Arrowhead launches mid-stage study of RNAi drug in rare liver disorder

The first patient has been dosed in an open-label Phase 2 clinical trial, AROAAT2002, evaluating Arrowhead Pharmaceuticals’ (NASDAQ:ARWR) RNAi candidate ARO-AAT in patients with alpha-1 liver disease, a rare inherited liver disorder associated with alpha-1 antitrypsin deficiency (AATD), a Fast Track-tagged indication in the U.S.

The primary endpoint of the 12-subject study is the change from baseline to week 24 in Histological Liver Disease Activity Scale (Cohort 1) or week 48 (Cohort 2).

A pivotal trial, Phase 2/3 SEQUOIA, is in process.

https://seekingalpha.com/news/3527703-arrowhead-launches-mid-stage-study-of-rnai-drug-in-rare-liver-disorder

Stealth Bio’s elamipretide flunks late-stage study

Stealth BioTherapeutics (NASDAQ:MITO) is poised for a significant down move after announcing unsuccessful results from a Phase 3 clinical trial, MMPOWER-3, evaluating elamipretide in patients with primary mitochondrial myopathy (PMM), a group of inherited disorders due to mutations in mitochondrial DNA or nuclear DNA.

The study failed to achieve the primary endpoints of the change from baseline to week 24 in the six-minute walk test and change from baseline to week 24 in a fatigue scale called PMMSA.

Management will host a conference call this morning at 8:30 am ET to discuss the results.

Trading, currently suspended, will resume at 8:30 am ET.

Update: Shares down 59% on resumption of trade.

https://seekingalpha.com/news/3527718-stealth-bios-elamipretide-flunks-late-stage-pmm-study

State Medicaid programs on board with Novartis’ Adakveo for sickle cell

Moving extraordinarily quickly, Medicaid programs in Florida and Alabama have approved coverage of Novartis’ (NYSE:NVS) sickle cell disease (SCD) med Adakveo (crizanlizumab-tmca), approved by the FDA only a month ago.

The selectin-inhibiting monoclonal antibody costs ~$113K per year.

https://seekingalpha.com/news/3527722-state-medicaid-programs-on-board-novartis-adakveo-for-scd

Bausch Health in-licenses dry eye candidate from Novaliq GmgH

Bausch Health Companies (NYSE:BHC) unit Bausch + Lomb inks an agreement with Heidelberg, Germany-based Novaliq GmbH for exclusive U.S. and Canadian rights to NOV03 (perfluorohexyloctane) for the potential treatment of dry eye disease associated with Meibomian gland dysfunction (gland that secretes oils onto the surface of the eye).

Under the terms of the deal, Novaliq will receive upfront and milestone payments and royalties on net sales. Specific financial terms are not disclosed. BHC has the right to pursue additional ophthalmic indications for NOV03, alone and in combination (Novaliq will be eligible for additional payments and royalties for the combo products).

A Phase 2 trial was successful, achieving the primary endpoint of a statistically significant improvement of total corneal fluorescein staining at week 8 versus placebo.

A Phase 3 study is in process. BHC plans to start another Phase 3 in 2020.

https://seekingalpha.com/news/3527736-bausch-health-in-licenses-dry-eye-candidate-from-novaliq-gmgh

Gilead’s Vosevi OK’d in China

The China National Medical Products Administration has approved Gilead Sciences’ (NASDAQ:GILD) Vosevi (sofosbuvir 400mg/velpatasvir 100mg/voxilaprevir 100mg) for the once-daily treatment of chronic hepatitis C virus (HCV) infection in adults without cirrhosis or with compensated cirrhosis who have failed prior treatment with direct-acting antiviral therapy.

There are ~10M Chinese with HCV infection.

https://seekingalpha.com/news/3527739-gileads-vosevi-okd-in-china

Congress approves raising tobacco age to 21

The minimum age to purchase cigarettes, e-cigarettes and other tobacco products will be raised to 21 nationwide under the spending bill passed by the Senate on Thursday.

The two biggest U.S. cigarette manufacturers, Altria (NYSE:MO) and Reynolds American (NYSE:BTI), both supported the age restriction, as did Juul Labs (JUUL), which has been sharply criticized over a surge in underage vaping.

Nineteen states have already passed laws raising the minimum tobacco purchase age to 21, including California, Texas and New York.

Selected tickers: PM, VGR, OTCPK:GLLA, OTCPK:VAPE, OTCQX:IMBBY

https://seekingalpha.com/news/3527657-congress-approves-raising-tobacco-age-to-21

Johnson & Johnson to acquire remainder of Verb Surgical

On the heels of its successful collaboration with Alphabet’s Verily Life Sciences, Johnson & Johnson (NYSE:JNJ) has agreed to acquire the remaining stake in robotics and data science venture Verb Surgical for an undisclosed sum. The transaction should close in H1 2020.

Verb was a joint venture formed between J&J unit Ethicon and Google Life Sciences in late 2015.

https://seekingalpha.com/news/3527678-johnson-johnson-to-acquire-remainder-of-verb-surgical