Raptor Pharma: Of Cystinosis and Huntingtons

Raptor Pharmaceuticals is a small Novato, CA. company that markets Procysbi for the treatment of nephropathic cystinosis. It has active clinical trials in Huntington’s Disease and Leigh Syndrome. A recent acquisition has provided entry into the Cystic Fibrosis field. Procysbi- also known as RP103- took a different route to market from that of most newly […]

STARTRK-1: Lost In Space [RXDX]

This is a brief analysis of recent entrectinib data presented by Ignyta. Entrectinib is a multi-kinase inhibitor with potent activity against TrkA/B/C, ROS1, and ALK. It was licensed from Nerviano Medical Sciences in November 2013, whereupon Ignyta took over development of the compound. Entrectinib is currently being studied in two Phase 1 studies- one in […]

INCY: Predicting Success in Pancreatic Cancer

Incyte Pharmaceuticals’ lead drug Jakafi (Ruxolitinib) is approved for the treatment of the bone marrow disorders Myelofibrosis and Polycythemia Vera. It is expected to reach blockbuster sales despite these relatively niche indications. The company has ambitions to expand Jakafi into solid tumors with Pancreatic cancer leading the way. Top line data from a Phase 2 […]

Loxo Oncology: One to Watch

Loxo Oncology (LOXO) is a biotech company built around the idea that cancer is best treated with highly targeted drugs paired to companion diagnostics. CEO Josh Bilenker, a partner at Aisling Capital, founded the company in 2013. Research and Development is outsourced, allowing about a dozen employees to manage company operations. Loxo acquired essentially all […]

Trevena Scores In Pain Study

Trevena Inc. (TRVN) presented positive Phase 2b results Monday August 31 from its TRV130 pain study. The primary endpoint of superior pain reduction compared to placebo was met, as well as superiority to morphine in the secondary endpoint of lower rates of nausea, vomiting, and hypoventilation. The results were quite good, and together with the previous […]

BioBlast Pharma: an under the radar ultra-rare disease company

  Bio Blast Pharma Ltd. (ORPN) – Nasdaq BioBlast Pharma (ORPN) is a clinical-stage biotech company, focused on treatments for rare and ultra-rare diseases, especially genetic and metabolic diseases. The company has three diversified platforms currently address five different diseases. The platforms include Cabaletta, which is a platform for diseases characterized by mutated proteins; BBrm platform is a […]

TRIL – Preparing For The Next Move Upward

  Trillium Therapeutics Inc. (TRIL) – Nasdaq Trillium (TRIL) is developing the next-generation of cancer immunotherapies. TRIL’s lead program, SIRPαFc, targets CD47 to prevent cancer cells from delivering a “do not eat” signal to macrophages. High expression of CD47 by tumor cells has been correlated with more aggressive disease and poor survival. SIRPαFc is initially being developed […]

The Reality for Mannkind

Mannkind, the maker of the inhaled insulin product Afrezza, is the 11th best performing biotech stock according to Finviz in the last month, up 62%. That performance is in line with several big ASCO winners such as Oncothyreon, Ignyta and Immunogen. Also on the list is DMD company Sarepta, which has new hope for an […]

Chimera Research Group Catalyst Summary for April 2015

At the end of each month Chimera Research Group provides you with a monthly summary of ten notable catalysts/events that have been completed during the past month. APRIL 2015: Gilead Sciences (NASDAQ: GILD) exceeded analyst expectations reporting $7.6 billion in first quarter revenue , including $4.55 billion from their HCV portfolio of Harvoni and Sovaldi. Revenue […]

The Week Ahead in Earnings

A second busy week of earnings is due out in the space this week. We will see 3 of the 4 remaining large caps report (Gilead, Celgene, Vertex) and a smattering of big pharma reports as well. Given the sell off today, earnings reports could stem the fall, or exacerbate it further with poor results. […]

CYNAF is OFF the Mark

Cynapsus is developing a sublingual film formulation of apomorphine for the treatment of acute “Off” episodes in patients with Parkinson’s Disease resulting from long term use of Levodopa. While Levodopa is considered the single best treatment for Parkinson’s, long term use of the medicine leads to motor fluctuations as its efficacy declines. Patients then begin […]

Chimera Research Group Catalyst Summary for March 2015

At the end of each month Chimera Research Group provides you with a monthly summary of TEN notable catalysts/events that have occurred during the past month. MARCH 2015: 1) Bristol-Myers Squibb Company (NYSE:BMY) announced that the FDA approved the expanded use of Opdivo to treat patients with advanced squamous non-small cell lung cancer (NSCLC). Approval came just over two […]

Adamas Pharmaceuticals: two interesting programs for Alzheimer and Parkinsons Diseases

Adamas Pharmaceuticals, Inc. (ADMS) –Nasdaq   Adamas (ADMS) is a company focused on CNS diseases, specifically Parkinson’s (PD) and Alzheimer’s Diseases (AD). It has a low market cap (~$300M), plenty of cash ($158M), low float with most of the shares (64.2%) held by insiders and institutions (link), two FDA approved products partnered with Forest/Actavis (for one […]

March 13 Biotech Update

I know that the sector is doing well and near highs but I cannot shake the feeling that the stocks feel heavy. I do not have a better way to describe it nor anything more solid than this impression. Perhaps this is simply the wall of worry that we are going to climb. If I […]

Quick Take on Marinus Pharmaceuticals

Marinus Pharmaceuticals is developing the allopregnanolone analog Ganaxolone as a treatment for epilepsy and other seizure disorders. Its most advanced program is a multinational P3 trial of patients with refractory focal onset seizures. The drug is also being tested in patients with Fragile X syndrome and in girls with epilepsy due to PCDH19 mutation. The […]