ORENCIA Approved for Psoriatic Arthritis

Bristol-Myers receives FDA approval for PsA treatment ORENCIA

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Bristol-Myers Squibb Company (BMY) announced the U.S. Food and Drug Administration has approved #ORENCIA for the treatment of adults with active Psoriatic Arthritis, a chronic, inflammatory disease that can affect both the skin and musculoskeletal system.

ORENCIA is approved and available in both intravenous and subcutaneous injection formulations.

ORENCIA should not be administered concomitantly with TNF antagonists, and is not recommended for use concomitantly with other biologic Rheumatoid Arthritis therapy, such as anakinra.

This approval marks the third autoimmune disease indication for ORENCIA.

The co-stimulation blockade of ORENCIA inhibits T-cell activation and the resulting cascade of events that contribute to inflammation.

T-cell activation is involved in the pathogenesis of PsA. The approval was based on results from two randomized, double-blind, placebo-controlled trials in which ORENCIA improved disease activity in both TNF-naive and exposed patients with high disease activity, high tender and swollen joints, and a disease duration of more than seven years.

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Cara Therapeutics’ Rise Could Continue

Watch Cara Therapeutics into osteoarthritis pain results

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Cara Therapeutics (CARA) has soared this month amid the wider biotech rally, but research firm Janney argues the stock could continue higher on a pending release of data from a study of its potential treatment for osteoarthritis pain.

BACKGROUND

Cara Therapeutics is a clinical-stage researcher focused on treating pain and pruritus, or itching, by targeting the body’s so-called “kappa” #opioid receptors.

The company’s pipeline includes #CR845 and #CR701, the former of which is in advanced studies for both oral and intravenous forms.

In its most recent earnings report on May 4, Cara said it expects top-line data from the Phase 2b trial of oral CR845 for osteoarthritis-related pain in Q2, and the company confirmed on its conference call that the trial is “now fully enrolled at 480 patients and on track for a data readout later in Q2.” Cara’s second quarter ends June 30.

JANNEY SEES BIG POTENTIAL MOVE

Janney’s Ken #Trbovich said Wednesday he expects “significant volatility” in Cara Therapeutics as investors await the Phase 2 osteoarthritis data.

Highlighting the stock’s all-time highs on the back of the biotech rally and Friday’s announcement of “breakthrough therapy” designation, the analyst nevertheless says positive osteoarthritis data can “easily justify” a move into the $31-$36 range, though he warns that negative results could take the stock to $10-$15.

The long-term potential of oral #CR845 in chronic pain is “tantalizing” for a company whose market cap remains below $1B — with Trbovich offering Nektar’s (NKTR) 42% surge on the back of Phase 3 opioid data as an example — but the analyst cautions that the pending results are only mid-stage and that “a portion” of the potential upside has already been priced in.

Modeling the company on a discounted cash flow basis while factoring any potential dilution necessary to fund and launch oral CR845, Trbovich ends up at $31-$34 per share assuming peak sales of just over $1B.

On the other hand, he expects a retrace to $15 if data is negative, or even as low as $10-$12 if results are particularly bad and investors apply the news to Cara’s pain program for intravenous CR845.

POSITIVE HINT FROM UNRELATED TRIAL

H.C. #Wainwright analyst Corey Davis wrote June 22 that the osteoarthritis data is “expected any day.” #Davis added that Cara’s announcement that week of continuing its Phase 3 trial of I.V.

#CR845 in postoperative pain after an interim analysis — while largely unconnected from the osteoarthritis trial — is “still a slight positive indicator” given the lack of futility or safety signals.

PRICE ACTION:

CARA last traded at $27.72.

See our recent post on CARA.

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FDA Grants Conatus’ IDN-7314 Orphan Drug Designation

Conatus (CNAT) granted orphan drug designation for IDN-7314 for treatment of PSC

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Conatus Pharmaceuticals (CNAT) announced that the U.S. FDA has granted Orphan Drug Designation to Conatus’ drug candidate IDN-7314 for the treatment of primary sclerosing cholangitis, a disease affecting bile ducts in the liver which can lead to cirrhosis and liver failure.

The FDA’s Orphan Drug Designation program is intended to encourage the development of drugs and biologics that may provide benefit to patients suffering from rare diseases or conditions.

IDN-7314 is an orally active pan-caspase protease inhibitor designed to reduce the activity of enzymes that mediate inflammation and cell death, which has demonstrated reduction of relevant biomarkers in two preclinical models of PSC. One nonclinical model, the Mdr2-/- mouse model, is considered the current benchmark nonclinical model of PSC.

A new preclinical model, second mitochondria-derived activator of caspases-mimetic induced PSC in mice, has recently been reported that reproduces much of the phenotype of human PSC. IDN-7314 significantly improved biochemical indices of hepatic and biliary damage in these murine models of PSC, and these results suggest the involvement of caspases in the progression of PSC.

Other stocks to watch include: ICPT, INCY and AZN.

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FDA Approves Portola Pharmaceuticals’ Bevyxxa

Portola Pharmaceuticals announces FDA approval of Bevyxxa, Shares rise 45%

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#Portola Pharmaceuticals (PTLA) announced the U.S. Food and Drug Administration has approved #Bevyxxa, which it said is “the first and only” anticoagulant for hospital and extended duration prophylaxis of venous thromboembolism, or #VTE, in adult patients hospitalized for an acute medical illness who are at risk for thromboembolic complications due to moderate or severe restricted mobility and other risk factors for VTE.

The drug, BevyxXa, known also as betrixaban, is the first oral treatment and first extended duration treatment for this patient population, the company said.

Roughly 200,000 people in the United States develop deep vein #thrombosis each year, with about 40,000 of them dying of pulmonary embolism, caused when a blood clot breaks loose and travels to the lungs, blocking blood flow, the company said.

The timeline on which Portola expects to launch Bevyxxa is between August and November 2017.

During this period, Portola will complete salesforce hiring and training, drug manufacturing validation and inventory buildup, the company said.

BevyxXa was tested using a novel clinical trial strategy designed to test a series of subgroups before testing the broader patient population. It first tested the highest risk patients. Then it tested a lower risk group, and finally the overall patient population.

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Cara Therapeutics Sharply Higher on FDA Action

FDA has granted Breakthrough Therapy designation to I.V. CR845 for the treatment of moderate-to-severe uremic pruritus in chronic kidney disease patients undergoing hemodialysis

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Cara Therapeutics announced that the FDA has granted Breakthrough Therapy designation to I.V. CR845 for the treatment of moderate-to-severe uremic pruritus in chronic kidney disease patients undergoing hemodialysis.

Cara Therapeutics, Inc. (CARA) is a biopharmaceutical company. The company is focused on developing and commercializing new chemical entities designed to alleviate pain and pruritus by selectively targeting peripheral kappa opioid receptors.

“The FDA’s decision to grant Breakthrough Therapy designation is recognition of both the significant unmet medical need among CKD patients with UP and the potential of I.V. CR845 to address it,” said Derek Chalmers, Ph.D., D.Sc., President and Chief Executive Officer of Cara Therapeutics.

“We have already initiated our Phase 3 program and look forward to working closely with the FDA to bring this potential new treatment option to hemodialysis patients as quickly as possible.”

Breakthrough Therapy designation is granted to expedite the development and review process for new therapies addressing serious or life-threatening conditions, where preliminary clinical evidence indicates that the drug candidate may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

CARA closed at $24.25. Shares are up $2 in pre-market trading.

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FDA Grants Thermo Fisher Pre-Market Approval for Oncomine Test

Thermo Fisher says FDA grants premarket approval for Oncomine Dx Target Test that simultaneously screens tumor samples for three FDA-approved therapies for non-small cell lung cancer

Stocks to Buy Now

Thermo Fisher ( $TMO ) announced that the FDA has granted premarket approval for its #Oncomine Dx Target Test, which the company called the first next-generation sequencing-based test that simultaneously screens tumor samples for biomarkers associated with three FDA-approved therapies for non-small cell lung cancer.

LabCorp’s ( $LH ) Diagnostics and Covance Businesses, NeoGenomics (NEO) Laboratories, and Cancer Genetics (CGIX) are among the first laboratories that will offer the Oncomine Dx Target Test as a service to oncologists, Thermo Fisher said.

All tests will be run on Thermo Fisher’s Ion PGM Dx System, which received FDA 510k clearance in parallel for use on formalin-fixed, paraffin-embedded tissue samples.

Thermo Fisher developed the Oncomine Dx Target Test in collaboration with Novartis (NVS) and Pfizer (PFE).

“This first iteration of the test is just the beginning since the diagnostic claims of the Oncomine Dx Target Test may be expanded in the future based on the existing panel.

Thermo Fisher has entered into discussions with several pharmaceutical companies looking to use the panel for FDA-approved targeted therapy applications beyond lung cancer,” the company noted.

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Novadaq sold for $701 million

Stryker to buy Novadaq for $701M

The transaction will be carried out by way of a court approved plan of arrangement under the Canada Business Corporations Act

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#Novadaq Technologies (NVDQ) announced that it has entered into a definitive arrangement with Stryker (SYK) pursuant to which Stryker has agreed to acquire all of the issued and outstanding shares of Novadaq for $11.75 per share in cash, implying a total equity value of approximately $701M.

Novadaq Technologies Inc. develops, manufactures, and markets fluorescence imaging products for use by surgeons in the operating room and other clinical settings in the United States and internationally. The company offers SPY Elite, a fluorescence imaging system that enables surgeons performing open procedures, such as breast and other reconstruction, gastrointestinal, and cardiothoracic surgery, to visualize microvascular blood flow and perfusion in tissue intraoperatively. It also provides PINPOINT endoscopic fluorescence imaging systems; LUNA fluorescence angiography system that provides clinicians with real-time visualization of tissue perfusion in patients.

Stryker Corporation (SYK) operates as a medical technology company.

The transaction will be carried out by way of a court approved plan of arrangement under the Canada Business Corporations Act and will require the approval of, among others, the holders of at least 66 2/3% of the Novadaq Shares present in person or represented by proxy at a special meeting of Novadaq shareholders to be called to consider the Arrangement.

The Special Meeting is expected to be held on or about August 4.

Novadaq’s board and the Special Committee have also received a fairness opinion from each of Piper Jaffray and Perella Weinberg Partners in connection with the Arrangement to the effect that, as of the date of such opinions, and subject to the assumptions, limitations and qualifications set forth therein, the consideration to be received by Novadaq’s shareholders pursuant to the Arrangement is fair from a financial point of view.

In addition to shareholder and court approvals, the Arrangement is subject to applicable regulatory approvals, including Canadian Competition Act and U.S. Hart-Scott-Rodino approvals, and the satisfaction of certain other closing conditions customary in transactions of this nature. The transaction is not subject to a financing condition.

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FDA Asks Removal of Endo Pharmaceuticals’ Opana from the Market

FDA requested that Endo Pharmaceuticals remove its opioid pain medication from the market

This is the first time the FDA has taken steps to remove a currently marketed opioid pain medication

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The U.S. Food and Drug Administration requested that Endo Pharmaceuticals remove its opioid pain medication, reformulated Opana ER (oxymorphone hydrochloride), from the market. After careful consideration, the agency is seeking removal based on its concern that the benefits of the drug may no longer outweigh its risks. This is the first time the agency has taken steps to remove a currently marketed opioid pain medication from sale due to the public health consequences of abuse.

“We are facing an opioid epidemic – a public health crisis, and we must take all necessary steps to reduce the scope of opioid misuse and abuse,” said FDA Commissioner Scott #Gottlieb, M.D. “We will continue to take regulatory steps when we see situations where an opioid product’s risks outweigh its benefits, not only for its intended patient population but also in regard to its potential for misuse and abuse.”

Endo said it is reviewing the request and is evaluating the full range of potential options as we determine the appropriate path forward.

“While the benefits of opioids in treating and managing pain are widely recognized, the misuse and abuse of these products have increased greatly in the U.S.,” the company said in a statement.

“As a pharmaceutical company with a demonstrated commitment to the improvement of pain management, Endo feels a strong sense of responsibility to improve the care of pain for patients while at the same time taking comprehensive steps to minimize the potential misuse of its products. Despite the FDA’s request to withdraw OPANA ER from the market, this request does not indicate uncertainty with the product’s safety or efficacy when taken as prescribed. Endo remains confident in the body of evidence established through clinical research demonstrating that OPANA ER has a favorable risk-benefit profile when used as intended in appropriate patients.”

Other Stocks to Watch

Shares of pain treatment makers Depomed (DEPO) and Insys Therapeutics (INSY) are weaker in extended trading.

Collegium Pharmaceuticals (COLL), shares are up  due to its competing drug. It last traded at $10.50. COLL has a 52-week trading range of $7.37 – $20.55.

ENDP last traded at $12.80. ENDP has a 52-weeks trading range of $9.70 – $24.92.

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Inovio Zika Vaccine Proves Effective in Mice

Inovio Pharmaceuticals INO announced its DNA-based Zika vaccine protected against Zika virus-induced damage to testes and sperm, and prevented persistence of the virus in the reproductive tract of all vaccinated male mice challenged with a high dose of the Zika virus.

This published data suggests another avenue of potential protection against the Zika virus

ino

#Inovio Pharmaceuticals #INO announced its DNA-based #Zika vaccine protected against Zika virus-induced damage to testes and sperm, and prevented persistence of the virus in the reproductive tract of all vaccinated male mice challenged with a high dose of the Zika virus.

This preclinical study data was published in Nature Communications in an article entitled, “DNA Vaccination Protects Mice Against Zika Virus-Induced Damage to the Testes,” written by Inovio scientists and collaborators.

Dr. Gary Kobinger, lead author of the study and Director of the Centre for Research in Infectious Diseases at Laval University in Quebec City, Canada, said, “Given that we know that Zika virus infection can involve the male reproductive tract and persist in humans for several months after onset of infection, this preclinical data warrants further examination as a potential means to reduce Zika virus infection of the male reproductive tract and the risk of sexual transmission of the virus.”

Dr. J. Joseph Kim, Inovio’s President and CEO, said, “This published data suggests another avenue of potential protection against the Zika virus. While detrimental effects on sperm and fertility have not yet been reported in Zika-infected human males, persistence of Zika in semen and sperm and sexual transmission by males has been documented. This new preclinical data suggests that our Zika vaccine may represent an opportunity to limit the potential for sexual transmission of the virus.

In addition to our ongoing ZIKA-001 and 002 clinical studies, we are planning for a larger phase 2 study in our efforts to bring our Zika vaccine to patients.”

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Amazon, the next Threat to Drug Prices

Wells Fargo said Amazon may be the next threat to drug pricing as the company’s move into the prescription pharmacy space could result in increased competition and increased pricing transparency

Although brand-name drugs comprise only 10% of all dispensed prescriptions in the United States, they account for 72% of drug spendingHttps://stockwinners.com/

Last month, #CNBC reported that #Amazon ( $AMZN ) is considering going into the prescription pharmacy business in the U.S. Now Wall Street analysts are questioning if the e-commerce giant has larger plans for the healthcare market.

One of the reasons drug prices are much higher in the United States compared to other industrialized countries is that the U.S. lacks a national healthcare system that directly negotiate with the pharmaceutical industry. Rather, most of the negotiations occur between the pharmaceutical companies and private insurers or vendors. The primary reason for increasing drug spending is the high price of branded products protected by market exclusivity provisions granted by the US Patent and Trademark Office and the Food and Drug Administration (FDA) (rather than a national healthcare system.)

Although brand-name drugs comprise only 10% of all dispensed prescriptions in the United States, they account for 72% of drug spending. Between 2008 and 2015, prices for the most commonly used brand-name drugs increased 164%, far in excess of the consumer price index.

Although brand-name drugs account for the greatest increase in prescription drug expenditures, another area that has captured the attention of the public and of policy makers has been the sharp increase in the costs of some older generic drugs. In 2015, Turing Pharmaceuticals raised the price of pyrimethamine (Daraprim), a 63-year-old treatment for toxoplasmosis, by 5500%, from $13.50 to $750 a pill.22 The company was able to set the high price despite the absence of any patent protection because no other competing manufacturer was licensed to market the drug in the United States.  Significant increases in the prices of other older drugs include isoproterenol (2500%), nitroprusside (1700%), and digoxin (637%). Even though the prices of most generic drug products have remained stable between 2008 and 2015, those of almost 400
increased by more than 1000%.

Having made the above statements, it should be noted that there are a number of middle men before a patient’s prescription is received by the consumer. It is estimated that a 28% – 48% cost increase for a typical prescription, and that is where Amazon could come in.

PRICING THREAT:

Wells Fargo analyst David #Maris said Amazon may be the next threat to drug pricing as the company’s move into the prescription pharmacy space could result in increased competition and increased pricing transparency.

He added that a Wells Fargo survey of nearly 2,900 U.S. adults found that 54% of those polled said they would use or would probably use “Amazon Pharmacy,” indicating patients’ willingness to shift from local pharmacies. While the e-commerce giant has not confirmed its U.S. pharmacy interest, if it did enter the market Maris believes it could see fast adoption and “usher in a new age of price transparency.” He also wonders if pharmacy “may be just the beginning” and if Amazon eyes the “even larger prize” of fully integrated digital healthcare as telemedicine becomes more widely accepted. Maris said while little is known about Amazon’s plans, investors and companies should question the impacts of such a move now.

SIMPLY AMAZON‘: In addition, #Maxim analyst Tom Forte said that as consumers increasingly interact with Amazon in physical locations, “Amazon.com will become simply Amazon.” He analyzed 18 market opportunities, including 14 that Amazon is already pursuing and four – credit, gas stations, pharmacy, and travel – that are new. Among them, he identified ten categories where the global total addressable market exceeds $1T, which included the pharmacy space. Forte said he believes Amazon’s potential expansion into the pharmacy market could serve as a driver of sustained revenue growth and its delivery initiatives, such as Prime Now, make it well-positioned for entry. Given Amazon’s opportunity to drive incremental sales growth and “further disrupt the retail sector,” Forte raised his price target on AMZN to $1,300 from $1,075 and keeps a Buy rating on the name.

WHAT TO WATCH:

Publicly traded large-cap pharmaceuticals companies include AstraZeneca (AZN), Bristol-Myers (BMY), Eli Lilly (LLY), GlaxoSmithKline (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Publicly traded retail pharmacy operators include CVS Health (CVS), Walgreens (WBA), Fred’s (FRED) and Rite Aid (RAD).

PRICE ACTION: Amazon rose 0.1% to $1,012.35 in Tuesday trading. The stock is just dollars away from its recently-reached all-time high of $1,016.50.

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TG Therapeutics’ Leukemia Drug Proves Effective

Adding TG-1101 to ibrutinib increased the number of patients with bone marrow confirmed CR’s, MRD negativity

The combination was well tolerated; the addition of TG-1101 did not appear to alter the safety profile of ibrutinib monotherapy

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American Society of Clinical Oncology’s #ASCO Annual Meeting is taking place June 2 to June 6 in Chicago. Several pharmaceutical firms and biotechnology companies are presenting their new drugs and/or present progress on the status of their drugs under development. TG Therapeutics is one of those companies.

#TG Therapeutics $TGTX announced positive results from its Phase 3 $GENUINE trial of #TG-1101 plus ibrutinib in patients with previously treated high risk Chronic #Lymphocytic #Leukemia.
The trial was powered to show a statistically significant improvement in ORR of 30%, with a minimal absolute detectable difference between the two arms of approximately 20%. The trial met its primary endpoint, demonstrating a statistically significant improvement in Overall Response Rate, as assessed by blinded independent central radiology and hematology review by iwCLL criteria, compared to ibrutinib alone in both the Intent to Treat population and Treated population.
The combination was well tolerated and, apart from infusion related reactions, the addition of TG-1101 did not appear to alter the safety profile of ibrutinib monotherapy.
#Neutropenia, occurring in 9% of patients, was the most commonly reported Grade 3/4 Adverse Event in the combination arm, followed by infusion related reactions and #anemia, each reported in 5% of patients.
Neutropenia or #neutropaenia, is an abnormally low concentration of neutrophils (a type of white blood cell) in the blood. Neutrophils make up the majority of circulating white blood cells and serve as the primary defense against infections by destroying bacteria, bacterial fragments and immunoglobulin-bound viruses in the blood.  Patients with neutropenia are more susceptible to bacterial infections and, without prompt medical attention, the condition may become life-threatening.
Notably, the majority of infusion related reactions were Grade 1 or 2 in severity, with only 5% Grade 3/4 IRR observed. Median follow-up for this study was approximately 11.4 months.
In addition to the improvements in ORR, CR and MRD-negativity, a trend in improvement of Progression Free Survival was observed in the combination arm of TG-1101 plus ibrutinib as compared to ibrutinib alone.
“In addition to increasing the overall number of patients that responded to treatment with ibrutinib, adding TG-1101 to ibrutinib increased the number of patients with bone marrow confirmed CR’s, MRD negativity in peripheral blood, deepened nodal responses, and resulted in fewer patients progressing on therapy. Collectively, we see the consistent pattern of enhanced benefit as providing a compelling case for combining TG-1101 with ibrutinib in these hard to treat patients with high-risk CLL. We look forward to sharing these data with the FDA later this year to discuss filing for accelerated approval,” the company noted.

Other stocks to watch in the group: $INCY, $TSRO, $AZN, $AMGN

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Merck’s Breast Cancer Drug Proves Effective

The NYU’s Langone Medical Center announced that Merck’s pembrolizumab has been found to be effective in patients with metastatic triple negative breast cancer

The most common side effects in both patient populations were fatigue and nausea

 

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American Society of Clinical Oncology’s #ASCO Annual Meeting is taking place June 2 to June 6 in Chicago. Several pharmaceutical firms and biotechnology companies are presenting their new drugs and/or present progress on the status of their drugs under development. Merck is one of those companies.

The New York University’s Langone Medical Center announced that #Merck’s #pembrolizumab has been found to be effective in patients with metastatic triple negative breast cancer, according to an international clinical trial led by the center.

The trial investigated the drug in two separate cohorts of patients: Cohort A, which included 170 patients with heavily pretreated metastatic triple negative breast cancer regardless of PD-L1 expression, and Cohort B, which included 52 patients with PD-L1-positive tumors who received it as first-line therapy.

In #Cohort A, pembrolizumab shrunk tumors by more than 30 percent in eight of 170 patients, or five percent, and stabilized the disease in 35, or 21 percent, of those previously treated for mTNBC. Of the eight who experienced tumor reduction, all of them lived at least another year. The remaining patients in this cohort had a lower chance of survival.

In Cohort B-those who received pembrolizumab as first-line therapy-12 of 52 patients, or 23 percent, saw tumors shrink by more than 30 percent, while the disease was stabilized in nine of them, or 17 percent.

Study presenter Sylvia Adams pointed out that Cohort A is the first phase II study of an immunotherapy for triple negative breast cancer to be reported and represents the largest cohort of patients with mTNBC treated with immunotherapy to date.

“The goals of Cohort B, for which survival data are not yet complete, were, primarily, to prove pembrolizumab’s safety and, secondarily, to explore its efficacy as a first-line treatment. Both goals appear to have been met,” the center said.

Merck $MRK funded this clinical trial.

Pembrolizumab, marketed under the name #Keytruda, was well tolerated by both cohorts at a 200mg dose every three weeks, according to study results.

Only 12 percent of patients in Cohort A experienced severe side effects and only eight percent experienced them in Cohort B.

The most common side effects in both patient populations were fatigue and nausea. Although side effects led to discontinuation of treatment in seven patients from Cohort A, no patients in Cohort B discontinued treatment due to adverse side effects.

Other stocks to watch in the group: $INCY, $TSRO, $PBYI, $AMGN

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Alnylam’s Givosiran receives FDA’s Breakthrough Therapy

Porphyria is a group of diseases in which substances called porphyrins build up, affecting the skin or nervous system

Givosiran was found to be generally well tolerated with no drug-related serious adverse events

ALNY-LOGO

Alnylam Pharmaceuticals $ALNY announced that it has received Breakthrough Therapy designation from the U.S. Food and Drug Administration for #givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the prophylaxis of attacks in patients with acute hepatic porphyria.

“Promising results from the ongoing Phase 1 study of #givosiran demonstrating meaningful reductions in the occurrence of porphyria attacks formed the basis of the Breakthrough application,” the company says Updated results from this trial will be provided in an oral presentation on June 26 at the International Congress on Porphyrins and Porphyrias being held in Bordeaux, France.

The ongoing portion of the Phase 1 study of givosira is being conducted as a randomized, double-blind, placebo-controlled study.

Data presented at the 2016 American Society of Hematology meeting held in Atlanta demonstrated initial evidence for clinical activity with givosiran including meaningful reductions in both the number and frequency of porphyria attacks, as well as meaningful reductions in annualized hemin doses required in patients with acute intermittent porphyria, the most common and severe form of AHP.

In the first two dose cohorts, givosiran was found to be generally well tolerated with no drug-related serious adverse events. In the third dose cohort, which remains blinded, one death due to acute pancreatitis, considered unlikely related to givosiran or placebo, was reported after the data transfer date.

Porphyria is a group of diseases in which substances called porphyrins build up, affecting the skin or nervous system. The types that affect the nervous system are also known as acute #porphyria. Symptoms of acute porphyria include abdominal pain, chest pain, vomiting, confusion, constipation, fever, and seizures. These symptoms typically come and go with attacks that last for days to weeks. Attacks may be triggered by alcohol, smoking, stress, or certain medications. If the skin is affected, blisters or itching may occur with sunlight exposure.

The disease is usually inherited from a person’s parents and is due to a mutation in one of the genes that make heme. Some types are autosomal dominant and others are autosomal recessive. One type, porphyria cutanea tarda, may also be due to increased iron in the liver, hepatitis C, alcohol, or HIV/AIDS. The underlying mechanism results in a decrease in the amount of heme produced and a build-up of substances involved in making heme. Porphyrias may also be classified by whether the liver or the bone marrow is affected.  About 1 in 75,000 people have acute porphyria attacks. They may either have one of the acute porphyrias or they may have a mixed porphyria.

Separately,  the company announced that management will present a company overview at the #Jefferies 2017 Healthcare Conference on Tuesday, June 6, 2017 at 9:00 am ET in New York City.

PRICE ACTION:  ALNY closed at $64.09. The stock has a 52-week trading range of $31.38 – $80.11. Other stocks to watch: ICPT, AZN, INCY, BIIB.

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