|Bid||87.82 x 800|
|Ask||87.82 x 800|
|Day's Range||87.30 - 88.73|
|52 Week Range||62.88 - 100.13|
|Beta (5Y Monthly)||1.31|
|PE Ratio (TTM)||N/A|
|Earnings Date||Feb 18, 2020 - Feb 23, 2020|
|Forward Dividend & Yield||N/A (N/A)|
|1y Target Est||115.77|
A London-based company setting up a series of one-dose-and-done gene therapies will begin construction in the first half of this year of a nearly 200-employee manufacturing facility in Fremont. Orchard Therapeutics plc (NASDAQ: ORTX) hopes to open the 152,995-square-foot facility by the end of 2021 to make gene therapies and viral vectors, the shells of a deactivated virus that carry genes that correct a genetic mutation. Orchard already has a manufacturing support team in Menlo Park of between 80 and 90 people who will move to the new facility.
After a stock takes a tumble, wait just a minute before you count it out. While falling share prices could be a sign that a company is under duress, it doesn’t always mean that its growth story has come to an end. After all, a tried and true strategy to turn a profit in the market is to buy low and sell high.Wall Street pros remind investors to take this into consideration when gauging the strength of investments in the healthcare space. As these names rely on only a few key catalysts, all it takes is a single piece of bad news to send shares spiraling downwards. However, the opposite is also true, so one favorable outcome can put a stock right back on track.With this in mind, we used TipRanks’ Stock Screener tool to pinpoint 3 Buy-rated healthcare names that the analysts believe are on the mend after recent nose dives. Here’s the scoop straight from the analysts.BioMarin Pharmaceutical (BMRN) BioMarin has established itself as a key player in the biopharma space, with seven therapies already on the market and several other candidates in development. Despite falling 21% in the first nine months of 2019, SunTrust Robinson analyst Robyn Karnauskas believes that important upcoming catalysts could drive a rebound.The four-star analyst points out that the drop came largely as a result of declining investor confidence in the market potential of its Hemophilia therapy. ValRox, its Hemophilia A candidate progressing through Phase 2, has seen factor levels drop since the first year. However, Karnauskas remains convinced that BMRN can deliver.After conducting a survey to determine if the concerns related to commercialization were valid, the analyst concluded that ValRox could drive significant growth. “Our survey indicates high level of confidence in approval of ValRox, with 100% of doctors expecting approval and steady uptake in severe Hemophilia A patients… Our survey also indicated that doctors are likely to prescribe based on label and do not expect all severe patients to qualify,” she commented.As a result, the analyst believes, “At current values, the stock is trading with little credit to late stage pipeline assets with potential launches in late 2020/2021.” At the peak, Karnauskas estimates the Hemophilia A opportunity to be $1.48 billion.On top of this, its vosoritide therapy for achondroplasia could be a major component of the company’s next leg of growth, with it reporting positive Phase 3 data in December.Based on all of the above, Karnauskas tells investors that she’s siding with the bulls, reiterating a Buy rating on BMRN alongside a $110 price target. Should the target be met, shares will be in for a 24% twelve-month gain. (To watch Karnauskas’ track record, click here)Turning now to the rest of the Street, it appears that other analysts are generally on the same page. With 13 Buys and 3 Holds assigned in the last three months, the consensus rating comes in as a Strong Buy. In addition, the $111.71 average price target puts the upside potential just above Karnauskas’ forecast at 25%. (See BioMarin stock analysis on TipRanks)Alexion Pharmaceuticals (ALXN)Fighting rare diseases like generalized myasthenia gravis (gMG), neuromyelitis optica spectrum disorder (NMOSD) and ALS, Alexion Pharmaceuticals wants to meet the large unmet medical needs of patients globally. Even though the company took a beating in 2019, the Street isn’t giving up on ALXN just yet.While recent data for its candidate to treat PNH, a severe and ultra-rare blood disorder, raised some questions, Wedbush analyst Laura Chico believes its growth prospects are still strong. According to its most recent 8-K filing, total revenue growth is expected to beat the consensus estimate, and Ultomiris is poised to meet its conversion goals. As a result, Chico commented, “we continue to see ALXN as holding a strong competitive position in the complement markets.”It also doesn’t hurt that its neurology franchise is continuing to grow at a steady rate. Over the previous quarter, neurology patients on treatment (gMG/NMOSD) increased 23% to 1,885 patients. Despite the fact that growth slowed by about 5% compared to September/June, management told investors that they are hoping to quadruple their US neurology patient volume by FY25.All of this prompted Chico to conclude, “Overall, we see multiple encouraging updates from ALXN in their upcoming presentation not only on the commercial front, but with several pipeline updates as well. We continue to hold a positive bias on ALXN shares.”To this end, Chico decided to leave the Outperform rating and $149 price target unchanged. At this target, a 32% gain could be in the cards for ALXN over the next twelve months. (To watch Chico’s track record, click here)Like Chico, a majority of the analysts covering ALXN take a bullish approach. With 12 Buys and 3 Holds, the word on the Street is that ALXN is a Strong Buy. The healthcare stock boasts 29% upside potential based on the $145 average price target. (See Alexion stock analysis on TipRanks)Mylan N.V. (MYL)Mylan, one of the largest generic drug makers, has had a rough going, to say the least. Back in May, shares shed 24% of their value in a single day after the company failed to provide an update on the strategic review that had already lasted ten months. More recently, the stock has been range-bound thanks to investor concern regarding a possible cut to Viatris, the combination of Mylan and Pfizer’s Upjohn, as well as its proforma numbers. That being said, some members of the Street still have high hopes for MYL.RBC Capital’s Randall Stanicky thinks that several key factors suggest that MYL has more fuel left in the tank. “We believe buyside expectations are already low with Street proforma models stale while synergies provide support into 2022 stabilization; even on our lowered EBITDA, Newco trades at only 6.6x 2021E EBITDA (<7x even if further cutting EBITDA to $7 billion) while able to support a new dividend yielding about 4%,” he explained.Additionally, the analyst highlights improving corporate governance as well as argues that Viatris’ U.S. generic revenue and limited opioid exposure could help the company “de-couple from generic peers.” He added, “The announced combination of MYL and PFE’s Upjohn off-patent business will further grow and diversify MYL’s revenue base beyond US generics.”Given that it still boasts “one of the best generic/ biosimilar pipelines in the industry”, Stanicky’s bullish thesis remains firmly intact. With this in mind, the analyst maintained an Outperform call. Not to mention, he bumped up the price target from $25 to $27. The price target conveys the analyst’s confidence in MYL’s ability to surge 27% in the next twelve months. (To watch Stanicky’s track record, click here)Looking at the consensus breakdown, we have an almost even split. 4 Buys and 5 Holds received in the last three months add up to a Moderate Buy consensus rating. With a $24.50 average price target, the upside potential is 16%. (See Mylan price targets and analyst ratings on TipRanks)
BioMarin (BMRN) is set to initiate dosing in an early-stage clinical study in the first quarter to evaluate its gene therapy candidate, BMN 307, in phenylketonuria, a rare disease affecting the brain.
The CEO of a Cambridge biotech startup promised the company would “deliver in 2020” after announcing a second delay with its first-ever clinical trial.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in treatments for the rare disease Phenylketonuria (PKU) and in gene therapy clinical research, announced today that both the U.S. Food and Drug Administration (FDA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. have granted the Company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307. BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU. BMN 307 will be evaluated to determine whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will present at Goldman Sachs 12th Annual Healthcare CEOs Unscripted: A View from the Top on January 9, 2020 at 8:45am ET in New York. To access the live webcast, please visit the investor section of the BioMarin website, www.biomarin.com. A replay will also be archived on the site for at least one week following each event.
Today BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Invitae Corporation (NYSE: NVTA) announced that Biogen (NASDAQ: BIIB), Encoded Therapeutics, Neurogene, Praxis Precision Medicines and PTC Therapeutics joined Behind the Seizure®, an innovative, cross-company collaboration that aims to provide faster diagnosis for young children with epilepsy. The program will also be expanded to make no-charge testing available for healthcare providers to order for any child under the age of eight who has an unprovoked seizure.
2020 just kicked off, but investors aren’t wasting any time. Rolling up their sleeves and getting straight down to business, they know the race is on to find the stocks ready to speed past the rest in the year ahead. To spot these investment opportunities, Wall Street pros recommend taking a step back and focusing on the bigger picture, telling investors to pay close attention to both upside potential and what the analyst community has to say.With this strategy in mind, what’s the next step? For us, it was to take advantage of TipRanks’ investing tools. The platform’s Stock Screener tool let us filter our search results by analyst consensus, price target upside and Smart Score (TipRanks’ numerical score made up of 8 metrics to measure the strength of an investment).As a result, we were able to pinpoint 3 stocks flagged by the Street’s analysts as potential winners, with each boasting a “Strong Buy” consensus rating. Not to mention each has a top Smart Score, at least an 8 out of 10. Here’s the scoop.BioMarin Pharmaceutical (BMRN)With seven products currently on the market as well as an impressive development pipeline, BioMarin has cemented its status as a biopharma heavyweight. While 2019 saw the company falter, this could be BMRN’s year according to some members of the Street.Part of the excitement surrounding the company is based on the recent positive Phase 3 study data for its vosoritide therapy in achondroplasia, a disorder characterized by dwarfism, which was announced on December 16. During the study, the drug was able to meet the primary endpoint for growth velocity after one year, with it demonstrating an increase of 1.6 cm/year compared to the placebo. On top of this, vosoritide was generally well tolerated and the results were consistent across the study.According to Cantor analyst Eliana Merle, the news puts BMRN in a solid position going into 2020, noting, “We think this sets up for 2020-2021 to be a key inflection for BMRN with two regulatory submissions/potential product launches between vosoritide and valrox (hemophilia A gene therapy).”Should the drug receive approval, Merle estimates about $700 million in peak unadjusted sales. While pointing out that the question of reimbursement has been raised, she argues that the “clean data, coupled with the building body of evidence from long-term Ph2 data and younger patient studies will help support reimbursement discussions.” Bearing this in mind, the analyst left the Overweight rating and $129 price target unchanged. (To watch Merle’s track record, click here)Like Merle, Wedbush’s Liana Moussatos sees vosoritide approval as very likely. However, she attached a more aggressive price target along with her Outperform rating. Lifting the target by $1 to $158, the analyst thinks an 88% twelve-month gain could be in the cards. (To watch Moussatos’ track record, click here)In general, the rest of the Street appears to echo the analysts’ sentiment. With 13 Buys and 3 Holds assigned in the last three months, the verdict is that BMRN is a Strong Buy. Based on the $111.71 average price target, the upside potential lands at 33%, with the stock also sporting an 8 Smart Score. (See BioMarin stock analysis on TipRanks)GoDaddy Inc. (GDDY)GoDaddy provides an all-in-one solution with everything customers need to design the perfect website, from domains to hosting and online marketing. On the heels of its solid Q3 performance, some analysts are placing their bets on GDDY.During the quarter, the company impressed with its $851 million in gross bookings, surpassing the $828.7 million consensus estimate. Not only did the figure beat the estimate, but growth accelerated 180 basis points from the second quarter. Adding to the good news, its customer count increased by 140,000 sequentially and 4.6% year-over-year to reach 19.1 million. While uFCF came in below the estimate, one analyst tells investors that he remains fully onboard.Piper Jaffray’s Michael Olson cites GDDY’s “long-term growth and sticky revenue, along with opportunities for up/cross-selling and M&A” as putting it on the path to success. To this end, the five-star analyst decided to stay with the bulls, keeping the rating as Overweight along with a $91 price target, which leaves room for possible upside of 32%. (To watch Olson’s track record, click here)Meanwhile, Brent Thill of Jeffries cites his meeting with new CEO Aman Bhutani as the reason for his optimism. Calling the company one of his favorite mid-cap names, the analyst believes Bhutani’s “strong product background should help GDDY become a more comprehensive software provider with higher ARPU.” Additionally, he notes that the CEO won’t sacrifice margins even as the speed of product innovations accelerates. Following the meeting, Thill reiterated the Buy recommendation and $95 price target, implying 38% upside potential. (To watch Thill’s track record, click here)Looking at the consensus breakdown, it’s clear that the broader analyst community is on the same page. Out of 8 total analysts covering the stock, 100% were bullish, making the consensus a unanimous Strong Buy. If that wasn’t enough, GDDY boasts an 8 Smart Score and upside potential of 31% thanks to the $89.86 average price target. (See GoDaddy stock analysis on TipRanks)Mimecast Ltd. (MIME)In an age in which cyberattacks have become a very real and prevalent threat, Mimecast wants to protect customers by offering email security and compliance solutions through its integrated cloud-based platform. Following a strong 2019 performance, one analyst believes an encore could be in store.Berenberg Bank’s Joshua Tilton is singing MIME’s praises because of its “consistent execution and best-in-class product.” He argues that the company has been able to produce consistent results as its customer count grew by 800 new logos and its net retention rate stayed at 110%. In addition, he sees its new strategy as a key point of strength.“We believe the new security 3.0 strategy, with its zone-based approach, will help new mid-to-large organizations adopt more products faster. Current ARPU stands at $37. If this new approach can drive increased adoption of IEP, Awareness Training, and Web Security, we estimate the TAM will double for each of these new customers,” Tilton noted.MIME’s progress with regards to its enterprise segment also plays into the analyst’s bullish thesis. During its most recent quarter, 30 transactions were above six figures and average order value rose to 11,700, up 14% year-over-year. Tilton added, “Supported by a technologically superior offering, we expect MIME to continue its penetration of the enterprise base as management reallocates resources from the lower end of the market.”It’s no surprise, then, that Tilton reiterated the Buy rating and $59 price target. At this target, shares could climb 32% higher in the coming twelve months. (To watch Tilton’s track record, click here)What does the rest of the Street have to say? As it turns out, other analysts are in agreement. 7 Buys and a single Hold add up to a Strong Buy consensus rating. The $56.43 average price target puts the upside potential just below Tilton’s forecast at 26%. However, it should be noted that MIME earns a perfect 10 Smart Score. (See Mimecast stock analysis on TipRanks)
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Jean-Jacques Bienaimé, Chairman and Chief Executive Officer, will participate in the 38th Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2020 at 7:30 am PT, or 10:30 am ET, in San Francisco, California. To access the live webcast, please visit the investor section of the BioMarin website, www.biomarin.com. A replay will also be archived on the site for at least one week following the event.
While it may not be enough for some shareholders, we think it is good to see the BioMarin Pharmaceutical Inc...
The Business Times devoted our first print edition of the year to previewing the companies, people and trends to watch across the Bay Area's main industries in 2020. Here's a look at what to expect in biotech and health. People to Watch Jennifer Doudna, UC Berkeley: Will 2020 be the year when gene-editing CRISPR technology co-inventor Jennifer Doudna wins the Nobel Prize?
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the New England Journal of Medicine (NEJM) published an independently peer-reviewed article on up to three years of data from an ongoing Phase 1/2 study to evaluate safety and efficacy of investigational AAV gene therapy, valoctocogene roxaparvovec, for severe hemophilia A. This is the second article published by the NEJM on valoctocogene roxaparvovec.
Investing in hedge funds can bring large profits, but it’s not for everybody, since hedge funds are available only for high-net-worth individuals. They generate significant returns for investors to justify their large fees and they allocate a lot of time and employ complex research processes to determine the best stocks to invest in. A particularly […]
BioMarin (BMRN) has been upgraded to a Zacks Rank 1 (Strong Buy), reflecting growing optimism about the company's earnings prospects. This might drive the stock higher in the near term.
BioMarin (BMRN) submits a BLA in the United States for its investigational gene therapy valoctocogene roxaparvovec to treat adult patients with severe hemophilia A.
Approaching the end of long drug-development journeys, these three Bay Area companies formally asked the FDA to approve potential treatments for hemophilia A, anemia in chronic kidney disease patients and migraines.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A. Subject to completion of the FDA's filing review, BioMarin anticipates the BLA review to commence in February 2020. BioMarin will provide an update in February 2020.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The MAA review will commence in January 2020 under accelerated assessment.
Shares of BioMarin Pharmaceutical Inc. are up 2.8% in morning trading on Monday following positive clinical findings from a Phase 3 trial for achondroplasia. Achondroplasia is a rare disorder that causes dwarfism. The experimental drug, vosoritide, is being tested to help support skeletal growth in children aged 5 to 14 years old who have the disorder. Nomura analysts in November said they were concerned that vosoritide data for children between birth and 5 years old would not be available until 2020. BioMarin said it plans to meet with officials next year to discussion regulatory submissions. BioMarin's stock is down 5.85% year-to-date, compared to the S&P 500 , which is up 26%.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today reported positive final results from its randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide. The placebo-adjusted change from baseline in growth velocity after one year of treatment with vosoritide, the primary endpoint, was 1.6 cm/yr (p<0.0001). Vosoritide is an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP). The study enrolled 121 children aged 5 to 14 with achondroplasia, the most common form of disproportionate short stature. The results were consistent across the broad patient population studied. Vosoritide was generally well tolerated with no clinically significant blood pressure decreases. Based on these results, the Company plans to meet with health authorities in the first half of 2020 to discuss plans for submitting marketing applications.
The Zacks Analyst Blog Highlights: uniQure, Sarepta Therapeutics, BioMarin Pharmaceutical and Sangamo Therapeutics
Companies with gene therapy capabilities are becoming more attractive to members of Big Pharma, who are eager to add the technology to their portfolios Continue reading...
Gene therapy continues to draw attention with the recent spate of deals and acquisitions. We list four stocks with promising candidates in their pipelines that make attractive acquisition targets.