U.S. Markets closed

Rare Disease Therapies Draw Big Biopharma's Interest

HENDERSON, NV / ACCESSWIRE / March 8, 2019 / The past few weeks have been busy with big bio taking over smaller biotechs developing novel therapies. Roche (RHHBY) said it would spend $4.8 billion in a buyout of Spark Therapeutics (ONCE). In January, Johnson & Johnson (JNJ) unveiled a collaboration and license deal with MeiraGTx (MGTX) in eye diseases.

This week, Biogen (BIIB) pledged $800 million to acquire Nightstar Therapeutics (NITE). Smaller biotech Nightstar is testing gene therapy in rare eye diseases. Investors in NITE saw their shares climb 66% since the announcement. With that in mind, we decided to find some other companies developing therapies for rare diseases.

Today we are highlighting: Delcath Systems, Inc. (DCTH), Nightstar Therapeutics (NITE), Spark Therapeutics, Inc. (ONCE), MeiraGTx Holdings plc (MGTX), and Voyager Therapeutics, Inc. (VYGR).

A company you should consider researching, Delcath Systems, Inc. (DCTH) (Market Cap: $3.448M Share Price: $0.2551), is an interventional oncology company focused on treating liver cancers with upside potential. DCTH sponsors a global Registration clinical trial for Patients with Hepatic Dominant Ocular Melanoma called The FOCUS Trial. Approximately 4,500 patients in the U.S. and Europe are diagnosed with ocular melanoma each year. In 2018, DCTH amended The FOCUS trial to single-arm non-randomized study, raised $7 million via a rights offering, and had positive data published in European Radiology among several other milestones.

DCTH just announced they've received medical device approval for the CHEMOSAT® Hepatic Delivery System (CHEMOSAT) from the national health authority in Brazil. This is a significant potential market for DCTH. According to the World Health Organization, liver and bile duct cancers account for over 10,000 deaths in Brazil annually.

This comes after DCTH entered a licensing agreement for CHEMOSAT® commercialization in Europe with medac Gesellschaft für klinische Spezialpräparate mbH (medac). Medac is a privately held, multi-national pharmaceutical company based in Hamburg area, Germany. Founded in 1970, Medac specializes in the treatment and diagnosis of oncological, urological and autoimmune diseases. The company has offices globally, worldwide partner agreements in over 90 countries, and approximately 1,200 employees.

DCTH also announced the independent Data Safety Monitoring Board (DSMB) of the Registration trial for Patients with Hepatic Dominant Ocular Melanoma (The FOCUS Trial) completed another pre-specified review of safety data for treated patients in the trial. This review was conducted on data collected from both the prior randomized protocol and the amended single-arm protocol for the FOCUS Trial. The DSMB again recommended continuation of the study without modification.


A couple companies who have already received agreements from big bio as mentioned above include:

Nightstar Therapeutics (NITE) (Market Cap: $850.154M; Share Price: $25.39) soared early Monday after Biogen pledged to spend $800 million to buy the gene therapy firm. The smaller biotech is working on gene therapies in rare eye diseases.

Spark Therapeutics, Inc. (ONCE) (Market Cap: $4.303B; Share Price: $113.71) received an agreement from Roche for approximately $4.3 billion at a significant premium of 122%.


Another company we highlighted above, MeiraGTx Holdings plc (MGTX), (Market Cap: $600.957M; Share Price: $18.11), will receive $100 million cash upfront payment upon closing from Johnson & Johnson's Janseen Pharmaceuticals, Inc. to fund all clinical development and commercialization costs of MeiraGTx inherited retinal disease gene therapy programs. Janssen Pharmaceuticals, Inc. to receive worldwide exclusive rights to commercialize product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, X-linked retinitis pigmentosa (XLRP) and options to additional IRD programs.


Finally, Voyager Therapeutics (VYGR) (Market Cap: $564.926M; Share Price: $17.32) announced that it has entered into an exclusive, global strategic collaboration and option agreement to develop and commercialize vectorized antibodies directed at pathological species of alpha-synuclein for the potential treatment of Parkinson's disease and other diseases characterized by the abnormal accumulation of misfolded alpha-synuclein protein with AbbVie.

Legal Disclaimer:

This article was written by Regal Consulting, LLC ("Regal Consulting"). Regal Consulting has agreed to a twelve-month term consulting agreement with DCTH dated 8/31/18. The agreement calls for $40,000 in cash, and 15,000 restricted 144 shares of DCTH per month. Issuer agreed to increase monthly cash payment in September to $120,000. All payments were made directly by Delcath Systems, Inc. to Regal Consulting, LLC. to provide investor relations services, of which this article is a part of. Regal Consulting also paid one thousand dollars cash to microcapspecultors.com to distribute this article. Regal Consulting may have a position in the securities mentioned in this article at the time of publication, and may increase or decrease its position without notice. This article is based on public information and the opinions of Regal Consulting. DCTH was given an opportunity to edit this article. This article contains forward-looking statements that are subject to certain risks and uncertainties that could cause actual results to differ materially from any results predicted herein. Regal Consulting is not registered with any financial or securities regulatory authority, and does not provide or claim to provide investment advice.

http://www.regalconsultingllc.com/full legal disclaimer/

Full Legal Disclaimer Click Here.

Contact Information:

Company Name: ACR Communication LLC.

Contact Person: Media Manager

Email: info@microcapspeculators.com

Phone: 1-702-720-6310

Country: United States

SOURCE: ACR Communication LLC

View source version on accesswire.com: