Wed, Oct. 7, 12:14 PM
- The FDA grants rare pediatric disease designation for privately held Marathon Pharmaceuticals' deflazacort for the treatment of Duchenne muscular dystrophy. The status supplements its Orphan Drug tag issued by the agency in August 2013. Daflazacort also has Fast Track status which allows for more frequent interactions with the FDA review team and a rolling review of the NDA, expected to be filed in H1 2016.
- If the NDA is cleared, the company will receive a Rare Pediatric Disease Priority Review Voucher that it can redeem for Priority Review (six-month review clock) for a subsequent product or sell it to a third party. The vouchers are quite valuable. A little more than a year ago, AbbVie (ABBV +1.7%) bought one from United Therapeutics (UTHR -2.4%) for $350M in cash.
- Deflazacort is a steroid hormone called a glucocorticoid which has anti-inflammatory and immunosuppressant properties.
- DMD-related tickers: (SRPT +1.3%)(BMRN -1%)(SMMT +5.9%)(CATB +0.4%)(PFE +0.5%)(FGEN)(OTCQB:MRNA -1.8%)(CAPR -5%)(OTCPK:SPHDF)(PTCT +12%)
Wed, Sep. 30, 9:18 AM
- United Therapeutics (NASDAQ:UTHR) settles its litigation with Sandoz (NYSE:NVS) over the latter's Abbreviated New Drug Application (ANDA) seeking clearance for a generic version of Remodulin (treprostinil). Under the terms of the settlement agreement, United grants Sandoz a non-exclusive license to manufacture and commercialize its generic version beginning June 26, 2018, earlier under certain circumstances.
- The license does not allow Sandoz to make generic versions of Tyvaso (treprostinil) Inhalation Solution or Orenitram (treprostinil) Extended-Release Tablets nor convey any rights to its implantable and pump systems currently under development.
- Remodulin is United's top seller. It accounted for 39% ($136M) of its Q2 revenues.
Tue, Sep. 22, 5:08 PM
- United Therapeutics (NASDAQ:UTHR) subsidiary Lung Biotechnology PBC and privately held Synthetic Genomics (SGI) expand their multi-year research and development agreement to develop transplantation-ready pig organs using advances in synthetic genomics. The initial focus of the partnership, initiated in April of last year, was lung diseases and will now add kidney diseases.
- Under the terms of the agreement, SGI will receive royalties and milestones from the development and commercialization of the organs. Lung Biotech will make a $50M equity investment in SGI, which follows an earlier investment of $50M in SGI preferred stock. Additional financial details are not disclosed.
- United Therapeutics Chairman and Co-CEO Martine Rothblatt, Ph.D., says, "Our expanded collaboration with Synthetic Genomics is significant for applying our growing xenotransplantation science platform to the problem of end-stage renal failure. Our combined expertise will accelerate our efforts to develop an expanded supply of transplantable kidneys, potentially helping tens of thousands of patients suffering from incurable kidney disease."
Mon, Sep. 14, 9:00 AM
- The FDA designates Catabasis Pharmaceuticals' (NASDAQ:CATB) investigational CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD) a Rare Pediatric Disease, which the agency defines as a disease that primarily affects people younger than 18 years of age and fewer than 200K people in the U.S. If CAT-1004 is approved, the company will be eligible to receive a Rare Pediatric Disease Priority Review Voucher, which it can use or sell. The vouchers are valuable. AbbVie (NYSE:ABBV) recently bought one from United Therapeutics (NASDAQ:UTHR) for $350M.
- Orphan Drug- and Fast Track-tagged CAT-1004, currently in Phase 1/2 development, is an inhibitor of a protein called NF-kB, which plays a key role in muscle health.
Tue, Sep. 8, 7:40 AM
- The FDA clears privately held Wellstat Therapeutics' XURIDEN (uridine triacetate) for the treatment of hereditary orotic aciduria (HOA), a rare, potentially life-threatening genetic disorder in which sufferers cannot synthesize adequate amounts of uridine which leads to excessive excretion of orotic acid in the urine. This leads to a type of anemia, failure to thrive, various developmental problems and episodes of crystalluria (crystals in the urine). It affects less than one in 1M newborns.
- XURIDEN is an analog of the organic compound pyrimidine. Oral administration of XURIDEN delivers four to seven times more uridine than oral administration of uridine itself. The commercial launch will commence in early 2016.
- The FDA also grants the company a Rare Pediatric Disease Priority Review Voucher, which provides the holder Priority Review for a future marketing application after giving the regulator only a 90-day notice. It is an asset that can be sold or transferred an unlimited number of times. The vouchers are quite valuable. AbbVie (NYSE:ABBV) bought one from United Therapeutics (NASDAQ:UTHR) in August for $350M.
- Wellstat will transfer its voucher to AstraZeneca (NYSE:AZN) per a September 2014 agreement. Financial terms are not disclosed.
Wed, Aug. 19, 4:29 PM
- The FDA designates BioMarin Pharmaceutical's (NASDAQ:BMRN) drisapersen a treatment of a rare pediatric disease, in this case Duchenne muscular dystrophy. The agency's definition pertains to pediatric diseases affecting fewer than 200K Americans.
- Under the Rare Pediatric Disease Priority Review Voucher program, a company that receives approval for such a drug is eligible to receive a voucher that can be redeemed to obtain priority review for a subsequent marketing application for a different product. The voucher may be sold or transferred an unlimited number of times. The vouchers are quite valuable. United Therapeutics (NASDAQ:UTHR) just sold one to AbbVie (NYSE:ABBV) for $350M.
- Drisapersen is also designated an Orphan Drug and a Breakthrough Therapy to be reviewed under Fast Track and Priority Review protocols.
- Orphan Drug status provides for a seven-year period of market exclusivity for the indication, if approved.
- Previously: United Therapeutics sells Priority Review Voucher to AbbVie for $350M (Aug. 19)
Wed, Aug. 19, 7:17 AM
- AbbVie (NYSE:ABBV) buys United Therapeutics' (NASDAQ:UTHR) Rare Pediatric Disease Priority Review Voucher (PPRV) for $350M in cash. United was awarded the voucher when the FDA cleared Unituxin (dinutuximab) for the treatment of neuroblastoma in March.
- The main benefit of the voucher, which can be bought and sold an unlimited amount of times, is a six-month review clock. Also, the voucher owner only has to give the FDA a short 90-day notice of its intention to use it.
Tue, Jul. 28, 6:24 AM| Tue, Jul. 28, 6:24 AM | Comment!
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- Screen criteria: market cap >=$1B; average daily turnover >=400K shares; % change in mutual fund ownership (most recent qtr) >=0%; PEG ratio: <=1.0.
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- This is not a list of Buy/Sell recommendations, but rather a targeted list of stocks that may be suitable for more in-depth research.
Wed, May 20, 5:41 PM
Tue, Apr. 28, 7:31 AM
- United Therapeutics (NASDAQ:UTHR) Q1 results ($M): Total Revenues: 327.5 (+26.3%); Product Sales: 325.9 (+14.5%); Operating Expenses: 321.5 (+645.9%); Net Income: (16.6) (-112.1%); EPS: (0.36) (-114.8%); Non-GAAP EPS: 2.55 (+36.4%).
- Sales by Product: Remodulin: 146.3 (+7.5%); Tyvaso: 113.4 (+5.9%); Adcirca: 45.4 (+9.7%); Orenitram: 20.9.
- No guidance provided in press release.
Tue, Apr. 28, 6:03 AM
Mon, Apr. 27, 4:38 PM
Tue, Mar. 10, 12:34 PM
- The FDA approves United Therapeutics' (UTHR +0.4%) Unituxin (dinutuximab) injection, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2) and 13-cis-retinoic acid (RA), for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line therapy.
- Neuroblastoma is the most common extracranial solid cancer in children and the most common cancer in infancy, affecting ~700 patients each year. About half are diagnosed as high-risk.
- Unituxin, a maintenance therapy for neuroblastoma, is a chimeric biologic antibody that induces cell lysis (breakdown) of GD2-expressing cells. GD2 is an attractive target for cancer immunotherapies since it is strongly expressed in tumors of neuroectodermal origin while highly restricted on normal cells.
Mon, Mar. 9, 5:21 PM
- Rehovot, Israel-based SteadyMed (Pending:STDY) is set for its IPO of 4.25M shares of common stock at $12 - 14. Underwriters over-allotment is an additional 637.5K shares.
- The specialty pharmaceutical company focuses on the development and commercialization of therapeutics that address the limitations of market-leading products in certain orphan and other well-defined, high-margin specialty markets. Its lead product candidate is Trevyent, a small, pre-filled, single-use device that delivers parenteral treprostinil (Remodulin) at a pre-programmed rate to patients with pulmonary arterial hypertension. Its value proposition is a more convenient dosing regimen enabled by its PatchPump technology which delivers a steady flow of drug to the patient either intravenously or subcutaneously.
- The company intends to apply for Orphan Drug designation with the FDA by mid-year and submit an New Drug Application (NDA) by Q1 2016.
- Net losses for 2014, 2013 and 2012 were ($19.0M), ($7.9M) and ($4.1M), respectively. Operations consumed $13.4M in cash in 2014 and $7.5M in 2013.
- Related ticker: (NASDAQ:UTHR)
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United Therapeutics Corp is a biotechnology company. The Company is engaged in thedevelopment and commercialization of products to address the unmet medical needs of patients with chronic and life-threatening conditions.
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