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Home  /  Forum  /  uniQure  /  Celladon

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Celladon

32 Posts
Pagina: 1 2 »» | Laatste | Omlaag ↓
  1. [verwijderd] 8 april 2015 18:22
    Beste Mensen,
    Weet niet of het hier de juiste plek is maar heeft een van jullie zich verdiept in Celladon?

    Wat ik zie is een aandeel dat bijna 50% gezakt is en dat deze maand zijn data voor een pivotal fase 2B trial vrijgeeft. Drager van de therapie is een AAV1 virus en dus een concurrent van QURE/BMS. GSK, J&J en ik meen Pfizer hebben allemaal een aandeel in Celladon.

    Ik zie het als een interessante mogelijkheid. Want deze maand komen de pivotal data en als die goed zijn haal je een goed rendement. Maar de fase 2a data moeten erg goed zijn geweest voor een breakthrough status. Inderdaad de significantie van het therapeutisch effect was meen ik p=0,0003, en dat betekent dat er of echt een stevig effect is OF dat er structureel op een verkeerde manier is gemeten, zodat de uitkomst in feite trivial is. Maar de kans dat zo'n significantie toeval is, is heel klein.

    Ik wil wel instappen geloof ik. Iemand zich al in dit aandeel verdiept?

    Hieronder wat quotes:

    Celladon's most advanced drug in its pipeline is Mydicar, an enzyme-targeting therapy for systolic heart failure patients that is currently in phase 2b studies. Last year, the FDA awarded Mydicar breakthrough therapy designation, and Celladon expects that it will be able to release top-line data from this Mydicar trial by the end of April.

    The potential for Celladon's positive upcoming data did little to support shares, however, as investors moved to the sidelines to avoid the risk of a Mydicar failure.

    Regardless, despite Celladon's investments to ramp up facilities and staff that could support an eventual commercialization of Mydicar, the company appears to be in solid financial shape.

    We are the first company to enter clinical development with a product candidate, MYDICAR, that selectively targets SERCA2a. We refer to our Phase 1 trial and Phase 2a trial of MYDICAR together as our CUPID 1 trial. In Phase 2a of our CUPID 1 trial, 39 patients with heart failure for reduced ejection fraction, or HFrEF, which is caused by the inability of the heart to pump blood efficiently due to weakening and enlargement of the ventricles, were enrolled in a randomized, double-blind, placebo-controlled trial. MYDICAR was safe and well-tolerated, reduced heart failure-related hospitalizations, improved patients’ symptoms, quality of life and serum biomarkers and improved key markers of cardiac function predictive of survival, such as end systolic volume. Based on these results, as well as our previous preclinical studies and clinical trials, we advanced MYDICAR to a 250-patient randomized, double-blind, placebo-controlled international Phase 2b trial in patients with HFrEF, which we refer to as CUPID 2. We completed enrollment of CUPID 2 in February 2014, reached the primary analysis data cutoff in February 2015 and expect to un-blind the data and announce results in late April 2015. If successful, these results, along with other studies, could form the basis for regulatory submissions for approval with the United States Food and Drug Administration, or FDA, and European Medicines Agency, or EMA.

    In April 2014, the FDA’s Center for Biologics Evaluation and Research, or CBER, granted Breakthrough Therapy designation to MYDICAR for reducing hospitalizations for heart failure in patients who test negative for adeno-associated viral vector 1, or AAV1, neutralizing antibodies, are class III or IV heart failure patients under the New York Heart Association.

  3. Prof. Dollar 8 april 2015 19:25
    quote:

    El Gaucho schreef op 8 april 2015 18:48:

    Zie net dat de CEO bijna 50.000 aandelen heeft verkocht afgelopen maandag... Hoe onprofessioneel! Daar gaat toch een enorme signaalwerking van uit!
    Goed opgemerkt!

    Een reden om niet in te stappen is dat het bedrijf geen alternatief product heeft mocht het mis gaan (alles is preclinical). Dit gegeven maakt CLDN een zeer risicovolle investering.
  4. flosz 8 april 2015 19:33
    $BMY deal with QURE should be a shot across the bow to $CLDN longs. If Bristol thought Celladon was on the verge of success ...
    Zachary Prensky
    twitter.com/zackfoot

    H.C. Wainwright & Co. on Tuesday initiated coverage on Celladon Corp (NASDAQ: CLDN) after impressive clinical trial results for its gene therapy Mydicar. Wainwright rates Celladon as a Buy with a $31 price target.
    m.benzinga.com/article/5387863

    Celladon's (CLDN) CEO Krisztina Zsebo on Q4 2014 Results - Earnings Call Transcript
    seekingalpha.com/article/3041786-cell...

    From time to time, we may offer up to 7,000,000 shares of common stock
    ir.celladon.com/secfiling.cfm?filingI...
  6. [verwijderd] 8 april 2015 22:51
    ik zou er ook niet aan komen. De daling is ook ingegeven door het gegeven dat er weinig overblijft(produkten/technologie) als de data tegenvallen.

    Als het meevalt, dan kan het hard omhoog en als het tegenvalt hard omlaag....en de statistieken wijzen uit dat het vaker tegen- dan meevalt.

    Ik ben echter niet medisch onderlegd om zelf die kansberekening te maken.

    Het zou puur een dobbel zijn...
  7. [verwijderd] 8 april 2015 22:55
    quote:

    El Gaucho schreef op 8 april 2015 18:48:

    Zie net dat de CEO bijna 50.000 aandelen heeft verkocht afgelopen maandag... Hoe onprofessioneel! Daar gaat toch een enorme signaalwerking van uit!
    mogelijk moeten CEO's lange tijd van tevoren aangeven wanneer ze willen verkopen. Je zou denken dat deze voorkennis heeft, maar de kans is groter dat hij deze strategische verkoop al veel eerder heeft ingegeven (hij is ook niet op de top uitgestapt).
  10. flosz 11 april 2015 14:56
    $CLDN $QURE LifesciCapital: Celladon is Approaching Expected Release of Phase IIb Heart Failure Data
    Celladon Corporation (NasdaqGM: CLDN) is expected to report topline data in late April from the 250 patient CUPID 2 trial of lead candidate MYDICAR, a gene therapy for the treatment of advanced heart failure (HF). This Phase IIb trial builds on positive Phase I/IIa data for MYDICAR, which revealed an improvement in patients’ ventricular function. The data release has been eagerly anticipated since the completion of patient enrollment in March 2014. Positive results have the potential to be a major catalyst for growth for Celladon.
    ¦ CUPID 2 Trial Results May Validate Celladon’s Gene Therapy Approach.
    MYDICAR is an adeno-associated virus (AAV)-mediated gene therapy designed to
    overexpress the sarco/endoplasmic reticulum Ca2+-ATPase (SERCA2) protein in cardiac muscle cells. Upregulation of this protein in the heart has been shown to improve contractility and relaxation of heart muscle in preclinical studies. Data from the CUPID 1 Phase I/IIa trial demonstrated an encouraging and statistically significant clinical benefit
    for HF patients. An announcement of positive results from the CUPID 2 trial may serve as an important validation of Celladon’s approach, including both the use of viral vectors and the targeting of intracellular calcium dynamics in cardiomyocytes to treat HF.
    ¦ Successful CUPID 1 Trial Supports the Ongoing Development of MYDICAR.
    Celladon reported positive results from its CUPID 1 Phase I/IIa clinical trial testing the safety and efficacy of MYDICAR in advanced systolic HF patients. Phase I of this study was an open-label, sequential dose-escalation study examining intracoronary infusion of three doses of MYDICAR versus placebo in 12 advanced HF patients. This study provided preliminary evidence of safety and efficacy. The Phase IIa portion was a randomized, double-blind, placebo-controlled trial of MYDICAR in 39 systolic HF patients, who received one of three doses of MYDICAR on top of their existing HF therapy. These patients were monitored for 12 months with an additional 2 years of telephone follow-ups.
    The primary study endpoint was an improvement in multiple efficacy categories without a worsening in any of them, and the secondary endpoint is safety. The seven efficacy parameters were assessed in four domains:
    ? Left ventricular (LV) function determined by ejection fraction and end-systolic volume.
    ? Symptom improvement scored by New York Heart Association (NYHA) and Minnesota
    Living with Heart Failure Questionnaire.
    ? Cardiac function tested with a six-minute walk test and peak max oxygen consumption.
    ? Reductions in N-terminal prohormone brain natriuretic peptide (NT-proBNP) levels, a biomarker of cardiovascular disease.
    Expected Upcoming Milestones
    ¦ Late April 2015 - Top-line data expected from CUPID 2 trial evaluating MYDICAR
    in systolic heart failure.
    ¦ 2015 – End of Phase II meetings with FDA and EMA to discuss CUPID 2 results.
    ¦ 2015 – Initiate CUPID 3 trial.
    ¦ 2015 – Presentation of full CUPID 2 dataset at major medical conference.
    ¦ 2016 – Initiate MYDICAR trial in diastolic heart failure indication (HFpEF).

    The high-dose group achieved the primary endpoint at 6 and 12 months as compared to placebo.1 Patients in the high-dose group
    showed improvements or stabilization in all four efficacy categories being studied. In particular, from baseline to 6 months, the LV end-systolic volume, which is the amount of blood that the heart failed to eject during a contraction, decreased by 10 ± 28 mL in the high-dose group as compared to an increase of 18 ± 39 mL in the placebo group. The graph in Figure 1 illustrates the reduction in
    cumulative clinical events for patients treated with high-dose MYDICAR as compared with the placebo group. Over the course of the three-year follow-up period there was a significant 82% reduction in the frequency of non-terminal events for high-dose MYDICAR treated patients compared to placebo (p=0.048), as measured by hospitalizations and myocardial infarctions. No safety concerns were
    noted during the three-year follow up period for patients who received MYDICAR.
    Figure 1. Rate of Clinical Events During CUPID 1 Phase II Trial Over 3 Year Period
    Source: Zsebo, K. et al., 2014 Celladon also found that the SERCA2a transgene introduced with MYDICAR infusion persisted long-term in the target tissue in 2
    patients – one at 31 months and one at 22 months – while a third patient still possessed AAV DNA at month 23. All three of these patients were from the high-dose group.
    ¦ CUPID 2 Phase IIb Trial Design. CUPID 2 is a double-blind, placebo-controlled Phase IIb trial evaluating MYDICAR for the treatment of HF.2
    The trial enrolled 250 patients who were randomized 1:1 to receive an intracoronary infusion of either 1 x 1013 particles of MYDICAR or placebo. After treatment, patients were observed until the trial accumulated at least 186 adjudicated events
    and all patients were observed for at least 12 months. The patients will undergo periodic follow-ups for 5 years. Inclusion criteria called for patients 18 to 80 years of age with systolic heart failure, ejection fraction measurements below 35%, having Class II, III and IV symptoms of HF on the NYHA scale, negative for AAV neutralizing antibodies (nAbs), and at high-risk of HF-related hospitalizations
    The primary endpoint of this study is time to recurrent HF-related hospitalization, which was accepted by the FDA in 2012 in a Special Protocol Assessment as an acceptable endpoint for pivotal trials. Secondary endpoints include time-to-first terminal event, symptoms, exercise capacity, quality of life, and safety. In April 2014, the program received Breakthrough Therapy Designation from the FDA.
    ¦ Heart Failure Represents a Significant Market Opportunity. According to the American Heart Association, there are about 6 million people in the United States and over 23 million people worldwide who suffer from heart failure (HF). We estimate that the target market for MYDICAR as currently indicated is around 360,000 patients in the US, with another 900,000 in Europe. There is an
    opportunity for any therapy that can significantly reverse the disease course and reduce the number of cardiac events.

    Captain Sequence
    mobile.twitter.com/captainfuture__

  12. [verwijderd] 22 april 2015 22:01
    CLDN stort aardig in (zojuist ruim 17% op 13,46$....gehalveerd in een maand tijd zonder bericht?), zeker na de sterke stijging van een tijdje terug...iemand idee waarom?

    QURE daardoor in kielzog? Ik vind niet zozeer relevante berichten en de koers van QURE blijft onrustig de bodem testen.
  13. [verwijderd] 22 april 2015 22:09
    thnx Prof, ik zet de link er meteen bij:

    www.nasdaq.com/article/celladon-cupid...

    Celladon: CUPID-2 Is Likely To Fail
    By SeekingAlpha, April 22, 2015, 09:21:54 AM EDT

    By Blair O'Neill :

    Much has been said among analysts ( here and here ), and Seeking Alpha authors ( here and here ), about Celladon's (
    CLDN
    ) MYDICAR's monumental upside if the upcoming Phase 2 trial results are positive. However, very little has been said about the chance for successful results. Therefore, the focus of this report is to look at the following:

    The preclinical and clinical data supporting Celladon's case
    Odds for CUPID-2's success
    Potential outcomes for the stock price

    MYDICAR

    "MYDICAR is designed to restore levels of an enzyme known to play a key role in the progression of heart failure." - Celladon

    That enzyme is called SERCA2a and is critical for the contraction of the cardiac muscle cell. If the enzyme becomes depleted, the heart's contraction cycle becomes unbalanced, which can lead to end-stage heart failure.

    If it were widely believed that increasing SERCA2a levels would lead to a meaningful clinical benefit, then maybe MYDICAR could have been approved based upon a surrogate endpoint. However, the FDA is wary when it comes to the use of unproven surrogate endpoints, and so Celladon must instead show that MYDICAR produces a meaningful clinical benefit.

    Preclinical data in pigs

    So far, Celladon has some preclinical data to show that MYDICAR can increase SERCA2a expression in the cardiac tissue. As for improving heart function, the data is a bit of a mixed bag.

    Celladon measured the following mean values at baseline, during the injection period 2 months later, and 2 months after that:

    Peak left-ventricle (LV) pressure: Peak blood pressure during a contraction
    End-distolic pressure: Blood pressure in ventricle immediately before contraction
    End-systolic pressure: Blood pressure in ventricle at the end of contraction
    LV internal distolic diameter: Width of LV immediately before contraction
    LV internal systolic diameter: Width of LV immediately after contraction
    Left-ventricle ejection fraction (LVEF): Percentage of blood pumped out of the heart with each contraction
    A couple of endpoints relating to how quickly the peak LV pressure is reached

    We only know of the LV internal systolic diameter, LVEF, and peak LV pressure rate endpoints showing a statistically significant benefit.

    Upon further examination, it appears that the endpoints relating to how quickly peak LV pressure was reached were marred by baseline imbalances, and so we're left with the other 2 remaining endpoints. My concern is that the baseline imbalances seen with the peak LV pressure rate could be a sign that the control arm had weaker cardiac muscles. However, such imbalances were not evident in the reported mean baselines for LVEF.

    Interestingly, after the heart failure was induced in the pigs, by poking a hole in the mitral valve, the experimental arm was measured as leaking 9% more blood than the control arm. Another 2 months later, the control arm would be leaking 30% more blood than the experimental arm. There is no clear explanation for why the leaking rate increased so dramatically in the control arm, when it had remained relatively unchanged in the experimental arm.

    Regardless, what makes the efficacy data such a mixed bag is the fact that no pigs in the control arm died after the injection of the placebo, while 3 would die after injection in the experimental arm (1 from general deterioration, and another from worsening HF). The data from the deceased pigs were excluded from all efficacy analysis. It seems likely that if no pigs had died, then the performance of the experimental arm would have been significantly impacted.

    In conclusion, the preclinical data seems to support MYDICAR's ability to increase SERCA2a expression. As for MYDICAR's ability to improve or maintain LVEF, that endpoint was marred by dead experimental pigs, worsening valve leaks among control pigs, and/or potential imbalances in cardiac muscle strength. So it remains unanswered whether or not the use of MYDICAR led to a meaningful enough increase in SERCA2a prevalence in order to cause the observed cardiac benefits.

    In later presentations , the company would use median, instead of mean, to assess the preclinical endpoints. Considering how few animals were studied, I feel that the use of the median would produce even more unreliable results.

    CUPID-1's efficacy data

    This study began as a Phase 1 open-label dose-escalation study. It would compare 4 different dosages of MYDICAR, before moving onto the 2nd phase of the study. The Phase 2 portion would be a placebo-controlled randomized dose-ranging study.

    A key pre-specified efficacy endpoint , which happens to be the primary endpoint in the on-going CUPID-2 trial, was the number of patients that experience at least 1 HF-related hospitalization event, or worsening heart failure, after 12 months post-injection. Otherwise worded as:

    "Time to recurrent events (HF-related hospitalizations, ambulatory worsening HF) in the presence of terminal events (all-cause death, heart transplant, LVAD implantation)" - CUPID-1 clinicaltrials.gov page

    Here were the results for that endpoint in CUPID-1:

    (click to enlarge) CUPID-1 pre-specified efficacy endpoint

    As we can see, there was no dose response between the low and mid doses, and neither of those dosages showed a benefit that was statistically significant with respect to placebo at 12 months.

    Another important endpoint would be the change in LVEF at 12 months. Those results were:

    (click to enlarge)

    Here we see an escalating efficacy response among the different doses. However, the placebo performed better than the low and mid doses, and so we really can't say there was actually a dose response. In addition, the LVEF data was confounded by dying patients.

    Alarmingly, only 1 of the remaining 16 efficacy endpoints showed some kind of dose response while also exhibiting a consistent benefit over the placebo arm. That 1 efficacy endpoint was the mortality rate, but there were so few deaths within the 12-month period that it is probably foolish to trust it.

    (click to enlarge)

    Even if we just ignored the lower dosage arms, and just focused on the high dose arm, Celladon still wouldn't have a good case for MYDICAR's efficacy. Why? Extensive patient baseline imbalances (courtesy of @AndyBiotech ).

    In almost all of the measurements, the experimental arms had significantly healthier baseline levels than the placebo arm. Even among the experimental arms, the higher dosed patients typically had healthier baseline levels than seen among the lower dosed patients.

    Here were the prevalence of pre-existing conditions and prior use rates of other therapies (courtesy of @chasingthealpha ):

    Once again, we see that the baseline characteristics favor the high-dose experimental arm by a considerable margin. You can look at a more comprehensive review of the CUPID-1 trial and results under the Publications section of this page.
  14. Prof. Dollar 22 april 2015 22:09
    quote:

    invoorentegenspoed schreef op 22 april 2015 22:01:

    CLDN stort aardig in (zojuist ruim 17% op 13,46$....gehalveerd in een maand tijd zonder bericht?), zeker na de sterke stijging van een tijdje terug...iemand idee waarom?

    QURE daardoor in kielzog? Ik vind niet zozeer relevante berichten en de koers van QURE blijft onrustig de bodem testen.
    Zie andere draadje. ;-)
    www.iex.nl/Forum/Topic/1286603/108/Wa...
  15. forum rang 10 rationeel 24 april 2015 10:36
    Grafiek van CELLADON:

    Het aandeel heeft een performance gemaakt van 7,28 naar 28,25. Het heeft vervolgens een retracement neergezet van 76,4%. Laagste koers 12,70. 76,4% is 12,64. Met de eindkoers blijft het aandeel in een uptrend. Zou de eindkoers lager zakken dan start een down trend.
    De indicator geeft aan, dat het zo wel weer eens mooi kan zijn geweest;)
  17. forum rang 10 rationeel 24 april 2015 10:55
    Om even te reageren op de opmerking...De koers stort in... In ta termen het retracen. Retracen is een onderdeel van het koersverloop van een aandeel. De mate waarin dat gebeurt geeft een indicatie van de stijgingskracht van het aandeel. Stel bij U blijft het bij 38,2%, dan is dat dus sterker dan de 76,4% van C.
  18. flosz 26 april 2015 23:27
    Celladon Reports Negative Results for CUPID2 Trial of MYDICAR(R) in Advanced Heart Failure

    - Investigational gene therapy fails to meet primary and secondary endpoints -

    - Investor conference call and webcast Monday at 8:30 a.m. ET (5:30 a.m. PT) -

    SAN DIEGO, April 26, 2015 (GLOBE NEWSWIRE) -- Celladon Corporation (Nasdaq:CLDN) today announced that its Phase 2b CUPID2 trial did not meet its primary and secondary endpoints. CUPID2 is a randomized, double-blind, placebo-controlled, multinational trial evaluating a single, one-time, intracoronary infusion of the cardiovascular gene therapy agent MYDICAR® (AAV1/SERCA2a) versus placebo added to a maximal, optimized heart failure drug and device regimen.

    In the study, the primary endpoint comparison of MYDICAR to placebo resulted in a hazard ratio of 0.93 (0.53, 1.65 95%CI) (p=0.81), defined as heart failure-related hospitalizations or ambulatory treatment for worsening heart failure. The secondary endpoint comparison of MYDICAR to placebo, defined as all-cause death, need for a mechanical circulatory support device, or heart transplant, likewise failed to show a significant treatment effect. The efficacy endpoint analyses were performed on the (n=243) modified intent to treat population (mITT), which excludes clinical events that occurred in patients who did not receive MYDICAR or placebo, or which occurred prior to dosing. All other exploratory efficacy endpoints (improvement in New York Heart Association classification, 6 Minute Walk Test, and Quality of Life) were also inconsistent with a treatment effect. No safety issues were noted.

    "We are surprised and very disappointed that MYDICAR failed to meet the endpoints in the CUPID2 trial, and we are rigorously analyzing the data in an attempt to better understand the observed outcome. We would like to express our sincere gratitude to our investigators and patients who participated in the study," said Krisztina Zsebo, Ph.D., CEO of Celladon. "At the same time we are evaluating our other programs in order to determine the best path forward to maximize shareholder value."

    "This trial was extremely well executed and adequately tested the hypothesis, but the therapy failed to achieve the primary and secondary endpoints. However, there were no safety issues," said Barry Greenberg, M.D., FACC, Director, Advanced Heart Failure Treatment Program; Distinguished Professor of Medicine, University of California, San Diego, and the Chairman of the Executive Clinical Steering Committee of the CUPID2 trial.

    About the CUPID2 Study

    CUPID2 is a Phase 2b, randomized, double-blind, placebo-controlled, multinational trial evaluating a single intracoronary infusion of the cardiovascular gene therapy agent MYDICAR versus placebo added to a maximal, optimized heart failure regimen. The study included 250 adult patients who had stable NYHA (New York Heart Association) class II to IV ischemic or non-ischemic heart failure despite optimal therapy, reduced left ventricular ejection fraction (= 35%) and a high risk for recurrent heart-failure hospitalizations. CUPID2 enrolled only patients with heart failure with reduced ejection fraction (HF-REF). The statistical analysis for the primary endpoint was performed on the modified intent to treat population (mITT), comprising all patients who, after randomization, underwent cardiac catheterization and drug or placebo administration.

    The primary endpoint was time to recurrent heart failure related events (defined as heart failure-related hospitalizations or ambulatory treatment for worsening heart failure), using a statistical analysis methodology called joint frailty modeling. The secondary efficacy endpoint was time to first terminal event (defined as all-cause death, heart transplant or placement of a mechanical circulatory support device), analyzed simultaneously with the primary endpoint using joint frailty modeling.

    Conference Call & Webcast

    Management will host an investment community conference call to discuss the information in this press release.

    ir.celladon.net/releasedetail.cfm?Rel...
  19. [verwijderd] 27 april 2015 16:16
    pfff 80% onderuit...1 produkt gedreven bedrijf meen ik? Tja, dan blijft er weinig meer heel.

    Daarom is een basistechnologie van belang, meerdere partners op verschillende trials...lukt het ene niet, dan kans met het ander...dan is QURE wat minder riskant als ik mijn bescheiden mening mag geven.

    finance.yahoo.com/q?s=cldn&ql=1
  20. [verwijderd] 27 april 2015 16:28


    www.thestreet.com/story/13126476/1/ge...

    Gene Therapy Stocks on Watch Following Failure of Celladon's Heart Failure Trial
    By
    Adam Feuerstein
    Follow
    | 04/26/15 - 08:45 PM EDT
    Exclusive FREE Report: Jim Cramer's Best Stocks for 2015.
    2

    SAN DIEGO (TheStreet) -- Celladon (CLDN) said Sunday that its experimental gene therapy for heart failure, Mydicar, was a total bust. To its credit, management was fully transparent about the mid-stage clinical trial results, even owning up to "negative results" in the headline of the press release. On the primary and secondary endpoints of the heart failure study, Celladon's Mydicar failed to demonstrate any benefit over placebo, the company said..

    Shares of Celladon will surely collapse Monday, but more interesting will be seeing what effect the company's blow up has on other high-flying gene therapy stocks -- Bluebird Bio (BLUE - Get Report), UniQure (QURE), Spark Therapeutics (ONCE) -- if at all.

    Investor demand for gene therapy stocks has been almost insatiable recently, erasing years of scientific doubts about the technology's ability to cure or even treat disease. No gene therapy stock exemplifies the investor mania more than Bluebird. Since December when Bluebird presented early, preliminary data suggesting its gene therapy might lead to a cure for the rare blood disorders beta thalassemia and sickle cell anemia, the company stock price has shot up 245%.

    Bluebird went public in June 2013 at a market value of just under $400 million. At Friday's close of $136.26, the company was worth more than $4.4 billion.

    QURE Chart

    Gene therapy is an umbrella term describing a technology which uses engineered viruses to replace defective, disease-causing genes. There are important differences in how individual gene therapies are formulated and delivered. Some diseases may be more amenable than others to treatment or cure with gene therapy. Logically, Celladon's Mydicar failure in heart failure doesn't necessarily doom the entire field. But it's also reasonable to ask if valuations of gene therapy stocks have grown too big too fast given the risks that still clearly exist.

    Celladon's Mydicar is a virus engineered to insert a working gene capable of producing a protein called SERCA2a into heart-failure patients. SERCA2a is responsible for helping heart muscles contract and pump blood more efficiently. Heart-failure patients have low levels of SERCA2a and hearts that do a poor job pumping blood around the body. Celladon was hoping to demonstrate that infusing Mydicar via a one-time infusion into a vein near the heart would lead to higher SERCA2a levels and improved heart function.

    Those hopes were dashed. In the largest study of a gene therapy in heart failure ever conducted, Mydicar reduced the risk of heart-failure related hospitalization by only 6 percent compared to a placebo. The difference was not statistically significant. Mydicar also failed to demonstrate a benefit over placebo across additional secondary endpoints, Celladon said.

    Celladon shares closed Friday at $13.68 ahead of Sunday's Mydicar results. The stock traded as high as $27.26 per share on March 19, but fell on concerns about mixed results from a previous Mydicar study and analysis of preclinical data suggesting the gene therapy wasn't potent enough to produce enough functional SERCA2a protein.

    The company has little else to fall back on now that Mydicar appears to be done in heart failure. The remainder of Celladon's pipeline is still in preclinical stages of development. The company had $85 million in cash at the end of 2014, equal to $3.57 per share.

    The next clinical catalyst for gene therapy of interest to investors may come in June when Bluebird is expected to present data on its gene therapy for sickle cell disease. The company has not confirmed a sickle cell disease update but abstracts for the European Hematology Association meeting, where the data would be presented, will be made public on May 21.

    Avalanche Biotechnologies (AAVL) and UniQure are also expected to announce results from gene therapy studies later this year.
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