ResTORbio higher on data

ResTORbio announces ‘positive’ topline results in Phase 2b trial of RTB101

ResTORbio higher on data, Stockwinners
ResTORbio higher on data, Stockwinners

ResTORbio (TORC) announced positive topline results from its dose-ranging Phase 2b clinical trial that enrolled 652 elderly patients at increased risk of morbidity and mortality associated with respiratory tract infections.

In this trial, RTB101, an oral, selective, and potent inhibitor of target of rapamycin complex 1, demonstrated a statistically significant and clinically meaningful reduction in the percentage of patients with one or more laboratory-confirmed RTIs during the 16-week treatment period compared to placebo, the primary endpoint of the study, with the 10 mg once daily dose.

Greater TORC1 inhibition with RTB101 10 mg in combination with everolimus 0.1 mg did not meet the primary endpoint, suggesting that that less TORC1 inhibition with RTB101 10 mg once daily may have greater benefit in high-risk elderly patients.

The Phase 2b trial was a two-part, randomized, double-blind, placebo-controlled clinical trial conducted during the winter cold and flu season in the southern hemisphere and northern hemisphere.

Patients enrolled were those at increased risk of morbidity and mortality from RTIs including patients who were: 85 years of age or older, or 65 years of age or older with asthma, type 2 diabetes mellitus, chronic obstructive pulmonary disease, or current smokers.

The doses investigated in Part 1 were RTB101 5 mg and RTB101 10 mg once daily. The doses investigated in Part 2 were RTB101 10 mg once daily, RTB101 10 mg twice daily and RTB101 10 mg in combination with everolimus 0.1 mg once daily.

The following was observed in an analysis of the primary endpoint: A 30.6% decrease relative to placebo in the percentage of all patients treated with RTB101 10 mg once daily who developed one or more laboratory-confirmed RTs. A 20.6% decrease relative to placebo in the percentage of all patients treated with RTB101 5 mg once daily who developed one or more laboratory-confirmed RTIs.

No decrease relative to placebo in the percentage of patients treated with either RTB101 10 mg twice daily or the combination of RTB101 10 mg + everolimus 0.1 mg once daily who developed one or more laboratory-confirmed RTIs, suggesting that less TORC1 inhibition with RTB101 10 mg once daily may have greater benefit in high-risk elderly patients.

To better understand the activity observed in the RTB101 10 mg once daily cohort, a pre-specified analysis of each patient subgroup enrolled in the study was conducted.

The following decreases in the percentage of patients with laboratory-confirmed RTIs were observed in the RTB101 10 mg once daily cohort as compared to the placebo cohort: A 68.4% decrease in all asthma patients.

A 66.7% decrease in all patients 85 years of age and older. A 26.9% decrease in all T2DM patients.

No decrease was observed in either COPD patients or current smokers; a 42.0% decrease in all patients was observed when excluding patients with COPD and a 43.9% decrease in all patients was observed when excluding current smokers.

All doses were observed to be well-tolerated. Data from the RTB101 10 mg once daily cohort are as follows: Adverse events were balanced between the RTB101 10 mg once daily and placebo treatment groups. 4.5% of subjects in the RTB101 10 mg once daily cohort and 7.2% of subjects in the placebo cohort had a serious adverse event, none of which were considered related to study drug. 4.5% of subjects in the RTB101 10 mg once daily cohort and 6.1% of subjects in the placebo cohort discontinued study drug due to an AE.

All AEs were mild or moderate except for 11 severe AEs in the RTB101 10 mg once daily cohort and 22 severe AEs in the placebo cohort.

TORC closed at $9.01, it last traded at $24.49.


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 Apollo in talks to acquire LifePoint Health

Apollo in advanced discussions to acquire LifePoint Health

Apollo in advanced discussions to acquire LifePoint Health, Stockwinners
Apollo in advanced discussions to acquire LifePoint Health, Stockwinners

Apollo Global (APO) is in advanced talks to buy LifePoint Health (LPNT), two people familiar with the matter told Reuters on Friday.

The deal could value LifePoint at nearly $6B, including debt, the sources said, adding that Apollo plans to combine LifePoint with RegionalCare Hospital Partners, another regional hospital operator that it owns.

If the negotiations are completed successfully, a deal could be announced as early as next week, the sources said, cautioning that it was possible talks could fail at the last minute. The sources asked not to be identified because the matter is confidential.

Rural healthcare providers such as LifePoint have been challenged in recent years because their reliance on federal insurers such as Medicare and Medicaid has made them particularly vulnerable to changing reimbursement programs. In addition, hospital operating costs have been rising faster than reimbursement rate increases.

Apollo, which raised a $24.6 billion private equity fund last year, acquired RegionalCare in 2015, and merged it with another hospital operator, Capella Healthcare, in 2016.

It would be by far the biggest acquisition for Apollo this year. LifePoint currently has a market capitalization of $1.9 billion and long-term net debt of $2.9 billion.

LPNT closed at $47.90.


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Zogenix sharply higher on data

Zogenix says primary endpoint achieved in second Phase 3 clinical trial of ZX008

Zogenix sharply higher on data, Stockwinners
Zogenix sharply higher on data, Stockwinners

Zogenix (ZGNX) reported positive top-line results from its second confirmatory Phase 3 study for its investigational drug, ZX008, for the treatment of children and young adults with Dravet syndrome.

Dravet syndrome is a rare, catastrophic, lifelong form of epilepsy that begins in the first year of life with frequent and/or prolonged seizures. Previously known as Severe Myoclonic Epilepsy of Infancy (SMEI), it affects one out of 15,700 individuals, 80% of whom have a mutation in their SCN1A gene.

The study results, which are consistent with those reported in Study 1, Zogenix’s first pivotal Phase 3 study, successfully met the primary endpoint and all key secondary endpoints, demonstrating that ZX008, at a dose of 0.5 mg/kg/day, is superior to placebo when added to a stiripentol regimen. Key Findings: Patients taking ZX008 achieved a 54.7% greater reduction in mean monthly convulsive seizures compared to placebo.

The median reduction in monthly convulsive seizure frequency was 62.7% in the ZX008 group compared to 1.2% in placebo patients. ZX008 also demonstrated statistically significant improvement versus placebo in both key secondary measures, including patients with clinically meaningful reductions in seizure frequency and longest seizure-free interval.

ZX008 was generally well-tolerated in this study with the adverse events consistent with those observed in Study 1 and the known safety profile of fenfluramine.

No patient exhibited cardiac valvulopathy or pulmonary hypertension at any time in the study.

Secondary endpoints assessed ZX008 compared to placebo in terms of the proportions of patients who achieved greater than or equal to 50% reductions and greater than or equal to 75% reductions in monthly convulsive seizures, as well as the median of the longest convulsive seizure-free interval.

ZX008 was generally well-tolerated in this study, with the adverse events consistent with those observed in Study 1 and the known safety profile of fenfluramine.

The incidence of treatment emergent adverse events was similar in both the treatment and placebo groups, with 97.7% of patients receiving ZX008 experiencing at least one treatment emergent adverse event compared to 95.5% of patients in the placebo group.

The most common adverse events in the ZX008 group were decreased appetite, diarrhea, pyrexia, fatigue, and nasopharyngitis.

The incidence of serious adverse events was similar in both the treatment and placebo groups, with 14% of patients in the ZX008 group experiencing at least one treatment emergent serious adverse event compared to 15.9% of patients in the placebo group. T

wo patients in the ZX008 group had an adverse event leading to study discontinuation compared to one in the placebo group.

ZGNX closed at $46.30, it last traded at $55.30.


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Pfizer shares lower after talk with President prompts rollback

Pfizer under pressure after talk with President prompts rollback

Pfizer shares lower after talk with President prompts rollback, Stockwinners
Pfizer shares lower after talk with President prompts rollback, Stockwinners

After speaking with President Donald Trump, Pfizer (PFE) stated that it will roll back its July 1 price increases “to give the president an opportunity to work on his blueprint to strengthen the healthcare system and provide more access for patients.”

Additionally this morning, the company announced that it will reorganize into three units, separating its consumer health-care business, which the drugmaker has been trying to sell since last year, from its groups focused on “innovative” medicines” and “established” medicines.

PRICE ROLLBACK

In a statement provided to CNBC’s Meg Tirrell yesterday, Pfizer said it will roll back its July 1 price increases “to give the president an opportunity to work on his blueprint to strengthen the healthcare system and provide more access for patients.”

The company released the statement following an “extensive discussion” with President Trump. Pfizer said it will return such prices to their pre-July 1 levels as soon as technically possible, and the prices will remain in effect until the earlier of when the president’s blueprint goes into effect or the end of the year. In addition, the drug giant said that the price declines the company took as of July 1 will remain in effect.

This comes after President Trump called out the company and other drugmakers for raising prices.

In a tweet, Trump previously said Pfizer and peers “should be ashamed that they have raised drug prices for no reason,” accusing the company and industry of “merely taking advantage of the poor and others unable to defend themselves, while at the same time giving bargain basement prices to other countries in Europe and elsewhere.”

BUSINESS REORGANIZATION

This morning, Pfizer also announced it will organize the company into three businesses, namely a science-based Innovative Medicines business that will now include biosimilars and a new hospital business unit for anti-infectives and sterile injectables; an off-patent branded and generic Established Medicines business operating with substantial autonomy within Pfizer; and a Consumer Healthcare business.

These changes will be effective at the beginning of the company’s 2019 fiscal year, and are not expected to impact current capital allocation priorities or full-year 2018 financial guidance.

READ-THROUGH TO OTHERS IN THE SECTOR

Commenting on the events, Wells Fargo analyst David Maris told investors that drug stocks will not react favorably to this news, given the chilling effect this will likely have on others looking to take price increases.

Nonetheless, the analyst pointed out that the price increases taken in July are only small compared to other increases taken over the past year or several years, so the impact of the rollback to the healthcare system is insignificant in the big picture.

Maris also added that he believes the administration’s and other key legislators’ focus is not only on drug pricing, but on the overall supply chain and delivery system, including drug rebating, co-pay coupons, etc.

WHAT’S NOTABLE

According to Bloomberg, Gilead (GILD), Roche (RHHBY), Novo Nordisk (NVO) and Novartis (NVS) have all sent notices to California health plans rescinding or reducing previously announced price hikes in the wake of a new drug pricing transparency law that was enacted in the state.

The California measure, which is among the most aggressive efforts by states to rein in drug costs, is being challenged in court by the drug industry’s lobbying group, the report noted.

Other large cap pharmaceutical companies include AstraZeneca (AZN), Bristol-Myers (BMY), Eli Lilly (LLY), GlaxoSmithKline (GSK), Johnson & Johnson (JNJ), Merck (MRK), and Sanofi (SNY).

PRICE ACTION

In morning trading, shares of Pfizer have dropped about 0.5% to $37.28.


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Bristol-Myers treatment for colorectal cancer approved

Bristol-Myers’ Opdivo approved for MSI-H/dMMR metastatic colorectal cancer

Bristol-Myers treatment for colorectal cancer approved, Stockwinners
Bristol-Myers treatment for colorectal cancer approved, Stockwinners

Bristol-Myers (BMY) announced Opdivo – nivolumab – 3 mg/kg plus low-dose Yervoy – ipilimumab – 1 mg/kg injections for intravenous use received approval from the FDA for the treatment of adult and pediatric patients 12 years and older with microsatellite instability high or mismatch repair deficient metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin and irinotecan.

Approval for this indication has been granted under accelerated approval.

Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

The application was granted Priority Review and Breakthrough Therapy Designation by the FDA.

Among the 82 patients who received prior treatment with a fluoropyrimidine, oxaliplatin and irinotecan, 46% responded to treatment with Opdivo + Yervoy.

The percentage of these patients with a complete response was 3.7%, and the percentage of patients with a partial response was 43%.

Among these 38 responders, the median DOR was not reached; 89% of those patients had responses of six months or longer, and 21% had responses of 12 months or longer.

This trial is ongoing. Among all enrolled patients, 49% responded to treatment with Opdivo + Yervoy; 4.2% experienced a complete response, while 45% experienced a partial response.

Opdivo was discontinued in 13% of patients and delayed in 45% of patients due to an adverse reaction.

Serious adverse reactions occurred in 47% of patients.

The Opdivo + Yervoy combination is also approved in two other tumor types, advanced renal cell carcinoma and unresectable or metastatic melanoma.

Continued approval for these accelerated approval indications may be contingent upon verification and description of clinical benefit in the confirmatory trials.

BMY closed at $56.18.


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Celgene announces positive breast cancer data

Celgene announces IMpassion130 study meets co-primary endpoint of PFS

Celgene announces positive breast cancer data, Stockwinners
Celgene announces positive breast cancer data, Stockwinners

Celgene (CELG) announced that the Phase III IMpassion130 study met its co-primary endpoint of progression-free survival, or PFS.

This is the first phase III study to demonstrate a statistically significant PFS improvement in first-line metastatic or unresectable locally advanced triple negative breast cancer, or TNBC, a type of breast cancer with high unmet need.

Results demonstrated that the investigational combination of Tecentriq plus Abraxane compared to Abraxane monotherapy, as an initial treatment, significantly reduced the risk of disease worsening or death in patients with metastatic or unresectable locally advanced TNBC in the intention-to-treat and PD-L1 positive populations.

Overall survival is encouraging in the PD-L1 positive population at this interim analysis, and follow up will continue until the next planned analysis.

Safety in the Tecentriq plus Abraxane arm appeared consistent with the known safety profiles of the individual medicines, and no new safety signals were identified with the combination.

ABRAXANE is a microtubule inhibitor indicated for the treatment of:

Metastatic breast cancer, after failure of combination chemotherapy
for metastatic disease or relapse within 6 months of adjuvant
chemotherapy. Prior therapy should have included an anthracycline
unless clinically contraindicated.
• Locally advanced or metastatic non-small cell lung cancer (NSCLC),
as first-line treatment in combination with carboplatin, in patients who
are not candidates for curative surgery or radiation therapy.
• Metastatic adenocarcinoma of the pancreas as first-line treatment, in
combination with gemcitabine.

TECENTRIQ is a prescription medicine used to treat:

A type of bladder and urinary tract cancer called urothelial carcinoma.

  • TECENTRIQ may be used when your bladder cancer:
    • has spread or cannot be removed by surgery, and
    • you are not able to take chemotherapy that contains a medicine called cisplatin, or
    • you have tried chemotherapy that contains platinum, and it did not work or is no longer working.

CELG closed at $83.85.


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Biogen sharply higher on data

Eisai and Biogen announce positive results of the final analysis for BAN2401

Biogen sharply higher on data, Stockwinners
Biogen sharply higher on data, Stockwinners

Eisai (ESALY) and Biogen (BIIB) announced positive topline results from the Phase II study with BAN2401, an anti-amyloid beta protofibril antibody, in 856 patients with early Alzheimer’s disease.

The study achieved statistical significance on key predefined endpoints evaluating efficacy at 18 months on slowing progression in Alzheimer’s Disease Composite Score and on reduction of amyloid accumulated in the brain as measured using amyloid-PET. Topline results of the final analysis of the study demonstrated a statistically significant slowing of disease progression on the key clinical endpoint after 18 months of treatment in patients receiving the highest treatment dose as compared to placebo.

Results of amyloid PET analyses at 18 months, including reduction in amyloid PET standardized uptake value ratio and amyloid PET image visual read of subjects converting from positive to negative for amyloid in the brain, were also statistically significant at this dose. Dose-dependent changes from baseline were observed across the PET results and the clinical endpoints.

Further, the highest treatment dose of BAN2401 began to show statistically significant clinical benefit as measured by ADCOMS as early as 6 months including at 12 months. BAN2401 demonstrated an acceptable tolerability profile through 18 months of study drug administration.

The most common treatment emergent adverse events were infusion-related reactions and Amyloid Related Imaging Abnormalities. Infusion related reactions were mostly mild to moderate in severity. Incidence of ARIA-E was not more than 10% in any of the treatment arms, and less than 15% in patients with APOE4 at the highest dose per the study protocol safety and reporting procedures.

BIIB closed at $298.81, it last traded at $338. ESALY closed at $71.10.


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Zynerba lower following disappointing results

Zynerba says target blood levels in ZYN001 THC-Prodrug patch study not achieved

Zynerba lower following disappointing results, Stockwinners
Zynerba lower following disappointing results, Stockwinners

Zynerba Pharmaceuticals (ZYNE) announced top line results from a Phase 1 clinical program studying ZYN001, the Company’s patent-protected, pro-drug of tetrahydrocannabinol delivered via a transdermal patch, in healthy volunteers.

The program assessed the safety and pharmacokinetics in single and multiple doses of several formulations of ZYN001.

The top line results of this Phase 1 study indicate that target blood levels of 5 to 15 ng/ml THC were not achieved.

ZYN001 was very well tolerated with minimal skin erythema.

There were no serious adverse events or discontinuations for subjects receiving ZYN001.

As a result of these data, the Company will focus its development efforts and investments on the ZYN002 Fragile X syndrome, developmental and epileptic encephalopathy and adult refractory epilepsy programs.

The company expects that this change will extend its cash runway into the second half of 2019.

This Phase 1 study was a single and multiple dose, placebo-controlled first-in-man study to assess the safety and pharmacokinetics of ZYN001 administered as a transdermal patch to healthy adult subjects.

Several formulations and patch wear times ranging from 24 hours to 14 days were assessed in in 60 healthy subjects who were randomized to ZYN001 or placebo.

ZYNE closed at $9.61, it last traded at $7.60.


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Juniper Pharmaceuticals sold for $139.6 Million

Catalent agrees to acquire Juniper Pharmaceuticals for $11.50 per share 

Juniper Pharmaceuticals sold for $139.6 Million, Stockwinners
Juniper Pharmaceuticals sold for $139.6 Million, Stockwinners

Catalent (CTLT) announced that it has agreed to acquire Juniper Pharmaceuticals (JNP), including its Nottingham, U.K.-based Juniper Pharma Services division.

When combined with Catalent’s existing industry-leading drug development and manufacturing capabilities in the U.S. and Europe, the acquisition of Juniper will expand and strengthen Catalent’s offerings in formulation development, bioavailability solutions and clinical-scale oral dose manufacturing, and will complement its integrated global clinical and commercial supply network. Juniper’s nearly 150 employees have deep scientific expertise in formulation development, and supply, and will augment Catalent’s current portfolio of solid-state screening, preformulation, formulation, analytical, and bioavailability enhancement solutions, including development of spray-dried dispersions, with integrated development, analytical, and clinical manufacturing co-located in its Nottingham facility.

Catalent will continue to support Juniper’s CRINONE franchise marketed by Merck KGaA outside of the U.S.

Juniper’s Intravaginal Ring development pipeline was previously licensed to Dare Bioscience, and Catalent will not be involved in the further development of this program.

The acquisition of Juniper is subject to certain customary closing conditions, including that a majority of Juniper’s shares are tendered into the offer, and is expected to close in the first quarter of Catalent’s 2019 fiscal year, which began on July 1, 2018.

Like Catalent, Juniper has expertise in solid-state and preclinical formulation screening for lead-candidate selection, phase-appropriate dose-form development, and superior technologies for challenging molecules, which will strengthen and expand on Catalent’s OptiForm Solution Suite platform.

Juniper provides bioavailability enhancement solutions for the development of poorly soluble compounds, including nano-milling, spray drying, hot-melt extrusion, lipid-based drug delivery, and cGMP clinical manufacturing, including specialized facilities and controls for potent and controlled substances. Under its acquisition agreement with Juniper, a subsidiary of Catalent will promptly commence a tender offer to purchase all of Juniper’s shares for a price of $11.50, net to the seller in cash.

Following the conclusion of the tender offer, Catalent intends to complete the transaction by acquiring the remainder of the Juniper shares at the same price through a merger with a newly formed wholly owned subsidiary of Catalent.

JNP closed at $8.70. CTLT closed at $41.82.


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Compugen Higher as FDA lifts clinical hold on its cancer treatment 

Compugen Higher as FDA lifts clinical hold on its cancer treatment 

Compugen Higher as FDA lifts clinical hold on its cancer treatment , Stockwinners
Compugen Higher as FDA lifts clinical hold on its cancer treatment , Stockwinners

Shares of Compugen (CGEN) surged in pre-market trading after the Israeli biotech company said that the clinical hold on its investigational new drug application for COM701 was lifted by the U.S. Food and Drug Administration.

Separately, Compugen said the FDA cleared Bayer’s (BAYRY) IND application for BAY 1905254.

CLINICAL HOLD LIFTED

Compugen said this morning that the FDA has lifted the clinical hold on its IND for COM701, an immuno-oncology therapeutic antibody targeting PVRIG in patients with advanced solid tumors.

The FDA said the company may now begin a clinical study of the antibody. PVRIG is an investigational monoclonal antibody, a molecule that has the ability to bind with high specificity to a given target, being developed by Compugen to treat various types of cancer.

“We believe the COM701 preclinical data suggest that targeting PVRIG may effectively stimulate an anti-tumor immune response in certain cancers such as breast, endometrial, ovarian and lung, and specifically in patient populations that are unresponsive to current checkpoint inhibitors,” Compugen President and Chief Executive Officer Anat Cohen-Dayag stated.

Looking ahead, Compugen said it plans to initiate a first-in-human Phase 1 study in the U.S. in patients with advanced solid tumors and for whom standard of care therapies are currently ineffective.

In April, Compugen said the FDA requested additional CMC information from the company in support of the COM701 IND application.

At the time, the FDA recommended a lower starting dose of COM701 for the trial.

BAYER IND APPLICATION CLEARANCE

Additionally this morning, Computgen said that the FDA approved Bayer’s (BAYRY) IND application for BAY 1905254, a a first-in-class immuno-oncology therapeutic antibody targeting the ILDR2 protein in patients with advanced solid tumors.

Compugen is entitled to receive a milestone payment upon the first patient dosing with BAY 1905254 in the Phase 1 clinical trial expected in 2018.

PRICE ACTION

In pre-market trading, Compugen is up 45c, or 13.6% to $3.75 per share.


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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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Dermira Receives FDA Approval to Treat excessive armpit sweating

Dermira Receives FDA Approval to Treat Primary Axillary Hyperhidrosis

Dermira Receives FDA Approval to Treat Primary Axillary Hyperhidrosis, Stockwinners
Dermira Receives FDA Approval to Treat Primary Axillary Hyperhidrosis, Stockwinners

Dermira, Inc. (DERM) announced that the U.S. Food and Drug Administration (FDA) has approved Qbrexza™ (glycopyrronium) cloth, an anticholinergic indicated for the topical treatment of primary axillary hyperhidrosis in adult and pediatric patients 9 years of age and older.

Primary axillary hyperhidrosis, also commonly known as excessive underarm sweating, is a chronic medical skin condition that results in sweating beyond what is needed for normal body temperature regulation. The exact cause is unknown, but it affects nearly 10 million people in the United States, with both men and women having similar prevalence. Qbrexza (pronounced kew brex’ zah) is applied directly to the skin and is designed to block sweat production by inhibiting sweat gland activation.

The approval is based on results from two Phase 3 clinical trials, ATMOS-1 and ATMOS-2, which evaluated the efficacy and safety of Qbrexza in patients with primary axillary hyperhidrosis. Both trials assessed the absolute change from baseline in sweat production (the weight or amount of sweat a patient produced) following treatment with Qbrexza and the proportion of patients who achieved at least a four-point improvement from baseline in their sweating severity, as measured by the Axillary Sweating Daily Diary (ASDD), Dermira’s proprietary patient-reported outcome (PRO) instrument. The PRO was developed in consultation with the FDA and in accordance with the agency’s 2009 guidance on PRO instruments.

DERM closed at $8.78, it last traded at $10.57.


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Cara reports positive top-line data from CR845

Cara reports ‘positive’ top-line data from Phase 2/3 trial of I.V. CR845

 

Cara reports positive top-line data from CR845, Stockwinners
Cara reports positive top-line data from CR845, Stockwinners

Cara Therapeutics (CARA) announced positive top-line data from the adaptive Phase 2/3 trial of I.V. CR845 in patients undergoing abdominal surgeries.

At the 1.0 mcg/kg dose, I.V. CR845 demonstrated statistically significant reductions in pain intensity compared to placebo at all pre-specified post-operative periods of 0-6 hours; 0-12 hours; 0-18 hours; and 0-24 hours.

Additionally, I.V. CR845 treatment resulted in statistically significant reductions in the incidence of post-operative nausea and vomiting over the 24-hour period post-surgery for both 0.5 and 1.0 mcg/kg doses.

The adaptive Phase 2/3 trial was a randomized, double-blind, placebo-controlled trial designed to evaluate the analgesic efficacy and safety of two doses of I.V. CR845 versus placebo given at pre-specified intervals pre- and post-surgery in 444 patients undergoing abdominal surgery, composed of 228 patients who underwent ventral hernia surgery and 216 patients who completed a hysterectomy procedure.

Patients received a 2X loading dose of I.V. CR845 pre-surgery and four additional doses given at 0, 6, 12 and 18 hours after surgery. The primary endpoint was pain relief as measured by Area Under the Curve of the Numerical Rating Scale pain intensity scores collected over the first 24-hour period after the baseline dose post-surgery for all combined surgeries.

In addition to safety, the secondary endpoints included incidence of vomiting, improvement in impact scores of post-operative nausea and vomiting, reduction in use of rescue analgesic medication, as well as patient global assessment at 24 hours post baseline dose after surgery. I.V. CR845 achieved statistical significance for the primary endpoint of pain relief over 24 hours post-surgery with the 1.0 mcg/kg dose versus placebo and also demonstrated statistical significance across two additional pre-specified sensitivity analyses for pain relief for the same period post-surgery. The 0.5 mcg/kg dose did not achieve statistical significance over the 0-24 hour period.

In addition, improvement in pain AUC was statistically significant for both the 0.5 and 1.0 mcg/kg doses over 0 to 6 hours and 0 to 12 hours periods and also statistically significant for the 1.0 mcg/kg dose over the 0 to 18-hour period post-surgery.

At 6 and 24 hours after baseline dose post-surgery, there were statistically significant improvements in PONV impact scores with both doses of I.V. CR845 compared to placebo: 0.5 mcg/kg and 1.0 mcg/kg. There were statistically significant differences between placebo and both doses of CR845 with respect to the total use of anti-emetic medication over the first 24 hours post-surgery.

The percentage of patients who did not take any anti-emetic medication over 24 hours was 56% for placebo compared to 70% for CR845 0.5 mcg/kg and 81% for CR845 1.0 mcg/kg.

There was a 73% reduction in the incidence of patient-reported vomiting in the group receiving the 1.0 mcg/kg dose versus placebo. Although the 0.5 mcg/kg also showed reduction in vomiting, it did not reach statistical significance.

Both doses of I.V. CR845 exhibited numerical trends toward reduced use of rescue analgesic medication compared to placebo, but did not achieve statistical significance.

There was no significant effect, compared to placebo, on patient’s global assessment of medication for either dose of I.V. CR845 over the 24-hour period. Common adverse effects reported in the placebo and both I.V. CR845 groups were generally low and similar in incidence, and included nausea, constipation, vomiting, flatulence, headache and dyspepsia.

CARA closed at $16.44, it last traded at $22.25.


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GE to spin off several divisions

  • GE Healthcare to become standalone company

  • GE plans to fully separate Baker Hughes

GE introduces new company AiRXOS, Stockwinners
GE to spin off several of its divisions

GE (GE) announced the results of its strategic review.

GE will focus on Aviation, Power and Renewable Energy, creating a simpler, stronger, leading high-tech Industrial company.

In addition to the pending combination of its Transportation business with Wabtec, GE plans to separate GE Healthcare into a standalone company, pursue an orderly separation from BHGE (BHGE) over the next two to three years, make its corporate structure leaner and substantially reduce debt.

GE’s Board of Directors unanimously approved the plans announced today.

GE is making fundamental changes to how it will run the company.

The new GE Operating System will result in a smaller corporate headquarters focused primarily on strategy, capital allocation, talent and governance.

It will result in better execution, increased speed and is expected to generate at least $500 million in corporate savings by the end of 2020.

Under the new GE Operating System, most resources and services traditionally held at the headquarters level will be realigned to the businesses. GE is targeting an Industrial net debt-to-EBITDA ratio of less than 2.5 times and a long-term A credit rating.

GE also plans to reduce Industrial net debt by approximately $25 billion by 2020 and maintain more than $15 billion of cash on the balance sheet.

GE expects to maintain its current quarterly dividend, subject to Board approval, until GE Healthcare is established as an independent entity.

At that time, the new GE Healthcare Board of Directors will determine GE Healthcare’s dividend policy, which GE expects to reflect healthcare industry practices.

Also at that time, the GE Board expects to adjust the GE dividend with a target dividend policy in line with industrial peers. Kieran Murphy, president and CEO of GE Healthcare, will continue to lead GE Healthcare as a standalone company, maintaining the GE brand.

GE expects to generate cash from the disposition of approximately 20% of its interest in the Healthcare business and to distribute the remaining 80% to GE shareholders through a tax-free distribution.

The structure, sequence and timing of these transactions will be determined and announced at a later date, but are expected to be completed over the next 12 to 18 months.

GE Healthcare will conduct business as usual throughout this process, continuing to serve its partners and customers.

GE plans to fully separate its 62.5% interest in BHGE in an orderly manner over the next two to three years. BHGE’s full stream offering brings together equipment, services and digital solutions to help its customers be more productive-a unique and powerful value proposition in a changing market.

The separation will provide BHGE with enhanced agility and the ability to focus on leading in the oil and gas industry.

Shares of the former DJIA component closed at $12.75.


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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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