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GWPH Faces FDA Panel Tomorrow, NVCR Reports STELLAR Data, 1st XLH Drug Cleared


Today's Daily Dose brings you news about Arcus' phase I trial data of AB928; FDA briefing documents related to GW Pharma's Epidiolex; STELLAR results of Novocure and FDA approval of Rigel's ITP drug and Ultragenyx Pharma's X-linked hypophosphatemia drug.

Read on…

Arcus Biosciences Inc. (RCUS) presented encouraging results from its ongoing Phase 1 trial of AB928, its dual adenosine receptor antagonist, in healthy volunteers.

The compound has been shown to be safe and well tolerated at all doses evaluated and achieves near complete inhibition of A2aR adenosine receptor activation in blood samples from healthy volunteers," said Terry Rosen, CEO of the Company.

The trial includes a single-ascending-dose (SAD) portion as well as a multiple-ascending-dose (MAD) portion. In the SAD portion, single doses of 10, 25, 75 and 150 mg and a twice-daily dose of 100 mg have been evaluated. In the MAD portion, doses of 10, 25, 75 and 150 mg QD and 200 mg QD (with food) have been administered to subjects for four consecutive days.

Complete results from this trial, including pharmacodynamic data for the 200 mg BID (with food) dosing cohort, will be released following the unblinding of data in mid-2018, added the Company.

RCUS went public on the New York Stock Exchange on March 15, 2018, offering its shares at a price of $15 each. The stock closed Tuesday's trading at $16.95, up 1.44%.

GW Pharmaceuticals PLC (GWPH) rose more than 11% on Tuesday, following the release of favorable briefing documents for the advisory committee meeting scheduled for April 19, 2018, to review the NDA for Epidiolex.

Epidiolex is the Company's proprietary oral solution of pure plant-derived cannabidiol, or CBD. It is proposed for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome in patients 2 years of age and older.

Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) are rare, severe forms of epilepsy with onset in early childhood and poor long-term prognoses.

In the briefing document, the FDA staffers wrote, "Overall, CBD-OS (Epidiolex) provides a positive benefit-risk for patients with drug-resistant LGS or DS and can satisfy an unmet need by providing an additional treatment option to reduce the number of seizures in LGS and the first indicated treatment option for DS.

The FDA's final decision on Epidiolex is expected by June 27, 2018.

If approved, analysts expect Epidiolex to bring in excess of $1 billion in annual peak sales.

GWPH closed Tuesday's trading at $134.13, up 11.24%.

Novocure (NVCR) has reported positive data from its phase II pilot trial of Tumor Treating Fields in mesothelioma, dubbed STELLAR.

The STELLAR trial demonstrated clinically meaningful improvements in overall survival and progression free survival among patients who received Tumor Treating Fields plus standard of care chemotherapy, pemetrexed and cisplatin or carboplatin, compared to historical control data of patients who received standard of care chemotherapy alone.

According to the Company, the final data exceeded the results of the interim analysis presented in December 2016 at the International Association for the Study of Lung Cancer (IASLC) 17th World Conference on Lung Cancer for all efficacy endpoints. No device-related serious adverse events were reported.

Novocure will submit the full data for presentation at an upcoming medical conference.

Tumor Treating Fields in combination with standard of care chemotherapy is an investigational treatment for pleural mesothelioma and is not approved for this indication yet.

NVCR closed Tuesday's trading at $23.25, up 7.89%.

The FDA has approved Rigel Pharmaceuticals Inc.'s (RIGL) Tavalisse, an oral drug, for chronic immune thrombocytopenia in adult patients.

Immune thrombocytopenia, or ITP, is a bleeding disorder in which the blood doesn't clot due to a low number of blood cell fragments called platelets (Source: NIH).

The Company plans to launch Tavalisse in the United States in late May 2018. Analysts expect Tavalisse to rake in peak sales of $360 million.

Amgen's Nplate and GlaxoSmithKline/Ligand Pharma's Promacta are the other two FDA-approved drugs for ITP.

RIGL closed Tuesday's trading at $4.11, up 10.19%. In after-hours, the stock was up 12.90% to $4.64.

Burosumab, whose trade name is Crysvita, has been approved by the FDA for the treatment of X-linked hypophosphatemia (XLH) in adult and paediatric patients one year of age and older.

X-linked hypophosphatemia, or XLH, is a rare, hereditary renal phosphate-wasting disorder characterized by hypophosphatemia, rickets and/or osteomalacia, and diminished growth.

Developed by Ultragenyx Pharmaceutical Inc. (RARE) and Kyowa Hakko Kirin Co. Ltd., Crysvita is an antibody that blocks fibroblast growth factor 23 (FGF23), a hormone that causes phosphate urinary excretion and suppresses active vitamin D production by the kidney.

Crysvita is the first drug to be approved by the FDA to treat X-linked hypophosphatemia.

RARE closed Tuesday's trading at $54.68, up 1.60%.

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