In Clementia Pharmaceuticals Inc (NASDAQ:CMTA), OrbiMed holds the largest stake, which contains 10.43 million shares. Other funds long the stock include Baker Bros. (250,000 shares), Partner Fund (311,755 shares), and RA Capital (584,400 shares). Clementia Pharmaceuticals Inc (NASDAQ:CMTA) specializes in developing treatments for people with ultra-rare bone disorders. The company is currently conducting enrollment for Phase 3 Trial in evaluating palovarotene for the treatment of patients with Fibrodysplasia Ossificans Progressiva, disease that causes fibrous tissue to be ossified when damaged. In April, Clementia Pharmaceuticals Inc (NASDAQ:CMTA) also started a Phase 2 trial assessing palovarotene in pediatric patients with multiple osteochondromas (benign bone tumors). The completion of the trial is estimated in January 2021. Palovarotene, Clementia Pharmaceuticals Inc (NASDAQ:CMTA)’s lead candidate, is a retinoic acid receptor γ agonist that blocks the abnormal growth of bone in soft tissue and it has received orphan drug designation from the FDA and EMA.
Ascendis Pharma A/S (NASDAQ:ASND) saw OrbiMed, Baker Bros., Deerfield, and RA Capital holding stock at the end of last year. Among these funds, RA Capital disclosed the largest position that contained 5.58 million shares, followed by OrbiMed with a 4.37 million-share position, Baker Bros. with 2.62 million shares, and Deerfield, which owns 789,000 shares. Ascendis Pharma A/S (NASDAQ:ASND) is working on three rare disease endocrinology programs that use its TransCon technology, which aims to extend the duration of a drug’s action in the body and improve its benefit. The company expects to see full-line data from its ongoing phase 1 trial of TransCon PTH (for hypoparathyroidism), top-line from planned phase 1 trial of TransCon CNP (for the treatment of achondroplasia, the most common form of dwarfism). In addition, Ascendis Pharma A/S (NASDAQ:ASND) anticipated to present top-line data from its phase 3 heiGHt Trial for TransCon Growth Hormone.
Partner Fund is one of the top shareholders of Aerie Pharmaceuticals Inc (NASDAQ:AERI), holding a stake of 2.61 million shares. It is followed by OrbiMed with a position containing 1.33 million shares, Deerfield Management with 484,100 shares and Baker Bros with 299,360 shares. However, it’s important to point out that Deerfield also reported ownership of 5.04 million shares underlying convertible notes. Aerie Pharmaceuticals Inc (NASDAQ:AERI) focuses on the development and marketing of therapies for the treatment of eye diseases. In December 2017, the FDA approved Aerie Pharmaceuticals Inc (NASDAQ:AERI)’s first product Rhopressa for the reduction of elevated intraocular pressure and at the end of April, the company launched the product for sale. For the fourth quarter, Aerie Pharmaceuticals (NASDAQ:AERI) reported a net loss of $1.38 per share, versus expectations of a loss of $1.27 and it had cash and equivalents of almost $250 million.
Then there is argenx NV – ADR (NASDAQ:ARGX), a clinical-stage biotech company focused on developing antibody-based therapies. In argenx NV – ADR (NASDAQ:ARGX), Baker Bros. Advisors disclosed ownership of 961,750 shares, followed by OrbiMed, Deerfield, and RA Capital with stakes containing 500,000 shares, 365,960 shares, and 75,820 shares, respectively. In March, argenx NV – ADR (NASDAQ:ARGX) received orphan drug status in the EU for ARGX-113 for the treatment of myasthenia gravis. The orphan drug status offers argenx NV – ADR (NASDAQ:ARGX) 10-year exclusivity in the EU if the drug is approved. The ARGX-113 also received FDA orphan drug designation, which gives a seven-year period of market exclusivity.
In bluebird bio Inc (NASDAQ:BLUE), OrbiMed reported a 384,050-share stake, while Baker Bros., Partner Fund, and Deerfield disclosed holding 200,000 shares, 139,790 shares, and 80,000 shares, respectively. Bluebird bio Inc (NASDAQ:BLUE) develops gene therapies for severe genetic and rare diseases. In March, bluebird bio Inc (NASDAQ:BLUE) teamed up with Celgene (NASDAQ:CELG) to co-develop CAR T bb2121, a drug that is currently in Phase 3 development for the treatment of multiple myeloma. The companies will equally share costs and profits in the US and bluebird bio Inc (NASDAQ:BLUE) stands to receive milestone payments and royalties from sales outside the US. In addition, bluebird bio Inc (NASDAQ:BLUE) has a number of other products under various phases of development.