Affordable and as an outpatient treatment. Could lead to heavy worldwide demand where an experienced partner like Roche may be willing to pay a HUGE upfront fee and royalty stream for distribution outside of US?
A huge improvement in patient outcome and affordable? Worldwide sales could become a serious focus and that would lead to partnering.
SCRIP Intelligence article
Celladon president and CEO Krisztina Zsebo:
KZ: Cost of goods is not going to be an issue. It's an important consideration and we have three strategic pharma investors – Pfizer, Novartis and Johnson & Johnson – and I can assure you that we endured the full extent of their diligence efforts on all aspects of our technology for many, many months at a time. I feel that we're on very solid footing with regards to the production and cost of goods.
investor relations Fredrik Wiklund:
We are working with Lonza, who are one of the absolute leaders in the industry of producing mass-scale biologics. We estimate that when we will be in full commercial mode and producing this, the cost of goods or the drug cost itself is really quite modest, probably $1,000 to $2,000 per dose per patient. When you put that in the context of what typical biological drugs cost on an annual basis, it's moderate.
Agree with your edit of my post in defining end stage. I do think the FDA will need to think hard about making Mydicar an option for the early stage Class 4 and Class 3 patients. Just need some strong responders in Cupid 2 to make the case
The enlarged heart population could be aiming for much earlier intervention and much larger patient population.
“In general the term ‘enlarged heart’ refers to heart failure,” said Clyde Yancy, M.D., past president of the American Heart Association and chief of the Division of Cardiology and the Magerstadt Professor of Medicine at Northwestern University Feinberg School of Medicine in Chicago. “This is a common condition that’s more likely to occur in older patients. It’s most strongly related to a history of high blood pressure or a previous heart attack.”
About one in five adults over age 40 is affected, with African-Americans facing a greater risk.
“There are other reasons for an enlarged heart or heart muscle disorders (like cardiomyopathies, which are diseases of the heart muscle) and not everyone with heart failure has an enlarged heart,” Dr. Yancy said. “But when we speak of an enlarged heart we are typically referring to heart failure.”
* Does Celladon sell their first drug for a few billion if the news on Cupid is good? Hope not.
Early availability approval for end stage patients?
In 2012, we obtained a Special Protocol Assessment, or SPA, whereby the FDA agreed to use time-to-multiple heart failure-related hospitalizations as the primary endpoint for a MYDICAR Phase 3 pivotal trial. Our ongoing CUPID 2 trial uses a similar clinical protocol with identical endpoints as agreed to in the SPA.
...The one- and six-month readmission rates after heart failure-related hospitalization are close to 25% and 50%, respectively, and there is growing pressure on hospitals to reduce readmissions for heart failure.
(2014 Mydicar gets Breakthrough Therapy status) - No idea why this company doesn't have a market cap much higher as Cupid 2 leaks should be obvious to the big boys following this trial.
GLTA, missed todays dip
Assistance Publique – Hopitaux de Paris primary endpoint ((six months))
My wild guess is that some previous trial patients were super responders in the first Cupid trial.
Assistance Publique – Hopitaux de Paris
dawg, another partner was impressed, in something they saw, and is conducting a trial to see if Mydicar can actually reverse some of the damage done to the heart by HF:
Celladon also announced that investigators have initiated the AGENT-HF Trial (AAV1-CMV-SERCA2a Gene Therapy Trial in Heart Failure). This trial is an investigator-initiated clinical trial and is partially funded by the French government and sponsored by Assistance Publique – Hopitaux de Paris. This trial is not required by any regulatory authorities for systolic heart failure indications, but the patients enrolled in this trial will be included in the overall safety database submitted in an MAA submission.
The primary objective of the AGENT-HF Trial is to determine whether treatment with MYDICAR leads to reverse remodeling of the heart. In patients with heart failure, the size, shape, structure and physiology of their heart changes over time, and these changes that lead to a progressive decline in left ventricular (LV) function are referred to as remodeling. In reverse remodeling, there would be changes back to the more normal, healthier state of the heart along with an improvement in the functioning of the heart. This trial will enroll approximately 44 heart failure patients in France with half receiving MYDICAR and the other half placebo. The primary endpoint at six months will be change, compared to baseline, in LV end systolic volume as measured by cardiac computed tomography.
(I suspect they will see improved function regardless of actual physiological turning back the clock. Maybe the organization has looked at data in a completed Celladon study and was impressed at some of the patients data they saw?)
Found an interesting take on the first Cupid trial
" Molecular Therapy" 2011
CUPID is a phase II randomized, double-blind, placebo-controlled trial that evaluated the effectiveness of a gene transfer vector based on adeno-associated virus 1 (AAV1) for delivery of SERCA2a complementary DNA in 39 patients with advanced heart failure.
There are several reassuring points in Jessup and colleagues’ data set. One is that this is the first clinical application of AAV vectors for cardiac gene transfer. By virtue of its exquisite and still largely unexplained capacity to transduce postmitotic cells, AAV is currently the vector of choice for cardiac applications. Second, it is interesting to learn that a single intracoronary infusion of the vector has been sufficient to provide a potential therapeutic benefit in a condition where the transgene must be expressed inside the cells and thus where a high efficiency of transduction is required. In this respect, the authors have taken advantage of AAV serotype 1
(Lots more detail in the article. It looks like a big endorsement that the first Cupid trial has shown cardiac gene therapy is more than scientific fantasy)
The Big Boyz have their ways of following trials and finding out about trends. Could be happening here?
The current 250 person trial has been going on for a few years and the control arm will likely be revealing itself early. The Journal of American College of Cardiology State-Of-The-Art Paper | January 2013:
" Patients with hospitalization for HF (HHF)....Discharge from a heart failure hospitalization is followed by a readmission within 30 days in ≈24% of cases."
Mydicar patients may be showing significant improvement in the 30 day post discharge reporting? If so, it's likely the "breakthrough" status is drawing interest in what the Data Monitoring Committee (DMC) is seeing.
* Just some wild guessing on why so few shares are up for sale at what has been very nice percentage gains. GLTA, this one has the potential for huge returns if all goes well.
see page 14 of Jeffries presentation.
Cost saving and effectiveness will show up much quicker than 12 months. FDA opening trial so that all patients may receive Mydicar benefit? Should be a very obvious move 6mths into therapy.
Early halt to trial?
Readmissions has become a huge focus for hospitals. FDA fines are now being used.
May 19, 2014
Patrick Malone and Associates
n a news release, the Department of Health and Human Services said, “National reductions in adverse drug events, falls, infections, and other forms of hospital-induced harm are estimated to have prevented nearly 15,000 deaths in hospitals, avoided 560,000 patient injuries and approximately $4 billion …”
More than 2,000 hospitals, according to KHN, have been fined each of the last two years because the number of patients they had to readmit within 30 days was higher than the health law standard.
The primary efficacy endpoint is time-to-recurrent HF-related hospitalizations in the presence of terminal events (all-cause death, heart transplant, LVAD implantation).
Mydicar passed with 83% improvement in first trial and is set up for approval if only half that rate in this trial. Take a look at the slide showing readmissions in first 12 months for placebo versus dose being used in this trial. Early halt to trial looks possible and even likely?
Some anxious buyers showing up after Jeffries conference. Pushing price up hard in down market to get shares, impressive.
"Breakthrough Therapy" status and accelerated approval seem possible.
During the early part of the in-patient stay, prolonged procedures may be best performed under general anaesthesia. This is especially true if access to the patient will be difficult or in the case of children where fear of repeated potentially painful procedures will be a significant barrier to trusting the staff.
NexoBrid (EU), Debrase (US)
The study compares the safety & enzymatic debriding efficacy of bromelain with that of the current standard-of-care (SOC) in hospitalised patients with deep partial thickness & full thickness thermal burns. The primary endpoints are percentage of treated wound excised in first surgery & percentage of treated wound autografted of deep partial wounds. Because of positive interim results, the trial met the criteria for early stoppage & was deemed to have met its primary objectives. Total enrolment was to be 270 pts; however, due to early completion, only 182 pts were recruited at sites in Australia, Brazil, France, Germany, Israel, Italy, Poland, Romania, Slovakia & the UK .
IMUC) was upgraded by Maxim Group to a “buy” rating in a research note issued on Monday. IMUC) was upgraded by Maxim Group to a “buy” rating in a research note issued on Monday. Maxim Group cut their price target from $12.00 to $4.00
* Looks like some analysts are impressed by ImmunoCellular's ability to narrow their target population by identifyling and targeting subgroups likely to respond. The ability to identify a responsive subgroup the FDA should be very happy not wasting chemo treatment of nonresponders. I'm still holding out promise of enlarging the subgroup target with the a new combination trials with one of the drugs coming to market that fights the bodys natural buildup to immune resistance.
Is IMUC entering that summer funk where so many stocks drift lower?
Before ASCO results
Roth Capital reiterated its Neutral rating and $2 price target on ImmunoCellular Therapeutics (AMEX: IMUC) following the Company's update on 2014 strategies and milestones.
Analysts Joseph Pantginis commented, We are now on the sidelines until more answers regarding ICT-107 materialize and the company's ability to adequately maintain manufacturing and capital needs becomes clear. We look forward to more information
Roth changed rating to buy and target to $3.00
"Updated data from the Phase II were presented, (failed primary endpoint of overall survival in December 2013). However, the company presented data based on prospectively defined subgroups (HLA status and MGMT status), which we believe will help propel ICT-101 back into the spotlight for glioblastoma. So based on these data, we believe the company now has clear efficacy data based on 1) prospectively defined subgroups, 2) immunological data and 3) a likely path forward for the drug," he added.
*Buy the dips and building a sizeable position (for me). Keeping high hopes for those late strong performers to possibly reveal one more group.
New Headquarters lease for 10,908 square feet of office space, old space 2,270 square feet of office space .
Just reading tea leaves folks. Someone is seeing encouraging results in the ongoing trial.
Mr. Cleveland brings decades of industry experience to his new leadership position. Most recently, Mr. Cleveland was Executive Vice President of Corporate Strategy and Chief Financial Officer of Aragon Pharmaceuticals, Inc. Aragon was sold to Johnson & Johnson in August 2013 for $1 billion.
Next trial will no doubt be all HLA-A2. The HLA-A1 looks like non responders and kept ImmunonCellular from grabbing some impressive headlines at ASCO. Not many analysts following this one so maybe the small investor is going to have a chance at analyzing this data for impressive subgroups before the big boys.
In US and Europe HLA-A2 is twice as common as HLA-A1