Capricor Therap
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Im Q2 kommen die Endergebnisse für die Phase 2 der Duchenne Muscular Dystrophy Studie von Capricor.
Die Aktie steht nahe des Allzeittief und ist finanziert bis Q1 2021 etwa.
Dabei ist ein großer potenzieller Gewinnhebel dieser Aktie ihr geringer Marktwert von etwa 5Mio €. Bei positiven Nachrichten, wenn der Kurs nur auf 25mio steigt, hätten wir eine Verfünffachung der Kurspreises.
Ein mögliches DMD-Medikament hätte Blockbuster-Umsatz (Seite. 3 https://capricor.com/wp-content/uploads/2014/05/...tation-100217.pdf)
Die Zwischenergebnisse schienen ganz ordentlich gewesen zu sein (muss dort aber noch nachforschen etwas- gerne gemeinsam in der Runde).
Für einen kurzfristigen Zock mit der Möglichkeit großer Profite, sehe ich gute Chancen.
Risiko und alle Angaben sind natürlich ohne Gewähr, jeder auf sein eigenen Risiko. Bin heute auf Tiefstkurs eingestiegen.
Per CEO Marban, the company is in active conversations with several suitors regarding partnerships or other arrangements that would result in non-dilutive financing to help accelerate commercialization of CAP-1002.
Capricor is well funded at this time having enough funds to fund operations through 1Q 2021 following a December 2019 equity raise of $5.1M.
Investors should realistically expect news regarding FDA discussions related to accelerated approval and/or partnerships/buyout in the near future based on 3Q 2019 conference call comments.
On October 11, 2019 HC Wainwright reiterated a “buy” rating on CAPR stock and set a $19.50/share price target. Capricor was also recently upgraded by equities research analysts at ValuEngine from a “hold” rating to a “buy” rating.
https://seekingalpha.com/instablog/...ial-300-upside-on-upcoming-news
19,5 $ wären keine 100 Mio MK für eine Firma, die einen Blockbusterkandidaten in der Pipeline hat. Entweder fallen wir 50-70% oder wir holen uns mindestens einen 5facher(25Mio MK) ins Haus. Risiko/Gewinn-Chancen sehe ich mehr als fair.
“I am incredibly pleased with the outcome of the interim analysis as it has demonstrated the biologic activity of CAP-1002 that has resulted in changes of clinically relevant outcomes including the upper limb, the hand and diaphragmatic function,” said Craig McDonald, M.D., the national principal investigator for the HOPE-2 clinical trial and UC Davis professor and chair of its Department of Physical Medicine and Rehabilitation. “For these older boys who have no further therapeutic options, these data support the hope that CAP-1002 may one day become an important therapeutic option and possibly slow the advancement of the disease.
HOPE-2 is a randomized, double-blind, placebo-controlled, Phase II clinical trial of the company’s lead investigational therapy, CAP-1002 in steroid-treated boys and young men who are in advanced stages of Duchenne muscular dystrophy (DMD), a debilitating genetic disorder. DMD is characterized by progressive weakness and chronic inflammation of the skeletal, heart, and respiratory muscles. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months.
In the interim analysis, top-line data from a total of 17 patients was analyzed in the per protocol population (10 placebo and 7 treated) at the 3 month time-point and 12 patients (6 placebo and 6 treated) were analyzed at the 6 month time-point. Approximately 80% of the patients were non-ambulant. Demographic and baseline characteristics were similar between the two treatment groups.
Skeletal Assessments
To assess skeletal muscle function, investigators used the mid-level dimension of the Performance of the Upper Limb (PUL) 1.2 and 2.0 tools. The PUL evaluates manual tasks that relate to activities of daily living that are very important for quality of life. The U.S. Food and Drug Administration (FDA) has suggested the use of the updated PUL 2.0 version as the primary efficacy endpoint in support of a Biologics License Application (BLA). Positive results were seen in the PUL 1.2 version which is consistent with the positive results seen in Capricor’s HOPE-Duchenne Phase I/II clinical trial published in Neurology, the medical journal of the American Academy of Neurology. Additional independent tests assessing grip strength showed statistically significant results at 6 months and tests assessing tip to tip pinch strength showed positive results.
Time-point§3 months 6 months
Treatment CAP-1002 Placebo p-value CAP-1002 Placebo p-value
Mid-level Performance of the Upper Limb PUL 2.0 0.1 (1.07) -0.4 (0.52) 0.0591 -0.2 (1.17) -0.8 (0.75) 0.0389
Tip to Tip Pinch Strength 0.9 (3.44) 1.9 (4.12) 0.9057 3.3 (2.88) -0.3 (1.51) 0.0674
Grip Strength -0.6 (3.15) -0.8 (2.30) 0.5897 0.8 (4.54) -2.2 (1.83) 0.0389
Table: Skeletal Assessments at 3- and 6-month time-points.
Mean Change from baseline (standard deviation) showed.
Pulmonary Assessments
To assess pulmonary function, investigators measured several clinically relevant parameters. At 3 months, inspiratory flow reserve (absolute), a reflection of diaphragmatic strength, showed a statistically significant improvement (p=0.0473). Additionally, positive trends were seen at 3 months in peak expiratory flow (% predicted), another measure of diaphragmatic strength.
Cardiac Assessments
Magnetic resonance imaging (MRI) was used to assess cardiac structure and function at 6 months. Positive trends were found in cardiac muscle function including systolic wall thickening and cardiac mass among those treated with CAP-1002 compared to placebo. Duchenne hearts atrophy progressively and have impaired systolic function. Improved mass and wall thickening suggest possible cardiac regeneration and functional improvement. Although these trends did not reach statistical significance, they were consistent with the cardiac findings seen in the previously published HOPE-Duchenne study.
Safety
In late December 2018, Capricor put a voluntary hold on dosing after two patients in the HOPE trials had a serious adverse event in the form of an immediate immune reaction. The investigation suggested the patient may have developed hypersensitivity to something contained in the investigational product, including an excipient or inactive ingredient in the formulation. To reduce the risk of future adverse events, Capricor initiated a commonly used pre-medication strategy including intravenous steroids and antihistamines to prevent or mitigate potential immune reactions during the administration. Since the initiation of the pre-treatment regimen, 30 infusions of investigational drug (CAP-1002 or placebo) have been administered to HOPE-2 patients with only one serious adverse event reported that required an overnight observation of the patient.
Summary
In summary, a statistically significant outcome relative to placebo controls was shown in PUL 2.0 at 6 months, with supportive, positive treatment effects also seen in some independent skeletal and pulmonary assessments. Positive trends, although not statistically significant, were observed in other skeletal, pulmonary and cardiac measures.
Although we have collected data from 2 treated patients at the 9-month timepoint, 1 of which at the 12-month time-point, Capricor is not able to draw any conclusions at this time with respect to this data.
“We are extremely pleased and it is truly extraordinary that even in such as small sample size, we achieved statistically significant improvements in several clinically relevant parameters. In these older patients, functional improvement in the upper limb is highly meaningful for their quality of life. To our knowledge, this is the first randomized double-blind, placebo-controlled study in DMD that has shown statistically significant functional improvement in steroid treated boys.” said Linda Marbán, president and CEO of Capricor.
Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne said, "I am encouraged by the prospects that this data has for people with Duchenne, especially our non-ambulatory community who have limited therapeutic options. Additionally, CAP-1002 potentially provides cardiac benefits in this patient population where heart failure continues to be the leading cause of mortality."
The FDA has granted Capricor RMAT and Orphan Drug Designation. Capricor met with the FDA in December 2018 as part of the expedited review afforded under the RMAT designation which the FDA granted to CAP-1002 in February 2018. Additionally, the FDA has granted a Rare Pediatric Disease Designation to CAP-1002. The Rare Pediatric Disease Designation, as well as the Orphan Drug Designation previously granted, covers the broad treatment of DMD. Upon receiving market approval for CAP-1002 by the FDA, Capricor would be eligible to receive a Priority Review Voucher.
Capricor will continue its ongoing discussions with the FDA about the DMD program and future plans.
https://www.globenewswire.com/news-release/2019/...lar-Dystrophy.html
Bei 57 Infusionen gab es zwei ernste Vorfälle von allergischen Reaktionen. Man hat dann das Medikationsschema geändert und Antihistaminika hinzugefügt, so wie ich das verstanden habe. http://capricor.com/wp-content/uploads/2018/04/CAPR-HOPE-2-K…
Ich kann schon etwas nachvollziehen, wieso der Kurs sich dann so schwer tut. Etwas creepy ist auch, dass ein "Rohstoff" von CAP-1002 Spenderherzen sind, die weiterverarbeitet werden.
Naja, hoffen wir auf gutes gelingen der Phase.
Man darf gespannt sein.
http://www.globenewswire.com/news-release/2020/03/...on-March-18.html
“One of the reasons the exosomes are potentially so useful and transformative is their ability to speak the language of a cell. We are excited by the commitment of Dr. Gould to help us explore the potential of exosome-based vaccines to help prevent human diseases and exosome-based therapeutics in treating human diseases. We look forward to announcing more updates shortly which will further outline some of our near-term goals within our exosomes program,” said Linda Marbán, Ph.D., Capricor’s president and chief executive officer.
Dr. Gould stated, “Exosomes are the body’s natural way of sending complex signals between cells and tissues. As a result, exosome-based vaccines have the potential to elicit more effective immune reactions against infectious agents and cancers, while exosome-based therapeutics have the potential to stabilize drugs and deliver them to their intended site of action. In addition, Capricor’s extensive expertise in cell culture and exosome purification provide it with numerous advantages for the scale-up and processing of exosome-based products, and together we will work to apply that ability to the production of viral vaccines, vesicle-mediated protein therapies, and treatment of inherited diseases. It’s a pleasure to be working with the talented Capricor team as we work together to develop exosome-based vaccines and drugs.”
Capricor has presented strong preclinical data using its proprietary cardiosphere-derived cell (CDC)-exosomes in many animal models. More specifically, Capricor has utilized this technology in pre-clinical studies of inflammation and intense immune activation such as DMD, sepsis, Graft- versus-host disease (GVHD) and trauma. While Capricor’s CDC-derived exosomes have strong clinical potential, Capricor is committed to developing precision-engineered exosomes that carry defined sets of effector molecules which exert their effects through defined mechanisms of action.”
http://www.globenewswire.com/news-release/2020/03/...s-Expansion.html
In Ami-Foren wird überlegt, dass dieses Programm zur Entwicklung von Impfstoffen gegen Virusinfektionen unter Verwendung von CARPs Exosomen basierenden Technologieplattform auch zur Verwendung gegen das Corona Virus genutzt werden könnte. Morgen werden wir in der Telefonkonferenz ja dann sehen. Ich erwarte eigentlich durch die positive Phase für das DMD, welche uns eine Kursexplosion wie bei den positiven Interimsdaten. Der Kurs scheint ja schon anzuziehen. Bin wieder im Plus. Hatte bei 0,9€ noch mal nachgelegt und mein EK gedrückt.
Vorbörslich Capricor Therapeutics Inc
CAPR 1.55
0.38 (32.48%)
https://newsfilter.io/a/f5570abb3292eba23d478f24c16e8337
Ich bin hier wegen dem DMD-Medikament drine, aber nehme den Push nehme ich gerne mit.
The Company reported a net loss of approximately $1.5 million, or $0.34 per share, for the fourth quarter of 2019, compared to a net loss of approximately $3.3 million, or $1.05 per share, for the fourth quarter of 2018. As of December 31, 2019, the Company’s cash, cash equivalents and marketable securities totaled approximately $9.9 million, compared to approximately $7.3 million on December 31, 2018. Additionally, in 2019, Capricor raised approximately $4.8 million in net proceeds at an average price of approximately $4.48 per share under its at-the-market offering programs. Capricor believes that based on the current operating plan and financial resources, the Company expects that the cash, cash equivalents and marketable securities at December 31 will be sufficient to cover expenses and capital requirements through at least the second quarter of 2021.
http://www.globenewswire.com/news-release/2020/03/...rate-Update.html
Finanziert bis 2021 Q2. Wir warten die DMD-Ergebnisse in Q2 2020 ab und sind seit neuestem ein Corona Play. Mit MK bei 5-8Mio. Gute Vorrausetzungen schon mal. Der Kurs hat die Tage schon Ausbruchversuche gestartet. Wäre schön, wenn wir den 1er Bereich endlich überwinden würden.
Überlege noch heute nachzulegen. Mein EK liegt bei 1,02
Neuer Artikel:
https://seekingalpha.com/instablog/...rate-covidminus-19-vaccines-and
am 26.3. conference call. Vielleicht kriegen wir paar Infos.
https://finance.yahoo.com/news/...ics-host-key-opinion-120010443.html