orphan Drug for both indications.
- The company's market cap is $2.2B while DMD competitor Sarepta Therapeutics is valued at $637M. BioMarin gobbled up the other DMD player, Prosensa Holding N.V. in November for $680M plus milestones.
- Previously: BioMarin buys Prosensa (Nov. 24, 2014)
Sun, Feb. 22, 6:53 AM
- Valeant Pharmaceuticals (NYSE:VRX) has agreed to buy Salix Pharmaceuticals (NASDAQ:SLXP) for about $10.1B, or $158 per share in cash, Bloomberg reports.
- Two other drugmakers were rumored to be on the hunt for Salix prior to the newly reported deal, including Endo International (NASDAQ:ENDP) and Shire (NASDAQ:SHPG).
- On Friday, Salix shares rose $7.11 to $157.85, almost completely eliminating any premium in the purchase price.
- Separately, Valeant received approval to buy the assets of bankrupt Dendreon (OTCPK:DNDNQ) for $495M. Dendreon is best known for its prostate cancer vaccine, Provenge.
Mon, Feb. 16, 5:50 PM
- A person close to the matter says Endo International (NASDAQ:ENDP) is and has been interested in acquiring Salix Pharmaceuticals (NASDAQ:SLXP). It has apparently sent Salix several letters stating its interest in a transaction, but Salix has yet to respond.
- Valeant Pharmaceuticals (NYSE:VRX) and Shire (NASDAQ:SHPG) are also rumored to be in hunt for the maker of products that treat gastrointestinal disorders.
- Previously: Valeant close to a bid for Salix (Feb. 12)
- Previously: Shire now on the hunt for Salix (Feb. 13)
Fri, Feb. 13, 1:58 AM
- Not only is Valeant Pharmaceuticals (NYSE:VRX) reportedly prepping a bid for Salix Pharmaceuticals (NASDAQ:SLXP), but Shire (NASDAQ:SHPG) is now rumored to be preparing an offer.
- Shire has taken initial steps towards a bid and is evaluating how to secure financing, sources told Reuters.
- Yesterday, Valeant Pharmaceuticals was reported to be arranging financing for a takeout of Salix at a likely price of more than $150 per share. Including net debt, Salix is valued at almost $11B.
- Previously: Valeant close to a bid for Salix (Feb. 12 2015)
- SLXP +1.2% premarket
Thu, Feb. 12, 5:33 PM
- Impax Laboratories (NASDAQ:IPXL) receives three Civil Investigative Demands (CIDs) from the Office of the Attorney General of the State of Alaska concerning its investigation into whether the company violated Alaskan state law by entering into settlement agreements with the respective brand name maker to delay the entry of generic versions of the products Adderall XR (NASDAQ:SHPG), Effexor XR (NYSE:PFE) and Opana ER (NASDAQ:ENDP).
- Such transactions are called "pay-to-delay" deals because the generic firm accepts a cash payment in exchange to delay the commercial launch of a lower-priced generic version to the branded product. This enables the branded manufacturer to maintain higher prices for an extended period.
- A CID is beefed-up subpoena which can require the recipient to produce documents, answer interrogatories or give testimony under oath.
- Impax plans to cooperate fully with the Alaska AG.
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