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safe to think AA is given in a few months
$100 coming sooner
CAPR
monstrous move by huge FDA news today
Another explosion over $10 tmrw again
$50 by Christmas time and $100 by Spring next year
Super volcanic explosion
CAPR
keep going up
FDA news is humongous indeed
Analyst price target raised to $40 today
CAPR
Potential 1000% runner in a few months
FDA nodded to file BLA finally
99% Accelerated Approval now
CAPR
Keep going higher
$15 doable today or tmrw
Huge FDA BLA news!
We will be millionaires very soon
CAPR
breaking out now
Buy all you can before $10
CAPR
1000% runner in the making very soon
Good luck all
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, announced today, following recent meetings with the U.S. Food and Drug Administration (FDA), its intent to file a Biologics License Application (BLA) based on existing cardiac and natural history data for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy.
Following the FDA meetings:
Capricor plans to commence the filing of a BLA in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected by year-end 2024.
The BLA filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center.
In order to support potential label expansion to treat DMD skeletal muscle myopathy, Capricor plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024.
“There are currently no approved therapies for DMD cardiomyopathy, which is the leading cause of death in those with Duchenne. Based on the strength of our cardiac data, combined with the FDA’s commitment to advancing therapeutics for the treatment of rare diseases, we are seeking approval for the cardiomyopathy associated with DMD and will look to expand the label for skeletal muscle myopathy post-approval,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “This approach is the result of multiple in-depth meetings with FDA where we showed robust and positive cardiac data from our HOPE-2 and HOPE-2 OLE studies compared to natural history data from a large cohort of patients.”
Dr. Marbán continued, “Deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. Based on the totality of evidence of the safety and efficacy data deramiocel has shown, we believe this is the best path forward to potential approval, allowing us to bring this novel, first-in-class treatment to patients in need in the most expeditious manner. We want to extend our appreciation to the patients, their families and advocates who continue to work with us and to the FDA for their commitment to accelerating treatments for DMD.”
Deramiocel for the treatment of DMD, has received FDA Orphan Drug Designation and the regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for deramiocel for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, announced today, following recent meetings with the U.S. Food and Drug Administration (FDA), its intent to file a Biologics License Application (BLA) based on existing cardiac and natural history data for deramiocel to treat all patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy.
Following the FDA meetings:
Capricor plans to commence the filing of a BLA in October of 2024 seeking full approval of deramiocel for the treatment of DMD-cardiomyopathy with full submission expected by year-end 2024.
The BLA filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to natural history data provided by Vanderbilt University Medical Center and Cincinnati Children’s Hospital Medical Center.
In order to support potential label expansion to treat DMD skeletal muscle myopathy, Capricor plans to combine Cohorts A and B of the Phase 3 HOPE-3 clinical trial to serve as a post-approval study and does not intend to unblind Cohort A at this time, which was expected to occur in the fourth quarter of 2024.
“There are currently no approved therapies for DMD cardiomyopathy, which is the leading cause of death in those with Duchenne. Based on the strength of our cardiac data, combined with the FDA’s commitment to advancing therapeutics for the treatment of rare diseases, we are seeking approval for the cardiomyopathy associated with DMD and will look to expand the label for skeletal muscle myopathy post-approval,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “This approach is the result of multiple in-depth meetings with FDA where we showed robust and positive cardiac data from our HOPE-2 and HOPE-2 OLE studies compared to natural history data from a large cohort of patients.”
Dr. Marbán continued, “Deramiocel has shown in multiple clinical trials attenuation of the cardiac implications of DMD. Based on the totality of evidence of the safety and efficacy data deramiocel has shown, we believe this is the best path forward to potential approval, allowing us to bring this novel, first-in-class treatment to patients in need in the most expeditious manner. We want to extend our appreciation to the patients, their families and advocates who continue to work with us and to the FDA for their commitment to accelerating treatments for DMD.”
Deramiocel for the treatment of DMD, has received FDA Orphan Drug Designation and the regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation). In addition, if Capricor were to receive FDA marketing approval for deramiocel for the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease designation.
CAPR
Humongous news just announced
FDA BLA news!!!
CAPR
keep going up AH
Huge news pendingbtmrw
$10 tmrw
Big FDA AA news
CAPR
Huge explosion tmrw
FDA AA news coming
Billion dollars milestone
SAN DIEGO, Sept. 17, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced it has entered into a binding term sheet with Nippon Shinyaku Co., Ltd., a Japanese pharmaceutical company listed on the TYO, for the commercialization and distribution in Europe of Capricor’s lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD), a rare neuromuscular disease with limited treatment options. The potential transaction covered by the term sheet is similar to the existing Commercialization and Distribution Agreements with Nippon Shinyaku in the United States and Japan with an opportunity for further product reach globally. In addition, Nippon Shinyaku has agreed to purchase approximately $15 million of Capricor common stock at a 20% premium to the 60-day VWAP.
Under the terms of the binding term sheet and further subject to finalization of a Definitive Agreement, which is expected to occur in the fourth quarter of 2024, Capricor will be responsible for the development and manufacturing of deramiocel for potential approval in all countries in the European Union, United Kingdom and several other countries in the region. Nippon Shinyaku will be responsible for the sales and distribution of deramiocel in those territories. Capricor will also receive an upfront payment of $20 million subject to execution of the Definitive Agreement and there are potential additional development and sales-based milestone payments to Capricor of up to $715 million and Capricor will receive a double-digit share of product revenue.
“Our expanded partnership with Nippon Shinyaku into the European region marks a pivotal moment for Capricor as we work together to bring deramiocel to DMD patients worldwide,” said Linda Marbán, Ph.D., Capricor’s Chief Executive Officer. “With the addition of the upfront payment and equity investment, we will be able to extend our runway into 2026 and be well positioned to advance toward potential approval of deramiocel in the United States and beyond. Furthermore, these funds will provide necessary capital for commercial launch preparations, manufacturing scale-up and product development for Europe, as we envision high global demand for deramiocel.”
Dr. Marbán continued, “As previously reported, we held a successful pre-BLA meeting with the U.S. Food and Drug Administration (FDA) in August. Since that meeting, we have now had several additional informal meetings with the agency to continue to refine our approval pathway for deramiocel in the United States and we plan to provide further updates as they become available.”
Toru Nakai, President of Nippon Shinyaku, commented, “We look forward to building deramiocel’s commercial footprint around the world and this partnership would allow us to continue to invest in Nippon Shinyaku’s DMD franchise and to potentially advance life-changing therapies for patients in need.”
Contemporaneously with the term sheet, Nippon Shinyaku has also agreed to purchase 2,798,507 shares of common stock at a price of $5.36 per share, which price represents a 20% premium to the 60-day volume-weighted average price (VWAP) of Capricor’s common stock, for an aggregate purchase price of approximately $15 million. The closing of the offering is expected to take place on or about September 20, 2024. The Company expects to use the proceeds from the transaction primarily to support product development as well as general, administrative and corporate purposes.
The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended (the “Securities Act”), or applicable state securities laws. Accordingly, the securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. As part of the transaction, the Company has agreed to file a registration statement with the Securities and Exchange Commission for purposes of registering the resale by the investors of the shares of common stock purchased by such investors.
CAPR
Huge explosion upon great news today
NKGN
keep going up
Huge Alzheimer news released this morning
The billion dollar stock indeed
NKGN
breaking out
NKGN
SANTA ANA, Calif., Sept. 12, 2024 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (Nasdaq: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics, today announced promising early interim data from the Phase 1 cohort and the dosing of the first Phase 2 patient in the Phase 1/2a clinical trial of troculeucel, NKGen’s cryopreserved autologous enhanced NK cell therapy for the treatment of moderate Alzheimer’s disease (“AD”).
The Phase 1 cohort is already demonstrating positive interim results. Early independent review of the data shows that after only three months treatment with a dose of 6 billion cells per treatment, the three patients in the Phase 1 cohort had no drug-related adverse reactions, and exploratory efficacy analyses showed that two of the three patients went from a moderate to a mild AD rating on the Clinical Dementia Rating-Sum of Boxes (“CDR-SB”) scale. These findings further support the company’s MX04 Phase 1 AD study, where the one moderate AD patient who received the highest dose in that study (4 billion cells) also went from a moderate to a mild rating on the CDR-SB. Additionally, six-month interim cognitive data from Phase 1 cohort patients receiving troculeucel is expected to be disclosed at an upcoming national Alzheimer’s conference in Q4 2024.
Building upon positive interim Phase 1 results of troculeucel in moderate Alzheimer’s patients, the Company is pleased to have dosed its first patient in the Phase 2 randomized, double-blind, placebo-controlled trial evaluating the efficacy of troculeucel compared to placebo in patients with moderate Alzheimer’s disease. Troculeucel will be evaluated for effectiveness and additional safety in a broader cohort of 30 patients with moderate Alzheimer’s disease, employing a randomized, double-blind setup (n=20 randomly assigned to the treatment arm and n=10 to placebo). The Phase 2 trial aims to provide a thorough understanding of both the potential benefits and limitations of troculeucel in treating Alzheimer’s disease, thereby validating its potential therapeutic efficacy. NKGen has now activated four clinical sites across North America and expects to increase enrollment in the coming months.
“We continue to make great progress with our troculeucel clinical program in AD,” said Paul Y. Song, MD, Chairman and CEO of NKGen. “In the Phase 1 cohort of the Phase 1/2a study, we can proudly announce two of our first three patients have been clinically upgraded from moderate to mild AD following only three months of treatment with troculeucel. Additionally, we have recently dosed the first patient in the Phase 2 cohort, which is a significant milestone, particularly since the dose used is cryopreserved and continuing at our highest dosing of 6 billion cells per treatment. With encouraging cognitive improvements and a favorable safety profile from the trial’s Phase 1 interim analysis, dosing our first patient in Phase 2 is a crucial step towards creating a much-needed treatment option for patients facing this challenging condition. We are particularly eager to explore the enhanced cognitive benefits that ongoing usage of the higher dosing may bring in our Phase 2 trial.”
SANTA ANA, Calif., Sept. 12, 2024 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (Nasdaq: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics, today announced promising early interim data from the Phase 1 cohort and the dosing of the first Phase 2 patient in the Phase 1/2a clinical trial of troculeucel, NKGen’s cryopreserved autologous enhanced NK cell therapy for the treatment of moderate Alzheimer’s disease (“AD”).
The Phase 1 cohort is already demonstrating positive interim results. Early independent review of the data shows that after only three months treatment with a dose of 6 billion cells per treatment, the three patients in the Phase 1 cohort had no drug-related adverse reactions, and exploratory efficacy analyses showed that two of the three patients went from a moderate to a mild AD rating on the Clinical Dementia Rating-Sum of Boxes (“CDR-SB”) scale. These findings further support the company’s MX04 Phase 1 AD study, where the one moderate AD patient who received the highest dose in that study (4 billion cells) also went from a moderate to a mild rating on the CDR-SB. Additionally, six-month interim cognitive data from Phase 1 cohort patients receiving troculeucel is expected to be disclosed at an upcoming national Alzheimer’s conference in Q4 2024.
Building upon positive interim Phase 1 results of troculeucel in moderate Alzheimer’s patients, the Company is pleased to have dosed its first patient in the Phase 2 randomized, double-blind, placebo-controlled trial evaluating the efficacy of troculeucel compared to placebo in patients with moderate Alzheimer’s disease. Troculeucel will be evaluated for effectiveness and additional safety in a broader cohort of 30 patients with moderate Alzheimer’s disease, employing a randomized, double-blind setup (n=20 randomly assigned to the treatment arm and n=10 to placebo). The Phase 2 trial aims to provide a thorough understanding of both the potential benefits and limitations of troculeucel in treating Alzheimer’s disease, thereby validating its potential therapeutic efficacy. NKGen has now activated four clinical sites across North America and expects to increase enrollment in the coming months.
“We continue to make great progress with our troculeucel clinical program in AD,” said Paul Y. Song, MD, Chairman and CEO of NKGen. “In the Phase 1 cohort of the Phase 1/2a study, we can proudly announce two of our first three patients have been clinically upgraded from moderate to mild AD following only three months of treatment with troculeucel. Additionally, we have recently dosed the first patient in the Phase 2 cohort, which is a significant milestone, particularly since the dose used is cryopreserved and continuing at our highest dosing of 6 billion cells per treatment. With encouraging cognitive improvements and a favorable safety profile from the trial’s Phase 1 interim analysis, dosing our first patient in Phase 2 is a crucial step towards creating a much-needed treatment option for patients facing this challenging condition. We are particularly eager to explore the enhanced cognitive benefits that ongoing usage of the higher dosing may bring in our Phase 2 trial.”
NKGN
Huge news out
I have a lot of shares here
The final stage for FDA approval for billion dollars market
My price target is $50 by the end of this year and $100 by June next year
I will make million dollars here
Breaking out over $10 upon FDA news
FDA news is imminent
Time for super volcanic explosion
FDA BLA news imminent
SNK01, a cryopreserved autologous non-genetically modified NK cell product, is able to effectively reduce a-synuclein (a-syn) protein levels in the cerebral spinal fluid (CSF) of Alzheimer’s patients; an important finding since increased a-syn has been correlated with worse cognitive performance and is not a target for any currently approved Alzheimer’s treatments
SNK01 treatment appears to stabilize or improve cognitive function and reduce a-synuclein levels in CSF in addition to improving previously reported amyloid, tau, and neuroinflammatory markers (GFAP, NfL, YKL-40) in the majority of patients
SANTA ANA, Calif., July 30, 2024 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (Nasdaq: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous, allogeneic and CAR-NK natural killer (“NK”) cell therapeutics, today announced the presentation of a poster entitled, “Subjects treated with expanded non-genetically modified autologous Natural Killer cells (SNK01) show improved changes in CSF a-synuclein and in cognitive function.” SNK01 is a first-in-kind, autologous non-genetically modified NK cell product with significantly increased cytotoxicity and over 90% activating receptor expression that can be consistently produced from any donor. Clinical trial data on the effect of SNK01 a-synuclein and cognitive assessments in patients with Alzheimer’s Disease (“AD”) was presented at the 2024 Alzheimer’s Association International Conference (“AAIC”) held in Philadelphia, PA on July 30, 2024.
Elevated levels of a-syn in the CSF have been linked to cognitive impairment in AD patients and correlate with worse cognitive performance (Twohig and Nielson, 2019). NKGen’s in vitro data demonstrated that SNK01 had, similarly to microglial (HMC3) cells, the ability to internalize and degrade a-syn aggregates which further supports the rationale for its use for a-synucleinopathies. Following an initial Phase I biomarker analysis that showed SNK01 could improve CSF levels of amyloid, tau, and neuroinflammation (GFAP, YKL-40, NfL), a secondary analysis was performed looking at the effect of SNK01 specifically on a-syn protein levels in CSF. Despite 70% of patients in this trial being treated with relatively low doses of SNK01, 60% had a reduction in their CSF levels of a-synuclein. In addition, 90% of patients (median MMSE score of 14 at baseline) showed stable or improved cognitive function at 11 weeks using the ADCOMS score.
“The additional data from our Phase I Alzheimer’s trial shows that SNK01 has the ability to reduce CSF a-synuclein levels in Alzheimer’s patients, which is important because studies have shown that high levels of a-syn are correlated with disease progression and worsened cognitive function in AD,” said Dr. Paul Y. Song, MD, Chairman and CEO of NKGen. “To our knowledge, there is no treatment for Alzheimer’s disease on the market that currently targets this protein as well as amyloid and tau while also reducing neuroinflammation like SNK01 does. We are excited that this additional data appears to show that treatment with SNK01 can also target, and reduce, a-syn while improving cognitive function in Alzheimer’s patients. We are working diligently to expand our investigation of SNK01 in a larger Phase II trial with higher doses and a longer treatment duration. We are also looking to expand the use of SNK01 for other synucleinopathy-related neurodegenerative diseases.”
Data Highlights from the Poster Presentation:
Our in vitro studies have shown that SNK01 is able to effectively internalize and degrade a-synuclein aggregates.
Despite 70% of subjects being treated at relatively low doses of SNK01:
90% of all evaluable subjects had either stable or improved (±0.1) composite ADCOMS scores at week 11 (one-week after the final dose).
60% of subjects (6/10) had a decrease in CSF a-syn compared to baseline values.
At Week 11, the decreases in a-syn corresponded to stable/decrease in ADCOMS in 5/6 subjects and where data was available, the a-syn levels continued below baseline through Week 22. One additional subject had a decrease in CSF a-syn compared to baseline values at Week 22.
No treatment related adverse events were observed. SNK01 is well-tolerated by AD subjects with no dose-limiting toxicities observed at all doses tested.
SNK01 appears to stabilize or improve cognitive function in the majority of subjects, despite 70% of subjects being treated at relatively low doses.
SNK01 appears to reduce a-synuclein levels in CSF.
We hypothesize that SNK01 is safe, can cross the blood brain barrier (BBB) and can impact both cognition and protein aggregates in Alzheimer’s patients.
This data warrants further investigation in a larger Phase Il trial with higher doses and a longer treatment duration.
NKGN
exploding now
100% e plosion tmrw
SANTA ANA, Calif., July 18, 2024 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (Nasdaq: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous, allogeneic and CAR-NK natural killer (“NK”) cell therapeutics, today announced that it will present new biomarker data on SNK01 (autologous non-genetically modified NK cell therapy) in Alzheimer’s disease during a poster presentation at the upcoming Alzheimer’s Association International Conference (“AAIC”) to be held in Philadelphia, PA at the Pennsylvania Convention Center and online from July 28 – August 1, 2024.
NKGen will present data on SNK01’s ability to reduce a-synuclein in Alzheimer’s patients, which is significant given that a-synuclein has been shown to correlate with worse cognitive function in Alzheimer’s disease. Presentation details are as follows:
Poster Title: Subjects treated with expanded non-genetically modified autologous Natural Killer cells (SNK01) show improved changes in CSF a-synuclein and in cognitive function
Presenting Author: Paul Y. Song, M.D.
Session Type:
Session Title:
Session Time:
Poster Board Number:
Location: Poster
Drug Development: Human
Tuesday, July 30, 2024; 7:30 AM – 4:15 PM ET
NKGN
starting to move up in anticipation of significant new data for Alzheimer tmrw
NKGN
Huge data release for Alzheimer tmrw
Easy 100% explosion
NKGN
BREAKING OUT NOW
SIGNIFICANT ALZHEIMER DATA PRESENTATION ON NEXT TUESDAY
NKGN
Keep on running in anticipation of new significant data release for Alzheimer
ridiculous $30 mil mktcap for successful P1 Alzheimer completion bio stock
Fair mktcap $300 mil mktcap
NKGN
Ridiculous $30 mil mktcap for successful P1 Alzheimer stock
$2 coming this week and $4 upon huge new data release on 07/30
NKGN
breaking out now
New data for Alzheimer’s worth billion dollars
NKGN
breaking out in anticipation of huge Alzheimer data release
NKGN
Keep an eye on huge data release for Alzheimer on next Tuesday
NKGN
The significant expanded P1 data presentation on the way
Keep an eye on breaking out this week and next week
Poster Title: Subjects treated with expanded non-genetically modified autologous Natural Killer cells (SNK01) show improved changes in CSF a-synuclein and in cognitive function
Presenting Author: Paul Y. Song, M.D.
Session Type:
Session Title:
Session Time:
Poster Board Number:
Location: Poster
Drug Development: Human
Tuesday, July 30, 2024; 7:30 AM – 4:15 PM ET
NKGN
huge data presentation coming on 07/30
$30 mil mktcap does not make sense for P1 successful completion bio company
Watch what happen next week and data release on 07/30 then
Good luck
This is humongous news fir alzheimer
NKGen will present data on SNK01’s ability to reduce a-synuclein in Alzheimer’s patients, which is significant given that a-synuclein has been shown to correlate with worse cognitive function in Alzheimer’s disease. Presentation details are as follows:
Poster Title: Subjects treated with expanded non-genetically modified autologous Natural Killer cells (SNK01) show improved changes in CSF a-synuclein and in cognitive function