Verrica Pharmaceuticals reports positive data on its molluscum contagiosum drug

Verrica Pharmaceuticals presents results from Phase 3 clinical trials of VP-102

Verrica Pharmaceuticals reports positive data on its molluscum contagiosum drug, Stockwinners

Verrica Pharmaceuticals (VRCA) presented data from the company’s pivotal Phase 3 CAMP-1 and CAMP-2 trials of lead product candidate, VP-102, at the American Academy of Dermatology annual meeting being held in Washington, DC from March 1-5.

Both trials of VP-102 in patients with molluscum contagiosum successfully met their primary endpoints.

In each trial, a clinically and statistically significant proportion of patients treated with VP-102 demonstrated complete clearance of all treatable molluscum lesions in 12 weeks.

On average, molluscum can take approximately 13 months to resolve without treatment, and in some cases can remain unresolved for several years.

The two randomized, double-blind, multicenter, placebo-controlled trials evaluated the efficacy of dermal application of VP-102 compared to placebo in subjects with molluscum.

In total, the trials enrolled 528 subjects two years of age and older with molluscum at 31 centers in the U.S. Subjects were treated once every 21 days with topical solution of 0.7% cantharidin for up to four applications.

Complete clearance of molluscum lesions was evaluated by assessment of the number of lesions at study visits over 12 weeks. Results from CAMP-1 and CAMP-2 showed 46% and 54% of subjects treated with VP-102, respectively, achieved complete clearance of all treatable molluscum lesions at the end of the trials versus 18% and 13% of subjects in the placebo groups.

By Day 84, VP-102 treated subjects had a 69% and 83% mean reduction in the number of molluscum lesions, a pre-specified endpoint, in CAMP-1 and CAMP-2 respectively, compared to a 20% increase and a 19% reduction for subjects on placebo. VP-102 was well-tolerated in both trials, with no serious adverse events reported in VP-102 treated subjects.

The most frequently reported adverse events were application site reactions that are well-known, reversible side effects related to the mechanism of action of cantharidin, a blistering agent, which is the active ingredient in VP-102.

There were no treatment-related serious adverse events reported in CAMP-1 or CAMP-2. Verrica previously announced topline results from both trials on January 3, 2019.

Based on the positive results, the company plans to submit a New Drug Application for VP-102 in the second half of 2019. If approved, VP-102 would be the first FDA-approved treatment for molluscum contagiosum.


Molluscum contagiosum is a relatively common viral infection of the skin, mainly in children, Stockwinners.com

VRCA closed at $12.11.

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Novartis receives positive CHMP opinion for Kymriah

Novartis receives positive CHMP opinion for Kymriah

Novartis receives positive CHMP opinion for Kymriah, Stockwinners
Novartis receives positive CHMP opinion for Kymriah, Stockwinners

Novartis (NVS) announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion recommending approval of Kymriah – a novel one-time treatment that uses a patient’s own T cells to fight cancer.

The positive opinion includes two B-cell malignancies: B-cell acute lymphoblastic leukemia that is refractory, in relapse post-transplant or in second or later relapse in patients up to 25 years of age; and diffuse large B-cell lymphoma that is relapsed or refractory after two or more lines of systemic therapy in adults.

If approved by the European Commission, Kymriah will be the first CAR-T cell therapy available in the European Union for both DLBCL and B-cell ALL.

Both B-cell ALL and DLBCL are aggressive malignancies with significant treatment gaps for patients.

In Europe, ALL accounts for approximately 80% of leukemia cases among children, and for those patients who relapse, the outlook is poor. This low survival rate is in spite of patients having to undergo multiple treatments, including chemotherapy, radiation, targeted therapy or stem cell transplant, and further highlights the need for new treatment options.

DLBCL is the most common form of non-Hodgkin lymphoma, accounting for up to 40% of all cases globally.

For patients who relapse or don’t respond to initial therapy, there are limited treatment options that provide durable responses, and survival rates are low for the majority of patients due to ineligibility for autologous stem cell transplant or because salvage chemotherapy or ASCT have failed.

The positive CHMP opinion is based on two pivotal Novartis-sponsored global, multi-center, Phase II trials, ELIANA and JULIET, which included patients from Europe, the US, Australia, Canada and Japan. The collaboration of Novartis and the University of Pennsylvania has led to historic milestones in CAR-T cell therapy since 2012, including the initiation of the first global CAR-T trials, the PRIME designation granted by the EMA for Kymriah in pediatric patients with r/r B-cell ALL, and the approval of Kymriah in two distinct indications by the US Food and Drug Administration.

ELIANA is the first pediatric global CAR-T cell therapy registration trial, treating patients in 25 centers in the US, Canada, Australia, Japan and the EU, including: Austria, Belgium, France, Germany, Italy, Norway and Spain. JULIET is the first multi-center global registration study for Kymriah in adult patients with r/r DLBCL.

JULIET is also the largest global study evaluating a CAR-T cell therapy in patients with DLBCL, enrolling patients from 27 sites in 10 countries across the US, Canada, Australia, Japan and the EU, including: Austria, France, Germany, Italy, Norway and the Netherlands.

The Novartis CAR-T cell manufacturing platform includes cryopreservation, the process of freezing patients’ harvested cells in order to preserve them, which provides physicians with the flexibility to decide when to initiate both the harvesting of patients’ cells and the infusion of Kymriah, based on each patient’s condition, and allows for this individualized treatment approach on a global scale.

The European Commission will now review the CHMP recommendation to deliver its final decision, applicable to all 28 EU member states, plus Iceland, Liechtenstein and Norway.

NVS closed at $73.00, it last traded at $75.50.


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Heron Therapeutics HTX-001 achieves primary endpoints

Heron Therapeutics HTX-001 achieves primary endpoints in Study 209, 211

Heron Therapeutics HTX-001 achieves primary endpoints, Stockwinners
Heron Therapeutics HTX-001 achieves primary endpoints, Stockwinners

Heron Therapeutics (HRTX) announced positive topline results from two completed Phase 2b studies of HTX-011: Study 209 and Study 211.
HTX-011 achieved the primary endpoints in both studies.
Study 209 was a randomized, placebo- and active-controlled, double-blind, Phase 2b clinical study in patients undergoing primary unilateral total knee arthroplasty to evaluate the analgesic efficacy, safety and pharmacokinetics of HTX-011 locally administered into the surgical site.
Following a dose-escalation phase, 222 patients were randomized to receive: HTX-011 400 mg administered via instillation into the surgical site; HTX-011 400 mg administered via instillation into the surgical site with a low dose of ropivacaine injected into the posterior capsule (HTX-011 combination); bupivacaine 125 mg administered via multiple injections into the surgical site; and placebo.
Ropivacaine and bupivacaine are generically available standard-of-care local anesthetics used in the management of postoperative pain.
This study included a pre-specified hierarchical testing strategy for the primary and key secondary endpoints for the HTX-011 400 mg treatment groups. The primary endpoint was pain intensity as measured by the area under the curve, or AUC, from 0 to 48 hours post-surgery for HTX-011 compared to placebo.
The key secondary endpoint was pain intensity as measured by the AUC from 0 to 72 hours post-surgery for HTX-011 compared to placebo.
The primary and key secondary endpoints were achieved.
Study 211 was a randomized, placebo- and active-controlled, double-blind, Phase 2b dose-finding study in patients undergoing augmentation mammoplasty to evaluate the analgesic efficacy, safety and pharmacokinetics of HTX-011 when administered by instillation into the surgical site or via ultrasound-guided lateral and medial pectoral nerve block before surgery.
The study consisted of three cohorts comparing HTX-011 nerve block to the standard dose of bupivacaine 50 mg, administered as a nerve block, and placebo, and a final cohort comparing both HTX-011 400 mg administered by instillation and HTX-011 400 mg administered as a nerve block to the same two control groups.
A total of 243 patients were enrolled.
The primary endpoint was pain intensity as measured by the AUC from 0 to 24 hours post-surgery compared to placebo. The primary endpoint of the study was achieved.
HRTX closed at $30.70. it last traded at $40.00


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Bluebird Bio reports data from Phase 1 HGB-206

Bluebird Bio reports interim data from Phase 1 HGB-206 study of LentiGlobin

https://stockwinners.com/
Bluebird Bio reports interim data from Phase 1 HGB-206 study of LentiGlobin

bluebird bio (BLUE) announced new interim data from the ongoing HGB-206 Phase 1 multicenter clinical study of LentiGlobin investigational gene therapy in patients with severe sickle cell disease will be presented in an oral presentation on Saturday, June 16 at the 23rd Congress of the European Hematology Association by Julie Kanter, M.D., Medical University of South Carolina, Charleston, South Carolina.

“The consistent production of increased amounts of anti-sickling HbAT87Q in the Group C patients reflects the substantial positive impact of the changes introduced with the amended HGB-206 study protocol and refined manufacturing process.

All four Group C patients with greater than or equal to three months follow-up are making over 30 percent anti-sickling HbAT87Q.

The first patient treated, now with six months of follow-up, is producing over 60 percent anti-sickling HbAT87Q with a normal total hemoglobin level of 14.2 g/dL,” said David Davidson, M.D., chief medical officer, bluebird bio.

“The upward trajectory in Group C at these early time points suggests the potential for these patients to exceed the initially proposed therapeutic target of 30 percent anti-sickling HbAT87Q. We continue to define the development plan with regulatory authorities, and with further follow-up, we hope to see even higher levels of HbAT87Q, as well as sustained clinical benefit for patients.”

“The early data from Group C patients are very exciting and provide increasing confidence that LentiGlobin has the potential to deliver transformative benefit to patients. The longer-term data from patients treated earlier in the study show that levels of anti-sickling HbAT87Q in patients with SCD treated with LentiGlobin remain stable for at least two years,” said Dr. Kanter, a lead investigator of the HGB-206 study.

“Treatment options that can address the underlying cause of sickle cell disease are limited and LentiGlobin gene therapy has the potential to prevent or substantially reduce damaging symptoms associated with this debilitating disease.”

Separately, bluebird bio announced that new data from the completed Phase 1/2 Northstar (HGB-204) study in adolescents and adults with transfusion-dependent beta-thalassemia and any genotype, and its ongoing, Phase 3 Northstar-2 (HGB-207) multicenter clinical study of LentiGlobin investigational gene therapy in patients with TDT and non-beta0/beta0 genotypes, will be presented in an oral session on June 16 at the 23rd Annual Congress of the European Hematology Association by Franco Locatelli, M.D., Ph.D., of the IRCCS Ospedale Pediatrico Bambino Gesu of Rome, Italy.

“The maturing data from HGB-204 and HGB-207 suggest that one-time treatment with LentiGlobin may address the underlying genetic cause of TDT.

With our refined manufacturing process, the majority of patients with TDT and non-beta0/beta0 genotypes are transfusion-free and producing total hemoglobin at normal or near-normal levels,” said David Davidson, M.D., chief medical officer, bluebird bio.

“We are on track to submit a marketing authorization application in the European Union later this year, and we continue to work closely with clinical investigators and regulatory authorities to complete our ongoing clinical trials and bring this important treatment option to patients as soon as possible.”

“Consistently higher in vivo vector copy numbers and HbAT87Q hemoglobin levels in patients indicate that LentiGlobin manufacturing refinements have resulted in improved gene therapy characteristics and may enable sustained transfusion independence for a great majority of patients,” said Professor Locatelli, the lead investigator of the Northstar-2 study.

“Further, we are now seeing more than three years of data from the Northstar study indicating that LentiGlobin therapy may enable long-term transfusion independence in the majority of patients with non-beta0/beta0 genotypes.

These results hold the promise to change the natural history of many patients with this severe genetic disorder of hemoglobin production.”

BLUE closed at $197.00. It last traded at $204.65.


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Ultragenyx reports ‘positive’ topline results for ornithine transcarbamylase deficiency.

Ultragenyx reports ‘positive’ topline results from Phase 1/2 DTX301 study

Ultragenyx announces FDA approval of MEPSEVII. See Stockwinners.com for details
Ultragenyx reports ‘positive’ topline results from Phase 1/2 DTX301 study

Ultragenyx Pharmaceutical (RARE) announced positive longer-term safety and efficacy data from the first dose cohort of the Phase 1/2 study of DTX301, an investigational adeno-associated virus gene therapy for the treatment of ornithine transcarbamylase deficiency.

The 52-week study is designed to enroll patients with late-onset disease who are clinically stable and on a stable dose of alternate pathway medication.

All three patients in the first, lowest-dose cohort received a single DTX301 dose of 2.0 10^12 GC/kg, and the pre-defined endpoint for efficacy evaluation occurred 12 weeks after dosing.

As of the February 15, 2018 data cutoff date, patient 1 has been followed for 24 weeks, patient 2 for 20 weeks, and patient three for 12 weeks The first patient’s rate of ureagenesis was normalized, maintained and then substantially increased over 24 weeks.

The rate of ureagenesis at baseline was 67% of normal, with the normal rate of ureagenesis defined as 300 umol/kg/hr. The patient had an initial peak effect at 6 weeks at 112% of normal, and then declined at week 12 to 87% of normal during the steroid regimen that was used to treat the patient’s mild ALT elevations.

After steroids were weaned, ureagenesis began to rebound to 91% of normal at week 20 and then substantially increased to 134% of normal at week 24. The protocol allows for the tapering or discontinuation of alternate pathway medications and all alternate pathway medications were stopped at week 24 for patient 1 based on their choice. In the 3 weeks since stopping these medications, the patient has been doing well clinically as reported by the investigator.

The second and third patients did not show a clinically meaningful change in rate of ureagenesis over 20 weeks and 12 weeks, respectively.

As of February 15, 2018 there have been no infusion-related adverse events and no serious adverse events reported. All adverse events have been Grade 1 or 2 and have resolved.

The only treatment-related adverse events were the previously-reported mild, clinically asymptomatic and manageable elevations in alanine aminotransferase in two patients, peaking at 45 and 118 IU/L.These ALT elevations were mild and similar to what has been observed in other programs using AAV gene therapy.

Both patients completed a standard tapering course of corticosteroids as outpatients to treat the ALT elevations and their ALT levels have remained in the normal range since completing the tapering course. The third patient’s ALT levels remained in the normal range through twelve weeks. All three patients have remained clinically and metabolically stable.

The Data Monitoring Committee has completed its review of the current Cohort 1 data, and Ultragenyx will proceed to the second, higher-dose cohort of the study. Three patients will be enrolled in Cohort 2 and will each receive a single DTX301 dose of 6.0 10^12 GC/kg.

The first patient is expected to be enrolled in March 2018, and data from the second cohort are expected in the second half of 2018.

RARE closed at $52.66. It last traded at $54.00


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GTx Phase 2 trial of enobosarm meets primary endpoint

GTx Phase 2 trial of enobosarm meets primary endpoint

 

GTx Phase 2 trial of enobosarm meets primary endpoint. Stockwinners.com
GTx Phase 2 trial of enobosarm meets primary endpoint

GTx  (GTXI) announced additional results from a Phase 2 proof-of-concept clinical trial of 3 mg enobosarm administered orally in postmenopausal women with stress urinary incontinence, or SUI, including magnetic resonance imaging, or MRI results from patients’ pelvic floor muscle.

New data in a subset of women also suggests a positive treatment effect of enobosarm for urge incontinence, or UI, suggesting a possible treatment effect for women with mixed incontinence.

Results from a pre-specified analysis of MRI data demonstrate a statistically significant increase in pelvic floor muscle thickness and urethral muscle diameter after enobosarm treatment.

Treatment with enobosarm also reduced mean UI episodes by approximately 68 percent in patients who experienced UI as well as SUI, based on a post hoc analysis of a subset of women with both UI and SUI.

These results were outlined during a podium presentation which took place at the Society of Urodynamics, Female Pelvic Medicine, & Urogenital Reconstruction.

The presentation included clinical data from all 18 patients completing 12 weeks of enobosarm treatment, which, as previously reported, demonstrated an 81% reduction in the number of mean stress leaks per day, the primary endpoint of the clinical trial, as well as additional data demonstrating duration of response following completion of treatment, including nine patients who have now reached seven months post-treatment.

MRI was used to quantitatively measure muscle in the pelvic floor of 17 women at 12 weeks compared to their baseline. The results showed a statistically significant increase in several important measurements and support the mechanism of action of enobosarm on the pelvic floor.

While all of the women in the trial had predominant SUI, some also experienced urge incontinence.

Eleven of the 18 women completing 12 weeks of treatment were determined to have both SUI and UI at baseline, and these 11 women with mixed incontinence demonstrated a mean reduction in their UI episodes of approximately 68%.

Consistent with previous findings, at the end of the 12-week treatment period, all of the 18 enobosarm-treated women showed a clinically meaningful reduction in stress urinary incontinence episodes per day.

The reduction in incontinence episodes was sustained, or durable, well beyond the 12-week treatment period.

There were no serious adverse events reported and reported adverse events were minimal and included headaches, nausea, fatigue, hot flashes, insomnia, muscle weakness and acne.

Mild transient elevations in liver enzymes that were within normal limits were observed, except for one patient with levels greater than 1.5 times the upper limit of normal which returned to normal following her 12-week treatment period. Reductions in total cholesterol, LDL-C, HDL-C and triglycerides were also observed.

GTXI closed at $16.81. It last traded at $19.00 in pre-market trading.


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Pieris Pharmaceuticals higher on collaboration with Seattle Genetics

Pieris Pharmaceuticals, Seattle Genetics enter collaboration, license agreement

Pieris Pharmaceuticals higher on collaboration with Seattle Genetics
Pieris Pharmaceuticals higher on collaboration with Seattle Genetics

Pieris Pharmaceuticals (PIRS) and Seattle Genetics (SGEN) announced they have entered into a collaboration and license agreement with the goal of developing multiple targeted bispecific immuno-oncology treatments for solid tumors and blood cancers.

The collaboration leverages the expertise and core technologies of both companies to develop novel Antibody-Anticalin fusion proteins.

Under the terms of the agreement, Seattle Genetics will pay Pieris a $30M upfront fee, tiered royalties on net sales up to low double-digits, and up to $1.2B in total success-based payments across three product candidates.

The companies will pursue multiple Antibody-Anticalin fusion proteins during the research phase, and Seattle Genetics has the option to select up to three therapeutic programs for further development.

Prior to the initiation of a pivotal trial, Pieris may opt into global co-development and US commercialization of the second program and share in global costs and profits on a 50/50 basis.

Seattle Genetics will solely develop, fund and commercialize the other two programs.

PIRS closed at $7.14. It last traded at $8.32.


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Biogen to acquire KPT-350 from Karyopharm

Karyopharm enters agreement for Biogen to acquire KPT-350

Karyopharm enters agreement for Biogen to acquire KPT-350, Stockwinners.com
Karyopharm enters agreement for Biogen to acquire KPT-350

Karyopharm Therapeutics (KPTI) announced its entry into an agreement for Biogen (BIIB) to acquire Karyopharm’s investigational oral SINE compound KPT-350 and other assets for the treatment of certain neurological and neurodegenerative conditions.

KPT-350 is a novel therapeutic candidate that works by inhibiting XPO1, resulting in reductions in inflammation and neurotoxicity, as well as increasing neuroprotective responses.

Under the terms of the agreement, Biogen is acquiring KPT-350 and other assets targeting certain neurological conditions, including amyotrophic lateral sclerosis.

In exchange, Karyopharm will receive a one-time upfront payment of $10M from Biogen and is eligible to receive additional payments of up to $207M based on the achievement by Biogen of future specified development and commercial milestones.

Karyopharm will also be eligible to receive tiered royalty payments from Biogen that reach low double digits based on future net sales of specified product candidates, including KPT-350.

KPTI closed at $10.45.


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Pluristem Therapeutics says its PLX cells ‘significantly inhibit’ cancer cell growth

Pluristem PLX cells ‘significantly inhibit’ cancer cell growth in new study

Pluristem PLX cells 'significantly inhibit' cancer cell growth in new study. Stockwinners.com
Pluristem PLX cells ‘significantly inhibit’ cancer cell growth in new study

Pluristem Therapeutics (PSTI) announced the publication of a peer-reviewed article in the journal Scientific Reports, from the publisher of Nature, titled, “Human Placental-Derived Adherent Stromal Cells Co-Induced with TNF-alpha and IFN-gamma Inhibit Triple-Negative Breast Cancer in Nude Mouse Xenograft Models.”

The article is based on studies which examined the effect of PLX cells that had been induced with tumor necrosis factor alpha and interferon-gamma, on the proliferation of over 50 lines of human cancerous cells.

The induction of the cells was carried out by adjusting their manufacturing process in order to transiently alter their secretion profile.

Data from the first study showed that the modified PLX cells exhibited an anti-proliferative effect on 45% of the tested cancer cell lines, with a strong inhibitory effect on various lines of breast, colorectal, kidney, liver, lung, muscle and skin cancers.

Comprehensive bioinformatics analysis identified common characteristics of the cancer cell lines inhibited by PLX cells.

This knowledge could potentially be used in the future for screening patients’ tumors to identify those patients most likely to show a positive response to treatment with PLX cells.

Based on these promising results, Pluristem conducted a pre-clinical study of female mice harboring human triple negative breast cancer, or TNBC.

TNBC is an aggressive form of breast cancer that does not respond to standard hormonal therapy due to a lack of estrogen and progesterone receptors. Current treatment for TNBC consists of a combination of surgery, radiation therapy, and chemotherapy, and yet the prognosis remains poor for patients with this type of breast cancer.

In this study, weekly intramuscular (IM) injections of the induced PLX cells produced a statistically significant reduction in mean tumor size in the treated group compared with the untreated group, with 30% of the treated mice exhibiting complete tumor remission.

In addition, a statistically significant reduction was seen in the percentage of proliferating tumor cells as well as in the level of blood vessels within the tumors.

Pluristem has filed patent applications relating to the technology for the induction of PLX cells and the use of these cells for the treatment of cancer.

PSTI  last traded at $1.62.


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Celgene is acquiring Impact Biomedicines for $7B

Celgene to acquire Impact Biomedicines, adds Fedratinib to pipeline

Celgene tumbles
Celgene to acquire Impact Biomedicines, adds Fedratinib to pipeline

Celgene (CELG) and privately held Impact Biomedicines announced the signing of a definitive agreement in which Celgene will acquire Impact Biomedicines, which is developing fedratinib for myelofibrosis and polycythemia vera.

Under the terms of the agreement, Celgene will pay approximately $1.1B upfront and up to $1.25B in contingent payments based on regulatory approval milestones for myelofibrosis.

Additional future payments for regulatory approvals in additional indications and sales-based milestones are also possible.

#Fedratinib, a highly selective JAK2 kinase inhibitor, was evaluated in 877 patients across 18 clinical trials.

Celgene to acquire Impact Biomedicines, adds Fedratinib to pipeline, Stockwinners.com
Celgene to acquire Impact Biomedicines, adds Fedratinib to pipeline

In a randomized, placebo-controlled, phase III pivotal trial for patients with treatment-naive myelofibrosis, fedratinib demonstrated statistically significant improvements in the primary and secondary endpoints of splenic response and total symptom score, respectively.

In an exploratory subgroup analysis, these improvements were observed regardless of a patient’s baseline platelet count. A multi-center, single-arm phase II trial evaluated fedratinib in myelofibrosis patients who were found to be resistant or intolerant to ruxolitinib, a JAK1/JAK2 inhibitor. In this second-line setting, fedratinib demonstrated clinically meaningful improvements in splenic response and total symptom score.

#JAKARTA-2 was stopped prematurely due to a clinical hold placed on the fedratinib program by the U.S. Food and Drug Administration after potential cases of Wernicke’s encephalopathy were reported in eight out of 877 patients receiving one or more doses. The FDA removed the clinical hold in August 2017.

Based on the reported benefit risk profile of fedratinib from the JAKARTA-1 and JAKARTA-2 clinical trials, regulatory applications in #myelofibrosis are planned beginning in the middle of 2018.

Myelofibrosis is a serious bone marrow disorder that disrupts body’s normal production of blood cells. The result is extensive scarring in one’s bone marrow, leading to severe anemia, weakness, fatigue and often an enlarged spleen.

Myelofibrosis is an uncommon type of chronic #leukemia — a cancer that affects the blood-forming tissues in the body. Myelofibrosis belongs to a group of diseases called myeloproliferative disorders.

 Many people with myelofibrosis get progressively worse, and some may eventually develop a more serious form of leukemia. Yet it’s also possible to have myelofibrosis and live symptom-free for years.
CELG closed at $104.99.


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Cellect Biotechnology reports stem cell breakthrough

Cellect Biotechnology announces successful transplant using ApoGraft

Cellect Biotechnology announces successful transplant using ApoGraft. Stockwinners.com
Cellect Biotechnology announces successful transplant using ApoGraft

Cellect Biotechnology (APOP) announced that it has successfully completed transplantation of the first group of three patients using Cellect’s ApoGraft technology in the company’s Phase I/II clinical trial and that after one month follow-up, all three patients have demonstrated complete acceptance of the stem cell transplant with no adverse events related to the study treatment, as determined by the clinical investigator, and no reported serious adverse events or suspected unexpected serious adverse reactions.

The Phase I/II, dose escalating, 4-cohort, open label clinical trial of up to twelve patients is designed to evaluate the safety, tolerability and efficacy of functionally selected donor derived mobilized peripheral blood cells that underwent the company’s ApoGraft process and were transplanted into patients with hematological malignancies in an allogeneic hematopoietic stem cell transplantation.

The primary endpoint of the study is overall incidence, frequency and severity of adverse events potentially related to ApoGraft at 180 days from transplantation. The company plans on recruiting a further three patients for the second cohort of patients following review of the independent data and safety monitoring board.

The company believes that these interim results of ApoGraft present the first signs of a breakthrough in stem cell transplantation.

The product is transplantable within less than 12 hours from donation through a simple process performed on the bedside after selective physiological elimination of immune reaction-causing cells.

The ApoGraft transplantation is intended to result in complete recovery of the patient’s immune system with no related safety concerns in contrast to the significant morbidity or even death causing standard medical procedure.

APOP closed at $7.12. It traded at $9.69 in pre-market trading.


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