BioMarin Pharmaceutical Inc.NASDAQ
Tue, Nov. 29, 3:28 PM
- Barclays' Geoff Meacham has downgraded BioMarin Pharmaceutical (BMRN -1.4%), Johnson & Johnson (JNJ -0.5%), Seattle Genetics (SGEN -0.2%), United Therapeutics (UTHR -4.1%) and Vertex Pharmaceuticals (VRTX -0.9%) to Equal Weight from Overweight.
- He trimmed BMRN's price target to $105 (21% upside) from $125 citing "moderating growth and an overall lack of catalysts in 2017."
- J&J's price target was sliced to $125 (11% upside) from $130 due to a "less compelling risk/reward profile."
- SGEN's fair value target was lowered to $53 (21% downside risk) from $70 due to valuation.
- UTHR's price target was revised to $100 (21% downside risk) from $115 citing the likely impact of Uptravi on Orenitram uptake.
- He lowered Vertex's price target to $90 (5% upside) from $100 citing the lack of upside for Kalydeco and Orkambi.
Fri, Nov. 18, 11:13 AM
- Barron's Ben Levisohn reports that Gilead Sciences (GILD -1.3%) has a range of attractive candidates should it decide to spend some its $32B cash hoard on an acquisition, a virtual certainty after the recent momelotinib disappointment.
- Considering the company's stated interest in oncology, obvious choices are Incyte (INCY -1.1%) and TESARO (TSRO -3%).
- Venturing out into the rare disease space, Alexion Pharmaceuticals (ALXN -1.6%), Vertex Pharmaceuticals (VRTX -1.6%) and BioMarin Pharmaceutical (BMRN -1.1%) are attractive fare.
- None of these companies are a surprise to biotech followers. All have been noted as buyout targets for some time now.
Fri, Nov. 11, 6:52 AM
- Valeant Pharmaceuticals (NYSE:VRX) downgraded to Neutral from Buy by Rodman & Renshaw. Price target lowered to $23 (37% upside) from $81.
- WellCare Health Plans (NYSE:WCG) downgraded to Neutral from Overweight by JPMorgan.
- Universal Health Services (NYSE:UHS) downgraded to Neutral from Buy by Bank of America. Price target lowered to $135 (12% upside) from $150.
- UnitedHealth Group (NYSE:UNH) downgraded to Neutral from Buy by Mizuho Securities.
- Globus Medical (NYSE:GMED) downgraded to Market Perform from Outperform by JMP Securities and William Blair. Downgraded to Market Perform from Outperform by Oppenheimer. Price target lowered to $23 (7% upside) from $29. Downgraded to Market Perform by Citigroup. Price target lowered to $23 (7% upside) from $29.
- Kindred Healthcare (NYSE:KND) downgraded to Sell from Hold by Stifel Nicolaus. Price target lowered to $5 (19% downside risk) from $10. Downgraded to Sector Perform by RBC Capital Markets and Royal Bank of Canada. Price target lowered to $9 (46% upside) from $16 by both.
- Impax Laboratories (NASDAQ:IPXL) downgraded to Underperform from Neutral with a $19 (32% upside) price target by Bank of America.
- BioMarin Pharmaceutical (NASDAQ:BMRN) downgraded to Neutral from Overweight by Piper Jaffray. Price target lowered to $84 (10% downside risk) from $105.
Mon, Oct. 31, 11:36 AM
- Shire plc (SHPG -3.3%) slumps in early trading on increased volume in apparent response to comments made by Express Scripts (ESRX +2.6%) Chief Medical Officer Steven Miller during a phone interview. He said the company is mulling ways to manage the rising costs of hemophilia medicines without forcing patients to switch treatments. Shire is a major player in the space after its merger with Baxalta. Hemophilia products accounted for over 46% of Baxalta's business in 2015.
- Related tickers: (OTCPK:BAYRY -0.7%)(PFE -0.2%)(NVO -0.4%)(BMRN -0.6%)(BAX +0.9%)(BIIB -0.4%)(SGMO +1.4%)
Thu, Oct. 27, 4:54 PM
- BioMarin Pharmaceutical (NASDAQ:BMRN) Q3 results ($M): 279.9 (+34.7%); Product Sales: 278.3 (+33.9%).
- Net Loss: (42.8) (+52.9%); Non-GAAP Net Income: 2.9 (+107.0%); Loss Per Share: (0.26) (+56.7%).
- Key Product Sales: Vimizim: 81 (+24.6%); Naglazyme: 78 (+44.4%); Kuvan: 91 (+42.2%); Aldurazyme: 24 (+14.3%).
- 2016 Guidance: Total Revenues: $1.10B - 1.15B (unch); Vimizim Sales: $340M - 360M (unch); Naglazyme Sales: $290M - 320M (unch); Kuvan Sales: $340M - 360M (unch); Net Loss: ($600M - 630M) from ($620M - 650M); Non-GAAP Net Loss: ($10M - 30M) from ($30M - 50M).
- Consensus view was a loss/share of ($0.39) on revenues of $289.4M.
- Shares are off 1% after hours on average volume.
Thu, Oct. 27, 4:07 PM
Wed, Oct. 26, 5:35 PM
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Tue, Oct. 25, 9:58 AM
- See J.P.Morgan note here
- JPM has a $13 price target on SGMO (vs. current $3.85).
- Says SGMO's hemophilia therapy may be more potent than BioMarin and Spark's.
Mon, Sep. 26, 8:10 AM
- The FDA designates Summit Therapeutics plc's (NASDAQ:SMMT) Orphan Drug-tagged ezutromid for Fast Track review for the treatment of Duchenne muscular dystrophy (DMD). Fast Track status provides for more frequent interactions with the FDA review team and a rolling review of the NDA.
- Ezutromid is currently being assessed in a Phase 2 study. Top-line data are expected in H2 2017.
- Unlike Sarepta Therapeutics' (NASDAQ:SRPT) eteplirsen or BioMarin Pharmaceutical's (NASDAQ:BMRN) drisapersen, which are restricted to DMD amenable to skipping exon 51 (~13% of DMD cases), ezutromid has the potential to treat all boys and young men with DMD. It modulates utrophin, a protein functionally and structurally similar to dystrophin, a protein that helps keep muscle cells intact and whose absence causes DMD.
- Previously: Summit moving ahead with DMD candidate, mid-stage study sites expand to the U.S. (April 26)
Tue, Sep. 20, 3:54 PM
- The good news keeps coming for Sarepta Therapeutics (SRPT +13.2%). Today the USPTO's Patent Trial and Appeals Board (PTAB) sided with Sarepta in its interference proceeding against BioMarin Pharmaceutical (BMRN +0.1%) related to patents covering "exon skipping."
- A year ago, the PTAB ruled in favor of BioMarin's claims on the use of exon 51 antisense oligonucleotides to treat Duchenne muscular dystrophy (DMD) specified in its U.S. Patent Application No. 14/198,992, a decision Sarepta appealed. BioMarin licensed the patent from Academisch Ziekenhuis Leiden. The ruling included the cancellation of Sarepta's U.S. Patent No. 8,486,907, which Sarepta licensed from the University of Western Australia.
Mon, Sep. 19, 6:55 PM
- Saying "we tried to be reasonable,", Sarepta Therapeutics (NASDAQ:SRPT) CEO Dr. Edward Kaye announces that the annual price of Exondys 51 (eteplirsen) will be ~$300K, apparently a lower price than some analysts predicted for the rare disease drug.
- The cost is comparable to Vertex Pharmaceuticals' (NASDAQ:VRTX) cystic fibrosis med Orkambi (ivacaftor/lumacaftor), but less than Alexion Pharmaceuticals' (NASDAQ:ALXN) Soliris (eculizumab), BioMarin Pharmaceutical's (NASDAQ:BMRN) Naglazyme (galsulfase) and Vimizim (elosulfase alfa) and Shire's (NASDAQ:SHPG) Elaprase (idursulfase).
- CureDuchenne advocacy group rep Debra Miller says, "Considering the cost of manufacturing, delivering and supporting patients (financial assistance programs), not to mention development costs over a decade, I don't think it's too much." Payer reimbursement should commence in no more than 90 days.
Thu, Sep. 15, 10:43 AM
- The European Medicines Agency validates BioMarin Pharmaceutical's (BMRN -1%) Marketing Authorization Application (MAA) seeking approval of enzyme replacement therapy Brineura (cerliponase alfa) for the treatment of CLN2 disease, a form of Batten disease, a rare inherited disorder in which the body's cells lose their ability to dispose of wastes. Validation means that the application is complete and the review process begins.
- The company requested accelerated review for the Orphan Drug-designated use, but the shortened time line is not guaranteed. Assuming a positive opinion from CHMP and the standard 210-day review period, a decision should be announced no later than Q3 of next year.
- The marketing application in the U.S. is currently under review with an action date of April 27, 2017.
Wed, Sep. 7, 7:24 AM
- Per the FDA's request, BioMarin Pharmaceutical (NASDAQ:BMRN) submits long-term efficacy data supporting the use of cerliponase alfa in children with CLN2 disease, a form of Batten disease, a rare inherited disorder in which the body's cells lose their ability to dispose of wastes. The majority of affected children are no longer able to walk and talk by age six.
- Data from an ongoing extension study showed that after 81 weeks, patients treated with cerliponase alfa continued to have motor-language scores representing substantial attenuation of disease progression compared to natural history. The results are consistent with what was observed at 48 weeks.
- The FDA requested the long-term data on the basis of its initial review of the company's Biologics License Application (BLA). The agency designated the submission as a major amendment so its action date (PDUFA) has been extended three months to April 27, 2017. An advisory committee meeting will also be held, although the specific date has not been set.
- Orphan Drug- and Breakthrough Therapy-tagged cerliponase alfa is an enzyme replacement therapy, specifically a recombinant form of human tripeptidyl peptidase (TPP1), the enzyme deficient in CLN2.
Thu, Aug. 25, 9:14 AM
- The FDA designates Mallinckrodt's (NYSE:MNK) Synacthen (tetracosactide) depot formulation (long acting) for Fast Track review for the treatment of Duchenne muscular dystrophy (DMD). A Phase 1 study is underway.
- Synacthen, a 24-amino acid melanocortin receptor agonist, is a synthetic version of the natural hormone ACTH (adrenocorticotrophic hormone). It is currently approved ex-U.S. to test if the adrenal glands are working properly and to treat adrenocortical insufficiency.
- DMD is an inherited disorder caused by the absence of a protein called dystrophin. It is characterized by progressive muscle degeneration and weakness.
- Fast Track status provides for more frequent interactions with the FDA review team and a rolling review of the New Drug Application (NDA).
- DMD-related tickers: (NASDAQ:SRPT)(NASDAQ:BMRN)(NASDAQ:SMMT)(NASDAQ:PTCT)(NYSE:PFE)(OTCQB:MRNA)(NASDAQ:CATB)
Wed, Aug. 10, 9:44 AM
- The FDA accepts privately held Marathon Pharmaceuticals, LLC's New Drug Applications (NDAs) seeking approval of deflazacort for the treatment of Duchenne muscular dystrophy (DMD). The agency will review the applications under Priority Review status which shortens the review clock to six months from the usual 10 months.
- The company submitted two NDAs, one for an oral suspension formulation and one for an immediate-release tablet formulation.
- The FDA action date (PDUFA) should be in February 2017.
- Deflazacort is a steroid called a glucocorticoid. It is a derivative of prednisone with less side effects.
- DMD-related tickers: (SRPT -8.8%)(BMRN -1%)(SMMT)(PTCT -1.2%)(PFE -0.2%)(OTCQB:MRNA)
Thu, Aug. 4, 4:06 PM