AGIO- Pivot to PKD

Agios Pharmaceuticals came to market in 2013 as a promising cancer metabolism company. Three years later, it has pivoted to rare disease. AG-120, an IDH1 mutant inhibitor being developed for.

ASH Update: TGTX

ASH 2016 has turned out to be an important convention for many small biotechs, and will likely be remembered as a break-out event for CAR-T therapies. Results from the Phase 1 TGR-1202/Ibrutinib.

ASH Update 2: BLCM

Bellicum Pharmaceuticals’ leading clinical candidate is BPX-501, individualized T-cells transduced with a controllable suicide gene. They are being studied for use as an add-back following T-cell depletion during Haploidentical Stem.

ASH Update: TRIL

Trillium Therapeutics presented interim data from its ongoing Phase 1 trial of the SIRPaFc TTI-621 at ASH this past weekend. These highly anticipated results gave a deeper look into the.

BLCM: Staying on the Sidelines

Bellicum is a small biotech developing drugs in the hot field of adoptive cell therapeutics. Their lead candidate is a personalized T-cell product for use after stem cell transplants to prevent early.

Achillion’s Pivot To Orphan Diseases

Through many years, Achillion focused on HCV, among the biggest prizes in drug discovery. The problem is they entered the field late while working on a very popular target. A nearly.

Eteplirsen: Reasonable Evidence of Efficacy

  The Adcom is over and Eteplirsen was voted down. A final decision still remains for approval. Much was discussed in the Adcom, the key arguments were over the following:.

GWPH: Is Epidiolex Nothing But Placebo?

GW Pharmaceuticals made the mistake of allowing expectations to exceed its abilities, forcing the company to announce a delay in the read-out of its pivotal Dravet study. Data originally anticipated for release.

Sarepta: Is Eteplirsen Dead?

This was originally going to be titled “Ahead of the AdCom” but that no longers seems fitting. The highly anticipated Advisory Committee meeting originally scheduled for Friday January 22 was.

BLCM: Limited Opportunities In a Changing Environment

The driving interest in Bellicum revolves around its safety switch for use in hematologic stem cell transplants. It’s product, BPX-501, is a T-cell therapy containing a caspase-9 gene that can.

LOXO: Staying On TRK

Loxo Oncology presented highly anticipated results from its ongoing Phase 1 study of TRK inhibitor LOXO-101 yesterday November 8, in a latebreaker at AACR-NCI-EORTC; they did not disappoint. Disclosure of.

SRPT: Better The Devil You Know

The Bull/Bear tussle in Sarepta has been ongoing for years now. Long term investors have ridden a rollercoaster as excitement has inevitably been followed by disappointment. Its Exon 51 skipping.

Global Blood Therapeutics: Targeting Sickle Cell Disease

Global Blood Therapeutics (GBT) is a recent graduate of Third Rock Ventures, a highly regarded firm with a list of innovative portfolio companies including Bluebird Bio, Foundation Medicine, Agios, and.

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.

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.

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.

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.

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.

ESPR Quick Note

Esperion shares have fallen over 8% today as The Medicines Co and Alnylam released early data on their RNAi candidate, ALN-PCSsc, targeting PCSK9. The drug was shown to efficiently and.

Quick Note on GWPH

Embargoed Data was released yesterday in an announcement from AAN regarding GW Pharmaceuticals’ Epidiolex study: https://www.aan.com/PressRoom/Home/PressRelease/1364 This paragraph summarizes the Key Takeaway: “For the 137 people who completed the 12-week.

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.

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.

Pfenex: Biosimilar Enthusiasm Is High

San Diego’s Pfenex Inc. is riding the market’s enthusiasm for biosimilars, molecules that although not considered identical, can be automatically substituted for biologic drugs. Previously considered immune to generic competition,.

Quick Note on GWPH

GWPH has been on one huge rollercoaster. After climbing back from $61 in mid-October to $80 in late November, it has given up 15% in just the last 3 trading.

What to Look For at ASH 2014

  The year is nearly over, though it feels like scientific conferences never end. December 6th marks the last big Cancer meeting of the year- then we turn the page to.

[Free Content] TGTX in Pictures

  TG Therapeutics has two programs, the CD20 targeting antibody TG-1101, and PI3K delta inhibitor TGR-1202. The focus of this post is on TGR-1202. Earlier coverage can be found here.

GWPH: A Compelling Story

GW Pharma’s Tuesday morning R&D meeting looked pretty good. Efficacy data were released for 58 patients with full 12-weeks of epidiolex treatment- 40 of whom have been treated for 16-weeks..

Sarepta at World Muscle Society: Follow-up

The only new data provided by Sarepta were various measures of lung function, including, including %predicted MIP, MEP, and FVC; all of which demonstrated stabilization through 144 weeks for the.

SRPT and SAGE: Quick Notes

Nice 36-month Natural history study (or “Pane Study”) in Duchenne published essentially confirming what’s pretty much already known- that boys age 7 and over decline more rapidly than those under.

GW Pharmaceuticals: Quick Update

Investors are waiting impatiently for GW Pharma’s December R&D day. The most sought-after information is updates on the Epidiolex Expanded Access Program in Dravet Syndrome. A previous analysis involved 27.

[Free Report] JNJ Aquires Alios: What This Means For Achillion

Johnson & Johnson announced today it will acquire privately held Alios BioPharma for $1.75 Billion in cash, gaining rights to the mid-stage treatment for infants with respiratory syncytial virus (RSV),.

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