July 3, 2015;
Phase 2 B trial published in 140 patients using nebulized non viral vector gene therapy for CF in England. FEV-1 after 12 months: 3.5 % improvement vs placebo group who lost 4%.
Servier trial has 15 active cases. All responded. Vitreous haze was significantly improved by pic shown for xoma trial. Lot of subjective input in the variability. My major concern is test objective of 70% reduction in exacerbation. I think bar is set very high.
Ann Rheum Dis 2012, 71: 4. 563-566.
All 7 patients responded to a single infusion of 0.3 mg/ kg. Complete resolution of intra-ocular inflammation between 4-21 days. No treatment related adverse events. Median duration of response was 49 days. Open label and very small cohort but results were strong enough that Servier chose to proceed with a phase III trial.
In contrast to common believe that this result is about XOMA, I think this trial is about Servier pharmaceutical which is a huge French company, markets products all over the World. It recently got US FDA approval of its drug Ivabradine for Chronic heart failure now marketed by AMGN under the name Corlanor.
Not exactly. Stock went from $3 to $84 first. DNDN had manufacturing problem and they were not capable of manufacturing for enough patients. Competition came from JNJ and they got toasted but they were the first approved drug that started cancer immune therapy. Efficacy of drug was prolonging life by 3 months, not much.
Expert opinions are bothering me. No doing wishy washy. J L himself was worried about 3% and he said drug is working in the distal airways and thus FEV I is not a good assessment of drug benefit Lung Clarence Index should be a better test ( which is not approve). I guess once FDA approves and I see a decent label, I will be okay. I thought approval could happen before PUDUFA date.
only 3 % improvement in FEV1 bothers me more. Also GLPG/ABBV approach to deal phenotypically seems more logical and using assays could improve their trial out come. Exacerbation reduction of 30-40% is not much and more of a relative number crunch. I know this board does't like negative post but I am honest and factual.
very unusual for a biotech.
Drug should do well in the clinical trial for Behcet disease and also in Pyoderma gangrenosum. Both Orphan indication and drug is shooting for superiority to steroids and biologics, so no near term competition if successful. This is gold. I will hold it through the Eyeguard B and Eyeguard US trials and also long term even if trial is reported negative. XOMA is unlucky but a good biotech which brought MAB therapies in the market. This one is like HGSI, if successful should see 20-30's.
VRTX has preclinical program for Calcitonin gene receptor peptide antagonist since 2009 and I hope to hear that this drug will enter into clinic this year. AMGN, LLY an BMY all have drug candidates in mid to late stage. MRK had the lead but unfortunately their drug has liver issues in phase 3 and they dropped out of this race. Drug is worth developing because it may find additional indications in Septic shock and Inflammation.
One bad side effects from this class of drug will be loss of taste as epithelial sodium channels are part of taste buds and apocrine glands. I am more worried about systemic absorption and toxicity. Obviously GILD is way smarter than VRTX when it comes to clinical development and there is something that we don't know yet. VRTX has bad history of partnering drugs and almost all their projects have failed. VRTX talks about second generation correctors but so far failed to bring one in the clinic in the last 48 months. VX 983 was touted to be superior to VX 661 but failed in the phase 1. I hope GILD writes a check to VRTX and we become part of GILD. I love dividends.
GILD had funded ENaC blocker development with Parion since 2007. In 2014 they terminated GS 5737( P 1037) COPD phase 1 trial and returned the drug development rights to Parion. Why GILD will do such thing when they are planning to enter pulmonary medicine franchise like IPF?
Various talks on CF correctors and ENaC inhibitor but no direct talks or updates on VX 661 or Orkambi. In fact, I didn't find no press release or presence from VRTX. GILD is there.
Why trial is planned with Orkambi and not with VX 661?
Also Parion is doing the current trial of the drug with hypotonic saline ( .019%) and hyper-hypertonic saline ( 4.7%) and what implications will be for VRTX from that trial?
You need sodium channels to survive and obviously systemic use is out of question ( instant death) topical inhalation use may also will have some (2% or so) systemic absorption, so safety issues may will arise.
This is an old concept which has been out there since 2008. My understanding is that partner drugs are to be taken as HFA/ inhalation form. I am very concern about Reata pharmaceutical, Irving, TX work in CF by phil Thomas group who have license it from Southwestern UT, Dallas. GSK also is working on correctors and one misstep by VRTX ( buying Virochem instead of Pharmasset in hep C) and we will repeat Incivek story. I hope Van Goor is still playing for VRTX team. NBD1, NBD2 and intermediate steps in misfolding are still not very clearly understood.
Checkout Gevokizumab for Behcet's Uvietis. XOMA
Orphan indication and 6 weeks loto ticket.
Sale could be 1-3 billions and market cap is only 400 million. $ 25 easy if trial is positive.
Train is leaving, oooo0000OOOOOOOOOOOO!!!!!!!!!!!!!!!!!!