Watch Shares of Adverum Biotechnologies!

Adverum Biotechnologies reports additional data from Phase 1 trial of ADVM-022

Adverum Biotechnologies (ADVM) announced additional clinical data for the first cohort of patients in the ongoing OPTIC phase 1 clinical trial of ADVM-022, the company’s intravitreal injection gene therapy, in treatment-experienced patients with wet age-related macular degeneration.

Adverum Biotechnologies reports additional data from Phase 1 trial of ADVM-022 in patients with wet age-related macular degeneration, Stockwinners

Adverum also announced enrollment plans for the third and fourth cohorts in the ongoing OPTIC trial.

The third cohort has been initiated and patients will be treated with ADVM-022 at a dose of 2 x 10^11 vg/eye, the same dose used in the second cohort.

Subsequently, patients in the fourth cohort will be treated with ADVM-022 at a dose of 6×10^11 vg/eye, the same dose used in the first cohort.

Since inflammation has generally been mild and responsive to steroid eye drops, patients in the third and fourth cohorts will receive prophylactic steroid eye drops instead of prophylactic oral steroids.

Previously, on September 12, 2019, Adverum presented data from the first cohort in the ongoing OPTIC trial at a pre-specified 24-week time point.

Wet and Dry Macular Degeneration, Stockwinners

The additional data for this cohort includes key outcomes with a median follow-up of 34 weeks.

In treatment-experienced patients previously requiring frequent anti-VEGF injections to maintain vision, the data continue to demonstrate that the single ADVM-022 injection in this cohort was sufficient to maintain vision, with zero rescue injections required for any of the six patients.

Aaron Osborne, MBBS, chief medical officer of Adverum, added, “The clinical profile of ADVM-022 demonstrates this gene therapy’s potential to be a significant advance for patients with wet AMD. It is very encouraging that there continues to be zero rescue injections in this cohort of treatment-experienced patients with more than 6 months follow-up on all patients. We are expanding the development of ADVM-022 and are excited to share that enrollment is now open for the third cohort in OPTIC.

We look forward to being able to deliver this novel intravitreal gene therapy candidate as soon as possible to patients with wet AMD and diabetic retinopathy, our second indication for ADVM-022. We are grateful for all of the investigators, patients, and caregivers who continue to participate in the OPTIC trial.”

Adverum plans to begin dosing patients in the third cohort of the OPTIC trial in the fourth quarter of 2019 and plans to begin enrollment in the fourth cohort in the first quarter of 2020.

Adverum plans to present 52-week data from the first cohort of patients in the OPTIC trial as well as 24-week data from the second cohort of patients in the first half of 2020.

Adverum plans to submit an investigational new drug application for the treatment of ADVM-022 in diabetic retinopathy in the first half of 2020.

Piper Jaffray

Commenting on Adverum Biotechnologies update at AAO, Piper Jaffray analyst Tyler Van Buren notes that zero rescue injections have been required and disease activity by OCT and visual acuity has been maintained with 6 ADVM-022 patients now out to a median of 34 weeks.

Importantly, despite the intense criticism over inflammation and visual acuity fluctuations during the initial data disclosure last month, numerous KOLs at the conference were unconcerned and believe that ADVM-022 looks like a potential treatment option so far, he contends.

Overall, the analyst argues that safety continues to be clean in these initial patients and looks forward to the 52-week update in first half of 2020.

Van Buren reiterates an Overweight rating and $14 price target on the shares as ADVM-022 continues to look like a viable gene therapy candidate for wet AMD, which has $1B-plus potential.

ADVM closed at $5.29.

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Sarepta reports positive results for muscular dystrophy Type 2E treatment

Sarepta announces results from gene therapy trial for beta sarcoglycanopathy

Sarepta Therapeutics (SPRT) announced the nine-month functional results from three Limb-girdle muscular dystrophy Type 2E ,LGMD2E, clinical trial participants who received SRP-9003. SRP-9003 is an investigational gene therapy intended to transduce skeletal and cardiac muscle with a gene that codes for the full-length, native beta-sarcoglycan protein, the lack of which is the sole cause of LGMD2E.

Sarepta drops after U.K. trial halt,
Sarepta rises on muscular dystrophy data, Stockwinners

In Cohort 1 of the SRP-9003 study, three participants ages 4-13 were treated with an infusion of SRP-9003 at a dose of 5x1013vg/kg.

Improvements in functional outcomes were observed at day 270 for all three participants.

“We have now observed consistent functional improvements, in addition to high levels of expression of the missing protein of interest and strong results in related biomarkers, in both of our first cohorts for Duchenne muscular dystrophy and LGMD2E. We intend to test one higher dose of SRP-9003 in LGMD2E participants, select our clinical dose and then advance our SRP-9003 program, along with our other five LGMD programs, as rapidly as possible,” said Doug Ingram, Sarepta’s president and chief executive officer.

“With the results of our first LGMD2E cohort, Sarepta continues to build its gene therapy engine, an enduring model created to design, develop and bring to the medical and patient community transformative therapies for those living with, and too often dying from, rare genetic disease.”

No new safety signals were observed and the safety profile seen to date supports the ability to dose escalate in the next cohort of the study. As previously disclosed, two participants in the study had elevated liver enzymes, one of which was designated a serious adverse event (SAE), as the participant had associated transient increase in bilirubin. Both events occurred when the participants were tapered off oral steroids and, in both instances, elevated liver enzymes returned to baseline and symptoms resolved following supplemental steroid treatment.

SPRT is up $5.52 to $86.86.

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AstraZeneca and Merck report prostate cancer data

AstraZeneca, Merck presents results from Phase 3 PROfound trial of LYNPARZA

AstraZeneca (AZN) and Merck (MRK) presented detailed results from the Phase 3 PROfound trial in 387 men with metastatic castration-resistant prostate cancer who have a mutation in their homologous recombination repair genes and whose disease had progressed on prior treatment with new hormonal agent treatments e.g. abiraterone or enzalutamide.

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AstraZeneca and Merck report prostate cancer, Stockwinners

The trial was designed to analyze men with mCRPC harboring HRR-mutated genes in two cohorts: the primary endpoint was in those with mutations in BRCA1/2 or ATM genes and then, if LYNPARZA showed clinical benefit, a formal analysis was performed of the overall trial population of men with HRRm genes.

Merck presents results from Phase 3 KEYNOTE-426 study, Stockwinners
AstraZeneca and Merck report prostate cancer , Stockwinners

Results showed a statistically-significant and clinically-meaningful improvement with LYNPARZA in the primary endpoint of radiographic progression-free survival in BRCA1/2 or ATM-mutated tumors reducing the risk of disease progression or death by a median of 7.4 months versus 3.6 months for those receiving abiraterone or enzalutamide.

LYNPARZA reduced the risk of disease progression or death by 66% for these men. The trial also met the key secondary endpoint of rPFS in the overall HRRm population, where LYNPARZA reduced the risk of disease progression or death by 51% and improved rPFS to a median of 5.8 months vs. 3.5 months for those receiving abiraterone or enzalutamide.

In the key secondary endpoint of time to pain progression, median TTPP was not reached with LYNPARZA and was 9.92 months with abiraterone and enzalutamide in patients with BRCA1/2 or ATM mutations.

Results also showed a trend at this interim analysis time point for improvement in overall survival, another key secondary endpoint. LYNPARZA extended OS to a median of 18.5 months versus 15.1 months for abiraterone or enzalutamide in men with BRCA1/2 or ATM-mutated tumors, of which 81% started on abiraterone or enzalutamide and, following confirmed disease progression, then switched to LYNPARZA.

At this interim analysis, the OS endpoint did not meet statistical significance. In an exploratory analysis, a similar trend in OS was observed at this interim analysis in the HRRm population with a median of 17.5 months for men treated with LYNPARZA vs. 14.3 months for those receiving abiraterone or enzalutamide.

The trial showed a confirmed overall response rate a key secondary endpoint of 33.3% for LYNPARZA vs. 2.3% for abiraterone or enzalutamide in patients with BRCA1/2 or ATM mutations.

In an exploratory analysis of patients in the overall HRRm population, confirmed ORR was 21.7 % for LYNPARZA vs. 4.5% for patients receiving abiraterone or enzalutamide. The safety and tolerability profile of LYNPARZA in the PROfound trial was in line with that observed in prior clinical trials.

The most common adverse events greater than or equal to20% for LYNPARZA compared to abiraterone or enzalutamide were anemia, nausea, fatigue and asthenia, decreased appetite, and diarrhea. Grade 3 or above AEs were anemia, fatigue and asthenia, vomiting, dyspnea, urinary tract infection, nausea, decreased appetite, diarrhea, and back pain. AEs led to discontinuation of treatment in 16% of patients on LYNPARZA vs. 9% on abiraterone and enzalutamide.

AstraZeneca and Merck are also exploring additional trials in prostate cancer, including the ongoing Phase 3 PROpel trial, evaluating LYNPARZA as a first-line therapy in mCRPC for patients with or without HRR mutations, in combination with abiraterone acetate.

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Cara Therapeutics shares jump on KALM-1 data

Cara Therapeutics announces topline data from KALM-1 Phase 3 trial

Cara reports positive top-line data from CR845, Stockwinners
Cara reports positive results from the KALM-1 pivotal Phase 3 trial of Korsuva Injection in hemodialysis patients , Stockwinners

Cara Therapeutics (CARA) announced topline data from the KALM-1 pivotal Phase 3 trial of Korsuva Injection in hemodialysis patients with moderate-to-severe chronic kidney disease-associated pruritus, or CKD-aP.

Chronic kidney disease-associated pruritus ( #CKD-aP ) is a distressing, often overlooked condition in patients with CKD and end-stage renal disease. It affects ~40% of patients with end-stage renal disease and has been associated with poor quality of life, poor sleep, depression, and mortality.  Despite being an annoyance, CKD-associated pruritus (CKD-aP) can adversely affect the quality of life (QOL) and medical outcomes. 

The proportion of patients on 0.5 mcg/kg of Korsuva Injection achieving a three-point or greater improvement from baseline in the weekly mean of the daily 24 hour Worst Itching Intensity Numeric Rating Scale, or WI-NRS, score at week 12 was 51% vs. 28% for patients on placebo.

The proportion of patients on 0.5 mcg/kg of Korsuva Injection achieving a four-point or greater improvement from baseline in the weekly mean of the daily 24 hour WI-NRS score at week 12 was 39% vs. 18% for patients on placebo.

Patients on #Korsuva Injection experienced a 43% improvement in the average total Skindex-10 score at week 12 vs. patients on placebo.

Patients on Korsuva Injection experienced a 35% improvement in the average total 5-D Itch score at week 12 vs. patients on placebo.

Korsuva was generally well-tolerated with a safety profile consistent with that seen in earlier Korsuva clinical trials.

Overall, the incidence of adverse events, or AEs, and serious AEs were similar across both Korsuva and placebo groups.

The most common treatment emergent AEs reported in greater than 5% of patients were diarrhea, dizziness, nasopharyngitis and vomiting.

CARA is up 12.5% to $20.21 per share.

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Intercept falls on NASH study

Intercept falls after releasing ‘supportive data’ from Phase 3 NASH study

Intercept Pharmaceuticals (ICPT) overnight announced additional “supportive data” from its Phase 3 Regenerate study of obeticholic acid in patients with liver fibrosis due to nonalcoholic steatohepatitis.

stockwinners.com ICPT

Intercept falls after releasing ‘supportive data’ from Phase 3 NASH study, Stockwinners

Nonalcoholic steatohepatitis (NASH) is liver inflammation and damage caused by a buildup of fat in the liver. It is part of a group of conditions called nonalcoholic fatty liver disease. You may be told you have a “fatty liver.”

The new data based on additional analyses show that obeticholic acid “demonstrated robust efficacy across a range of additional histologic and biochemical parameters,” Intercept said in a statement.

The data are being presented today at the International Liver Congress in Vienna, Austria.

The primary efficacy analysis assessed efficacy at 18 months in 931 patients with stage 2 or 3 liver fibrosis due to NASH.

Patients with biopsy proven NASH with fibrosis were randomized 1:1:1 to receive placebo, OCA 10 mg or OCA 25 mg once daily.

A repeat biopsy was conducted after 18 months for histologic endpoint assessment.

Overall, study discontinuations in the primary efficacy analysis population were balanced across treatment groups.

As previously reported, in the primary efficacy analysis, once-daily OCA 25 mg met the primary endpoint of fibrosis improvement with no worsening of NASH in 23.1% of patients compared to 11.9% of placebo patients at the planned 18-month interim analysis, the company said.

In the primary efficacy analysis, a numerically greater proportion of patients in both OCA treatment groups compared to placebo achieved the primary endpoint of NASH resolution with no worsening of liver fibrosis; however, this did not reach statistical significance.

As agreed with the FDA, in order for the primary objective to be met, the study was required to achieve one of the two primary endpoints, it added.

Additional supportive efficacy analyses were conducted using the per protocol population.

Approximately three-fold more patients in the OCA 25 mg group achieved an improvement of fibrosis by greater than or equal to 2 stages compared to placebo, Intercept reported.

ICPT is down 12%, or $14.14, to $106.54.

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Sorrento Therapeutics reports positive Parkinson’s disease study, shares rise

Sorrento Therapeutics announces top line results from Parkinson’s disease study


Sorrento Therapeutics announces top line results from Parkinson’s disease study, Stockwinners

Sorrento Therapeutics (SRNE) announced top line results in a discovery study aimed at exploring potential benefits of resiniferatoxin in controlling neuro-inflammatory processes associated with Parkinson’s disease in a rodent model.

Many clinical and pathological studies have shown that the disease extends beyond the substantia nigra and involves non-dopaminergic neurotransmitter systems that mediate both motor and non-motor symptoms.

Several therapeutic strategies have been proposed to treat such symptoms. However, despite the significant morbidity associated with these symptoms, research into and drug development for these problems remain scarce.

Transient receptor potential vanilloid 1, or TRPV1, is involved in pain perception and is highly expressed in sensory neurons. TRPV1 is also present in the brain where it may play a role in modulating neuronal function, controlling motor behavior and regulating neuroinflammation.

TRPV1 receptors are considered an important modulator of basal ganglia functions, and pharmacological changes to cells expressing those receptors might lead to protective therapies in Parkinson-induced motor symptoms.

Intrathecal administration of RTX at 7 days after AAV-A53T resulted in improved motor function analyzed by fine motor kinematic analysis when compared to control mice administered with vehicle for RTX.

No significant adverse effects after RTX infusion were observed. However, no significant effects in dopamine and its metabolites levels in striatum were observed.

The findings demonstrated activity of intrathecal administration of RTX at the early intervention paradigm to rescue motor deficits in AAV-A53T model.

The lack of effect on dopamine levels suggest that behavioral recovery is driven by mechanisms other than direct protection of nigrostriatal dopaminergic neurons, including modulation of neuroinflammation and/or analgesic effect of RTX after IT delivery.

A provisional patent has been filed for non-dopaminergic therapeutic approach with resiniferatoxin for symptomatic relief of motor symptoms associated with Parkinson’s disease.

The full results of the study will be released in publication in a scientific journal and presented at an upcoming neurosciences conference later this year.

Enabling preclinical work will continue with the goal of filing an IND by the end of the year. If results are confirmed, human clinical trials to start soon after. announces top line results from Parkinson’s disease study

Sorrento Therapeutics announced top line results in a discovery study aimed at exploring potential benefits of resiniferatoxin in controlling neuro-inflammatory processes associated with Parkinson’s disease in a rodent model.

Many clinical and pathological studies have shown that the disease extends beyond the substantia nigra and involves non-dopaminergic neurotransmitter systems that mediate both motor and non-motor symptoms.

Several therapeutic strategies have been proposed to treat such symptoms. However, despite the significant morbidity associated with these symptoms, research into and drug development for these problems remain scarce.

Transient receptor potential vanilloid 1, or TRPV1, is involved in pain perception and is highly expressed in sensory neurons.

TRPV1 is also present in the brain where it may play a role in modulating neuronal function, controlling motor behavior and regulating neuroinflammation. TRPV1 receptors are considered an important modulator of basal ganglia functions, and pharmacological changes to cells expressing those receptors might lead to protective therapies in Parkinson-induced motor symptoms.

Intrathecal administration of RTX at 7 days after AAV-A53T resulted in improved motor function analyzed by fine motor kinematic analysis when compared to control mice administered with vehicle for RTX.

No significant adverse effects after RTX infusion were observed. However, no significant effects in dopamine and its metabolites levels in striatum were observed.

The findings demonstrated activity of intrathecal administration of RTX at the early intervention paradigm to rescue motor deficits in AAV-A53T model.

The lack of effect on dopamine levels suggest that behavioral recovery is driven by mechanisms other than direct protection of nigrostriatal dopaminergic neurons, including modulation of neuroinflammation and/or analgesic effect of RTX after IT delivery.

A provisional patent has been filed for non-dopaminergic therapeutic approach with resiniferatoxin for symptomatic relief of motor symptoms associated with Parkinson’s disease. The full results of the study will be released in publication in a scientific journal and presented at an upcoming neurosciences conference later this year.

Enabling preclinical work will continue with the goal of filing an IND by the end of the year. If results are confirmed, human clinical trials to start soon after.

SRNE closed at $4.09.

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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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Spark Therapeutics sold for $4.8 billion

Roche acquires Spark Therapeutics for $114.50 per share, a 122% premium

Spark tumbles on hemophilia study , Stockwinners

Roche acquires Spark Therapeutics for $114.50 per share , Stockwinners

Spark Therapeutics (ONCE) announced that it has entered into a definitive merger agreement for Roche (RHHBY) to fully acquire Spark Therapeutics at a price of $114.50 per share in an all-cash transaction.

This corresponds to a total equity value of approximately $4.8B on a fully diluted basis, inclusive of approximately $500M of projected net cash expected at close.

The per share price represents a premium of 122% to Spark’s closing price on Feb. 22, 2019. The merger agreement has been unanimously approved by the boards of both Spark and Roche.

Under the terms of the merger agreement, Roche will commence a tender offer to acquire all outstanding shares of Spark’s common stock, and Spark will file a recommendation statement containing the unanimous recommendation of the Spark board that Spark shareholders tender their shares to Roche.

Spark Therapeutics will continue its operations in Philadelphia as an independent company within the Roche Group.

The closing of the transaction is expected to take place in Q2 of 2019.

Bernstein analyst Vincent #Chen notes that he has long seen Spark Therapeutics (ONCE) as a takeout candidate in gene therapy after the Wall Street Journal reported Roche (RHHBY) is near acquiring the company for close to $5B.

The analyst thinks the deal makes sense as adding hemophilia gene therapy to Hemlibra sets up Roche as a leader in next-gen non-factor hemophilia drugs, CNS gene therapy pipeline is a good fit for Roche’s growing neuroscience franchise, and as it serves as a cornerstone in establishing Roche as a gene therapy leader, to rival the likes of Novartis (NVS).


Credit Suisse analyst Martin Auster notes that the Wall Street Journal recently reported that Roche (RHHBY) is nearing a deal with Spark Therapeutics (ONCE) for $5B.

The analyst believes the transaction value implies the re-emergence of Spark’s hemophilia A gene therapy program with a competitive profile and substantial value assigned to additional pipeline programs, such as SPK-3006 for Pompe, and the technology platform.

Spark’s acquisition would further strengthen the company’s position in the hemophilia space, he contends. Further, Auster argues that the deal may increase M&A interest among other gene therapy names in his coverage universe, which include BioMarin (BMRN), Sarepta (SRPT), Ultragenyx (RARE), PTC (PTC) and Solid Biosciences (SLDB). He sees particularly strong read-through to Sarepta.


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Zosano reports positive migraine data, Shares jump

Zosano says Qtrypta long-term study shows ‘well-tolerated safety profile’

Zosano says Qtrypta long-term study shows 'well-tolerated safety profile', See Stockwinners for more

Zosano says Qtrypta long-term study shows ‘well-tolerated safety profile’ , Stockwinners

Zosano Pharma (ZSAN) announced earlier today the completion of the second and final goal of the long-term safety study for Qtrypta, in which patients treated migraine attacks over a one year period.

“The long-term data generated in this trial reinforced the well-tolerated safety profile and strong efficacy results previously reported in the six-month dosing portion of this safety study and in the randomized Phase 2/3 ZOTRIP pivotal study,” the company said in a statement.

Throughout the clinical program, over 5,800 migraine attacks have been treated with Qtrypta to date, it added.

The Qtrypta long-term safety trial is an open-label study evaluating the safety of the 3.8 mg dose of intracutaneous zolmitriptan in adults with migraine who have historically experienced at least two migraine attacks per month.

The study evaluated over 150 adults with migraine disease for six months, and more than 50 patients for a year at 31 sites in the U.S.

Of more than 5,800 migraines treated, investigators reported 832 adverse events, of which 298 were reported as application site reactions and 161 were reported as triptan related adverse events.

Observational efficacy parameters continued to demonstrate a rate of pain freedom at two hours following patch application of approximately 44% and most bothersome symptom freedom of approximately 68%, while pain relief at two hours was reported at 81% of migraine attacks treated, said Zosano.

The company expects to file an New Drug Application for Qtrypta in the fourth quarter of 2019.

ZSAN is up 95% to $4.41.

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Merck reports positive results of its renal cell carcinoma drug

Merck presents results from Phase 3 KEYNOTE-426 study

Merck presents results from Phase 3 KEYNOTE-426 study, Stockwinners
Merck presents results from Phase 3 KEYNOTE-426 study, Stockwinners

Merck (MRK) announced presentation of the full results from the pivotal Phase 3 KEYNOTE-426 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with Pfizer’s (PFE) Inlyta, a tyrosine kinase inhibitor, for the first-line treatment of advanced renal cell carcinoma at the 2019 Genitourinary Cancers Symposium.

Merck's anti-PD-1 therapy works in combination with Pfizer's (PFE) Inlyta, Stockwinners

Merck’s anti-PD-1 therapy works in combination with Pfizer’s (PFE) Inlyta

This is the first combination regimen to significantly improve overall survival, progression-free survival and objective response rate compared to sunitinib.

Results were consistent across all IMDC subgroups, including favorable, intermediate and poor risk groups, and regardless of PD-L1 expression.

As previously announced, the U.S. Food and Drug Administration has granted priority review for a supplemental Biologics License Application for #KEYTRUDA in combination with axitinib for the first-line treatment of patients with advanced RCC based on the results of KEYNOTE-426, and has set a Prescription Drug User Fee Act, or target action, date of June 20, 2019.

Findings from the first interim analysis showed KEYTRUDA in combination with axitinib reduced the risk of death by 47% – significantly improving OS compared to sunitinib.

For the dual primary endpoint of PFS, the KEYTRUDA combination showed a reduction in the risk of progression of disease or death of 31% compared to sunitinib.

In the study, the ORR was 59.3% for patients who received KEYTRUDA in combination with axitinib and 35.7% for those who received sunitinib, with a complete response rate of 5.8% and 1.9% and a partial response rate of 53.5% and 33.8%, for patients receiving the KEYTRUDA combination or sunitinib, respectively.

Median duration of response was not reached in the KEYTRUDA combination arm and was 15.2 months in the sunitinib arm. The results for OS, PFS and ORR were consistent across all IMDC risk groups and seen regardless of PD-L1 expression.

The observed adverse event profile was as expected based on the known profiles of KEYTRUDA and axitinib.

There was a higher incidence of grade 3 or 4 liver enzyme elevation with KEYTRUDA plus axitinib than previously observed with each agent as monotherapy.

Merck has filed these data with regulatory authorities worldwide.

Merck has an extensive clinical development program in RCC and is advancing multiple potential registration-enabling studies with KEYTRUDA, as monotherapy and in combination with other treatments, including KEYNOTE-564 and KEYNOTE-581.

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Sarepta gene therapy showed functional improvements

Sarepta gene therapy showed functional improvements across all measures 

Sarepta drops after U.K. trial halt,
Sarepta gene therapy showed functional improvements

Sarepta Therapeutics (SRPT) announced  at the 23rd International Congress of the World Muscle Society in Mendoza, Argentina, Jerry Mendell, M.D., of Nationwide Children’s Hospital presented updated results from its gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne muscular dystrophy.

Dr. Mendell presented the following updated data on the four patients enrolled in the study: All patients showed robust expression of transduced micro-dystrophin, which is properly localized to the muscle sarcolemma, as measured by immunohistochemistry.

Mean gene expression for the study, as measured by percentage of micro-dystrophin positive fibers was 81.2% and the mean intensity of the fibers was 96.0% compared to normal control.

All post-treatment biopsies showed robust levels of micro-dystrophin as measured by Western blot, with a mean of 74.3% compared to normal utilizing Sarepta’s method, or 95.8% compared to normal pursuant to Nationwide Children’s quantification of Sarepta’s method that adjusts for fat and fibrotic tissue.

Gene expression for the fourth patient was robust, as follows: As measured by immunohistochemistry, micro-dystrophin positive fibers was 96.2% and the mean intensity of the fibers was 160.0% compared to normal control. As measured by Western blot, patient 4 showed robust levels of micro-dystrophin, with a mean of 182.7% compared to normal utilizing Sarepta’s method, or 222% compared to normal pursuant to Nationwide Children’s quantification of Sarepta’s method that adjusts for fat and fibrotic tissue.

In all patients, expression of micro-dystrophin was associated with significant expression and up-regulation of the dystrophin-associated protein complex, an additional indication of functionality of dystrophin.

All patients showed significant decreases of serum creatine kinase levels at last measure, with a mean reduction of CK of over 78% from baseline.

Doug Ingram, Sarepta’s president and CEO, said, “The encouraging results that we previously saw and reinforced in the fourth patient strengthen our resolve to rapidly move to a confirming trial and, assuming successful, to bring this therapy to the Duchenne community around the world with a sense of urgency.”

SRPT closed at $147.38; it last traded at $152.50.


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Clovis Oncology is in focus

Clovis receives Breakthrough Therapy Designation for Rubraca

CLVS to submit NDA to FDA
Clovis is in focus

 

Clovis Oncology (CLVS) announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation for Rubraca as a monotherapy treatment of adult patients with BRCA1/2-mutated mCRPC who have received at least one prior androgen receptor-directed therapy and taxane-based chemotherapy.

Breakthrough Therapy designation is granted by the FDA to investigational agents intended to treat a serious or life-threatening disease or condition and whose preliminary clinical evidence may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy.

The FDA previously granted Breakthrough Therapy designation to Rubraca for the monotherapy treatment of certain advanced ovarian cancer patients and then in December 2016 approved Rubraca for the treatment of certain adult patients with deleterious BRCA mutation associated epithelial ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more chemotherapies.

The FDA subsequently approved Rubraca in a second indication, the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy, in April 2018.

This most recent Breakthrough Therapy designation was granted to Rubraca based on initial efficacy and safety results from TRITON2, the Phase 2 study of Rubraca in men with advanced prostate cancer with BRCA 1/2 mutations and deleterious mutations of other homologous recombination repair genes, in the metastatic castration-resistant setting.

JP Morgan Comments

JPMorgan analyst Cory Kasimov is encouraged by Clovis Oncology’s announcement this morning that rucaparib received breakthrough designation from the FDA for the third line treatment of patients with BRCA mutated metastatic castrate-resistant prostate cancer on the basis of data from the Triton-2 study.

The analyst says the news further increases his confidence in the potential for rucaparib to produce response rates that are meaningfully better than currently available options.

His model implies $9 per share for prostate, assuming a 55% probability of success and $450M in peak unadjusted sales. #Kasimov keeps an Overweight rating on Clovis.

CLVS closed at $29.14, it last traded at $31.50.


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Amarin sharply higher on data

Amarin soars after REDUCE-IT study meets primary endpoint

Amarin sharply higher on data, Stockwinners
Amarin sharply higher on data, Stockwinners

Amarin (AMRN) announced topline results from the Vascepa cardiovascular outcomes trial, REDUCE-IT, a global study of 8,179 statin-treated adults with elevated CV risk.

REDUCE-IT met its primary endpoint demonstrating an approximately 25% relative risk reduction, to a high degree of statistical significance, in major adverse CV events in the intent-to-treat patient population with use of Vascepa 4 grams/day as compared to placebo, Amarin said in a statement.

Patients enrolled in REDUCE-IT had LDL-C between 41-100 mg/dL controlled by statin therapy and various cardiovascular risk factors including persistent elevated triglycerides between 150-499 mg/dL and either established cardiovascular disease or diabetes mellitus and at least one other CV risk factor. Key topline results include approximately 25% relative risk reduction, demonstrated to a high degree of statistical significance, in the primary endpoint composite of the first occurrence of MACE, including cardiovascular death, nonfatal myocardial infarction, nonfatal stroke, coronary revascularization, or unstable angina requiring hospitalization.

This result was supported by robust demonstrations of efficacy across multiple secondary endpoints, the company said.

It added that Vascepa was well tolerated with a safety profile consistent with clinical experience associated with omega-3 fatty acids and current FDA-approved labeling.

The proportions of patients experiencing adverse events and serious adverse events in REDUCE-IT were similar between the active and placebo treatment groups.

Median follow-up time in REDUCE-IT was 4.9 years. Amarin said it is “eager to share REDUCE-IT data in greater detail with both the medical community and regulatory authorities.”

REDUCE-IT results have been accepted for presentation at the 2018 Scientific Sessions of the American Heart Association on November 10, 2018 in Chicago, Illinois.

“We are delighted with these topline study results,” said John Thero, president and CEO of Amarin.

“Given Vascepa is affordably priced, orally administered and has a favorable safety profile, REDUCE-IT results could lead to a new paradigm in treatment to further reduce the significant cardiovascular risk that remains in millions of patients with LDL-C controlled by statin therapy, as studied in REDUCE-IT.”

It notes, “As previously described, given the successful topline results of REDUCE-IT, Amarin is in the process of increasing the number of company sales representatives promoting Vascepa to over 400 people in the United States.”

Shares of Amarin (AMRN) closed at $2.99, it last traded at $12.30.


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Today’s stock disaster

GTx announces placebo-controlled ASTRID trial did not meet primary endpoint

GTx Phase 2 trial of enobosarm meets primary endpoint. Stockwinners.com
Today’s stock disaster

GTx (GTXI) announced that the ASTRID Trial, a Phase 2 double-blind, placebo-controlled clinical trial of orally-administered enobosarm in post-menopausal women with stress urinary incontinence, did not achieve statistical significance on the primary endpoint of the proportion of patients with a greater than 50% reduction in incontinence episodes per day compared to placebo.

The percentage of patients with a greater than 50% reduction after 12 weeks of enobosarm treatment was 58.9% for 3mg, 57.7% for 1mg and 52.7% for placebo.

Enobosarm was generally safe and well tolerated.

Reported adverse events were minimal and similar across all treatment groups.

“We are very disappointed that the ASTRID Trial did not achieve its primary endpoint,” said Robert Wills, executive chairman of GTx.

“We plan to conduct a full review of all the data. We want to thank the patients, physicians, study coordinators and the entire GTx team for their support of this novel study. We have an ongoing preclinical program assessing the potential of SARDs, our novel selective androgen receptor degrader technology, to treat castration-resistant prostate cancer. We are currently on target to have development candidates by year end, which we potentially plan to take into IND-enabling studies.”

GTXI closed at $23.29, it last traded at $2.00.


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Spark Therapeutics tumbles on hemophilia study

Spark says Phase 1/2 data for SPK-8011 shows a 97% reduction in ABR

Spark tumbles on hemophilia study , Stockwinners
Spark tumbles on hemophilia study , Stockwinners

 

Shares of Spark Therapeutics (ONCE) are sinking after the company announced preliminary Phase 1/2 data for its investigational gene therapy candidate SPK-8011 for hemophilia A. A dose response as demonstrated by FVIII expression ranged from 16% to 49%, with a mean of 30% post 12 weeks in five of the participants in the 2×1012 vg/kg cohort, Spark announced in its Q2 earnings release.

As of the July 13, 2018, data cutoff, 12 participants in the Phase 1/2 trial have received a single administration of investigational SPK-8011, including two at a dose of 5×1011 vector genomes /kg body weight, three at a dose of 1×1012 vg/kg and seven at a dose of 2×1012 vg/kg.

Across all participants, at all three doses, beginning four weeks after vector infusion, there has been a 97-percent reduction in annualized bleeding rate and a 97-percent reduction in annualized infusion rate.

The first two trial participants, who have been followed for greater than one year, have shown stable FVIII activity levels since reaching plateau for up to 66 weeks, with follow up ongoing.

Additionally, there is evidence of a dose-dependent increase in mean FVIII activity levels across the three dose cohorts.

Five of the participants in the 2×1012 vg/kg cohort have FVIII activity levels between 16 and 49 percent, with follow-up ranging from 12 to 30 weeks.

The mean FVIII activity for these five participants is 30 percent, based on average FVIII levels post-12 weeks after vector infusion. These five participants have reduced their overall ABR by 100 percent and reduced their overall AIR by 100 percent.

The other two participants in the 2×1012 vg/kg cohort had an immune response that caused their FVIII levels to decline to less than 5 percent. Clinically, both participants have moved from prophylactic to on-demand treatment and have seen meaningful reductions in their bleeding and infusion rates.

One of these participants did not rapidly respond to oral steroids and he elected to be admitted to the hospital to receive two intravenous methylprednisolone infusions rather than have the infusions on an outpatient basis.

The event was subsequently resolved. The admission to hospital for these infusions met the criteria for a serious adverse event.

Of note, across the study, seven of the 12 participants received a tapering course of oral steroids in response to an alanine aminotransferase elevation above patient baseline, declining FVIII levels and/or positive IFN-g enzyme-linked immunospots.

For these seven participants, steroids led to normalization of ALT and ELISPOTs.

For all but the two above mentioned 2×1012 vg/kg cohort participants, oral steroids led to stabilization of target FVIII levels. Based on the totality of the results to date, Spark Therapeutics intends to initiate a Phase 3 run-in study in the fourth quarter of 2018. Following completion of the run-in study, Phase 3 participants are expected to receive 2×1012 vg/kg of SPK-8011.

Additional details on the Phase 3 trial design will be determined following continued discussions with FDA and EMA, which are expected in the fourth quarter.

Finally, the company has successfully scaled-up its mammalian-based manufacturing process in suspension to a capacity level of 200 liters and amended its agreement with Brammer Bio to secure a dedicated manufacturing suite, both of which will enable Spark Therapeutics to meet supply needs for Phase 3 clinical development as well as expected commercial requirements.

ONCE closed at $77.61, it last traded at $56.00.


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