AcelRx +4% premarket on corporate update

AcelRx Pharmaceuticals (NASDAQ:ACRX) provides an update on its business and DSUVIA launch metrics.

166 healthcare facilities are REMS-certified and 148 formulary approvals have been achieved through December 31, 2019, exceeding year-end goals of 125 for each metric.

Year-end 2020 goals of 465 REMS-certified facilities and 465 formulary approvals.

FY 2019 total revenues of $2.3M.

Cash and short-term investments of $66.1M as of December 31, 2019.

Department of Defense Milestone C meeting for DSUVIA is expected in Q2 2020.

Commencement of investigator-initiated studies of DSUVIA in post-operative pain management and Enhanced Recovery protocols is anticipated beginning in H1 2020.

Shares are up 4% premarket.

#JPM20

https://seekingalpha.com/news/3531281-acelrxplus-4-premarket-on-corporate-update

Inogen expects Q4 revenue drop, looks to improvement in 2020

Inogen (NASDAQ:INGN) -18.2% pre-market after saying it expects Q4 revenues of $78.4M-$79.4M based on preliminary data, down 8.2%-9.4% from the year-ago quarter, citing softer than expected demand primarily in its direct-to-consumer and international channels.

For FY 2019, INGN expects revenues of $361M-$362M, up 0.9%-1.2% from 2018.

INGN updates FY 2020 revenue guidance to $385M-$400M, which would represent 6.4%-10.5% Y/Y growth vs. the 2019 preliminary revenue mid-point of $361.9M, with direct-to-consumer sales expected to be its fastest growing channel.

The company also expects manufacturing challenges to continue in Q1, where certain component part shortages may delay shipments.

https://seekingalpha.com/news/3531278-inogen-expects-q4-revenue-drop-looks-to-improvement-in-2020

Bluebird bio launches Zynteglo for thalassemia in Germany

Bluebird bio (NASDAQ:BLUE) announces the launch in Germany of ZYNTEGLO (autolog

https://seekingalpha.com/news/3531175-bluebird-bio-launches-zynteglo-for-tdt-in-germanyous CD34+ cells encoding β^A-T87Q-globin gene), a one-time gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β⁰/β⁰ genotype, for whom hematopoietic stem cell transplantation is appropriate but a human leukocyte antigen-matched related HSC donor is not available. This is the first time ZYNTEGLO is commercially available.

Bluebird has also collaborated with University Hospital of Heidelberg as the first qualified treatment center in Germany.

In addition, bluebird has entered into value-based payment agreements with multiple statutory health insurances in Germany to help ensure patients and their healthcare providers have access to ZYNTEGLO.

Bluebird has initiated the rolling BLA submission for U.S. FDA approval. The company is planning to complete the BLA submission in H1 2020.

https://seekingalpha.com/news/3531175-bluebird-bio-launches-zynteglo-for-tdt-in-germany

Ultragenyx files U.S. application for expanded use of Crysvita

Ultragenyx (NASDAQ:RARE) and collaboration partner Kyowa Kirin (OTCPK:KYKOF) announce the filing of a supplemental marketing application in the U.S. seeking approval to use Crysvita (burosumab) to treat FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia) that cannot be curatively resected or localized.

The companies are co-commercizing burosumab in the U.S. under a 2013 agreement.

The agency approved the FGF23-blocking antibody in April 2018 for X-linked hypophosphatemia.

https://seekingalpha.com/news/3531265-ultragenyx-files-u-s-application-for-expanded-use-of-crysvita

Blueprint Medicines lays out growth plans

Blueprint Medicines Corporation (NASDAQ:BPMC) formally sets out its corporate goals for 2020.

“As we complete our evolution into a fully-integrated biopharmaceutical company this year, we will also aim to bring a second product to market, expand across multiple indications and extend our global commercial footprint with our first anticipated regulatory approval in Europe.”

The company says its future growth will also be fueled by an expanded strategic focus on systemic mastocytosis and related mast cell disorders, which represent a large population of underserved patients with significant medical needs.

In particular, Blueprint expects to report top-line data from the Phase 3 VOYAGER trial of avapritinib in third-line GIST in Q2 and gain regulatory approval and launch avapritinib in fourth-line GIST in the U.S. During Q3, the company aims to gain regulatory approval and launch avapritinib in PDGFRA D842V GIST in Europe.

#JPM20

Source: Press Release

https://seekingalpha.com/news/3531264-blueprint-medicines-lays-out-growth-plans

OrthoPediatrics updates guidance

OrthoPediatrics Corp. (NASDAQ:KIDS) reports Q4 revenue is anticipated to be $19M on a preliminary basis. The tally is up 30% from a year ago.

For the full year, OrthoPediatrics’ revenue is expected to be up 26% to $72.6M.

The company plans to release full results in early March.

Source: Press Release

https://seekingalpha.com/news/3531261-orthopediatrics-updates-guidance

Verona Pharma up 59% premarket on positive ensifentrine data

Thinly traded nano cap Verona Pharma (NASDAQ:VRNA) is up 59% premarket on modest volume in reaction to positive results from a Phase 2b clinical trial evaluating lead drug ensifentrine or placebo as an add-on treatment to tiotropium (Boehringer Ingelheim’s Spiriva Respimat) for the treatment of moderate-to-severe chronic obstructive pulmonary disease (COPD).

The study met the primary endpoint of improved lung function as measured by the improvement in peak forced expiratory volume in one second (FEV1) at week 4 in patients receiving ensifentrine added on to tiotropium compared to placebo added on to tiotropium. The primary objective was achieved at all doses tested.

The results support dose selection for Phase 3 studies.

https://seekingalpha.com/news/3531187-verona-pharma-up-59-premarket-on-positive-ensifentrine-data