MyoKardia (NASDAQ: MYOK), a clinical-stage biotech, is having a tremendous Monday morning. Ahead of the opening bell, MyoKardia announced that its experimental obstructive hypertrophic cardiomyopathy drug mavacamten met all of its primary and secondary endpoints in a late-stage trial. Obstructive hypertrophic cardiomyopathy is a genetically based disorder characterized by abnormal thickening of the heart muscle, which can make it harder for the heart to pump blood.
MyoKardia, Inc. (MYOK) today provided an update to the status of its clinical programs in the context of the COVID-19 (coronavirus) pandemic, and specifically, the impact of the pandemic on the global healthcare system’s present ability to support the conduct of clinical trials. At this time, the company does not foresee changes to key clinical and regulatory milestones associated with the development of mavacamten for obstructive hypertrophic cardiomyopathy (HCM), including reporting topline data from the Phase 3 EXPLORER-HCM study in the second quarter of 2020. Enrollment in EXPLORER-HCM concluded in August 2019 and as of this month, all patient visits have been conducted through the 30-week treatment period.
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MyoKardia, Inc. (MYOK), a clinical-stage biopharmaceutical company discovering and developing targeted therapies for the treatment of serious cardiovascular diseases, today announced the pricing of an underwritten public offering of 5,250,000 shares of its common stock at a public offering price of $105.00 per share, before underwriting discounts. MyoKardia anticipates using net proceeds from the offering, together with its existing cash, cash equivalents and short-term and long-term investments, to support the regulatory approval process and potential commercial launch of mavacamten for the treatment of obstructive hypertrophic cardiomyopathy (HCM), if approved; to fund ongoing and potential later-stage clinical studies of mavacamten in non-obstructive HCM and targeted heart failure with preserved ejection fraction (HFpEF), danicamtiv in targeted segments of systolic heart failure and MYK-224; to advance ACT-1 and LUS-1 into clinical development; to fund ongoing preclinical, discovery and research programs; and for working capital, business development and other general corporate purposes.
BRISBANE, Calif., Feb. 27, 2020 -- MyoKardia, Inc. (Nasdaq: MYOK), today reported financial results for the fourth quarter and full year ended December 31, 2019. “MyoKardia.
MAVERICK-HCM Phase 2 Clinical Trial Results Consistent with Tolerability Observations from Prior Studies of Mavacamten Improvement in NT-proBNP and Troponin Levels Support.
MyoKardia, Inc. (MYOK) today announced positive topline data from the company’s Phase 3 pivotal EXPLORER-HCM clinical trial of mavacamten for the treatment of patients with symptomatic, obstructive hypertrophic cardiomyopathy (HCM) (clinicaltrials.gov NCT03470545). Mavacamten demonstrated a robust treatment effect: the primary and all secondary endpoints of the EXPLORER trial were met with statistical significance (p≤0.0006 for all endpoints).
This was a disappointing week for at least two major drug makers. French firm Genfit reported a trial failure in an intensely competitive indication, and New York-based Bristol Myers Squibb encountered a delay when the FDA declined to review its experiment…
Investors need to pay close attention to MyoKardia (MYOK) stock based on the movements in the options market lately.
With the outbreak of the coronavirus, volatility has become a seemingly ever-present facet of the stock market. Since the end of February, the major U.S. indexes have experienced violent swings in either direction, with these movements culminating in a circuit breaker being triggered on March 9 after the S&P 500 slipped by over 7%.While the current economic landscape has spurred fear among investors, others view market volatility as a possible avenue to huge rewards. Pointing to the healthcare sector specifically, those ready to take on some risk cite the potential returns that can be delivered should a name inhabiting this space release positive data or receive FDA approval. As healthcare companies generally rely on these few key indicators, the results can either send shares skyrocketing or down the drain.Bearing this in mind, we used TipRanks’ Stock Screener to identify three beaten-down biotech stocks that have significant catalysts coming up. The tool not only revealed that each of these Buy-rated tickers has attracted significant praise from analysts, all three also boast lofty upside potential. Here’s the scoop.MyoKardia Inc. (MYOK)This biotech name specializes in developing cutting-edge therapies for orphan cardiovascular diseases like hypertrophic obstructive and non-obstructive cardiomyopathy as well as dilated cardiomyopathy. Despite the year-to-date hit shares have taken, analysts are betting on MyoKardia ahead of an important data readout.During the second quarter of 2020, the company is expected to release data from the Phase 3 EXPLORER trial, which is evaluating its mavacamten therapy in patients with obstructive hypertrophic cardiomyopathy (oHCM). Management stated in its Q4 earnings call that 50% of oHCM patients are treated by 2,800 cardiologists, and the company is planning on targeting this physician group. Additionally, the HCM awareness campaign, which began in November, also stands to benefit MYOK by increasing awareness of the disease, with a new online resource being added for doctors this spring.For BMO Capital’s George Farmer, the previous mavacamten data is what has him excited about MYOK’s prospects. “We believe the primary endpoint will be met in EXPLORER for the following reasons: (1) the unprecedented efficacy profile with improved pVO2 and NYHA achieved with mavacamten in PIONEER sustained over 48 weeks; (2) the attractive mavacamten safety profile that emerged from the placebo-controlled nHCM MAVERICK trial (note here); and (3) the apparent utility of monitoring LVOT gradient in order to achieve optimal dosing in the oHCM treatment setting,” the analyst explained.While Farmer added that the impact of mavacamten on left ventricular ejection fraction (LVEF) has been a cause for concern, he argues that the trial’s design to include careful dose-titration could reduce some of the risk.To this end, the five-star analyst stays with the bulls, reiterating his Outperform rating. Along with the call, the assigned $90 price target implies that shares could climb 57% higher in the next year. (To watch Farmer’s track record, click here)What does the rest of the Street have to say? It turns out all 7 analysts that have published a recent review see the stock as a Buy, making the consensus rating a Strong Buy. At $85.83, the average price target implies 50% upside potential. (See MyoKardia stock analysis on TipRanks)Cara Therapeutics (CARA)By developing a class of peripherally acting kappa opioid agonist therapies, Cara Therapeutics is hoping to provide a more effective treatment option for chronic pruritis (itchy skin). While Cara shares tumbled nearly 17% year-to-date, some biotech pros see the upcoming top-line KALM-2 efficacy data release as being a major turning point.Back in October, the healthcare company increased the patient enrollment for the study by 20% to 430 patients at the request of an Independent Data Monitoring Committee (IDMC). KALM-2 is the second of two trials looking at the intravenous (IV) form of its CR845 drug in hemodialysis patients with chronic kidney disease (CKD)-associated pruritis, the results of which are slated for a Q2 2020 announcement.Weighing in on CARA for Piper Sandler, analyst David Amsellem explains that the increase in the number of patients was likely due to the fact that the KALM-2 study has more patient variability because its sites are located in multiple countries, while KALM-1 was only in the U.S. “Given that CR845 was highly statistically superior to placebo in the KALM-1 study (to the tune of a very strong p-value of .000019) and that KALM-2 has essentially the same design as KALM-1 (refer to our note on 10/14/19 for more details), we would be surprised to see results from KALM-2 diverge dramatically from KALM-1,” the analyst commented.On top of this, Amsellem also highlights the oral form of the candidate as setting the company up for serious growth. While oral CR845 failed to impress in its Phase 2 study as it missed on a responder analysis at week 12, a large and “well-powered” pivotal study could go a long way for CARA. With this in mind, Amsellem stated, “We continue to believe that both the IV and oral forms of CR845 will reach the market, driving a potentially $1 billion-plus franchise and hence transformative value creation.”In line with his optimistic approach, Amsellem left his Overweight call and $39 price target intact. Should this target be met, a twelve-month gain of 187% could be in the cards. (To watch Amsellem’s track record, click here)Judging by the consensus breakdown, the analyst communitity is on the same page. Given that 4 Buys have been issued in the last three months compared to no Holds or Sells, the message is clear: CARA is a Strong Buy. While less aggressive than Amsellem’s forecast, the $33.50 average price target still suggests 148% upside potential. (See Cara stock analysis on TipRanks)Eloxx Pharmaceuticals (ELOX)With the goal of treating rare and ultra-rare premature stop codon diseases, Eloxx has become one of the leaders in the eukaryotic ribosomal selective glycosides (ERSG) space. Even though shares suffered a beating after cystinosis data for its already approved Procysbi product was released last month, Janney analyst Yun Zhong sees a key upcoming catalyst related to its investigational candidate, ELX-02, as capable of driving a turnaround.Zhong points out that each of the three patients in the low-dose cohort from Procysbi's clinical study had gone through renal transplantation, with the result indicating that data interpretation from transplant patients could be difficult. In his opinion, this makes the recent sell-off of ELOX “overdone”. The analyst added, “We believe Eloxx will modify the protocol after the ongoing data review with cystinosis experts and we believe a higher dose and a younger patient population could lead to improved efficacy.”Additionally, Zhong thinks the top-line data readout for the Phase 2 study of ELX-02 in cystic fibrosis (CF) in first half of 2020 represents a significant catalyst, stating that he doesn’t believe there will be a readthrough from the Procysbi results. It should be noted that the CF program features two Phase 2 open-label studies located in the U.S. and Israel.With patient enrollment expected to wrap up in the first quarter of 2020, Zhong believes a positive outcome is likely. “Based on data from the Phase II study of Kalydeco in CF patients with the G551D mutation, we believe a reasonable expectation would be to see a meaningful change from baseline in sweat chloride, which will serve as the primary biomarker for ELX-02 dose selection for pivotal studies,” he commented.This reduction in sweat chloride is essential as it could allow the candidate to progress to a pivotal Phase. Taking all of this into consideration, Zhong recommends snapping up shares on recent weakness. Along with a Buy rating, he puts a $14 price target on the stock. This conveys his confidence in ELOX’s ability to soar 344% in the next twelve months. (To watch Zhong’s track record, click here)Turning now to the analyst community, a Moderate Buy consensus rating breaks down into 2 Buys and a single Hold. Given its $10.33 average price target, the upside potential comes in at 232%. (See Eloxx stock analysis on TipRanks)
High-security cryptographic key management for the hybrid-cloud user edlyn-teske Wed, 05/13/2020 - 23:02 When migrating business-critical applications and cryptography to the cloud, banks and financial institutions face a number of concerns. The push to adopt…
An experimental MyoKardia drug developed to treat an inherited cardiovascular condition that restricts blood flow is showing it can improve on the symptoms of this condition as well as the heart’s ability to function. MyoKardia (NASDAQ: MYOK) is testing it…
BRISBANE, Calif., March 19, 2020 -- MyoKardia, Inc. (Nasdaq: MYOK) today announced that the company will be presenting data from its Phase 2 MAVERICK-HCM clinical trial during.
MyoKardia (NASDAQ: MYOK) soared 96% this week after reporting its investigational heart drug met primary and all secondary endpoints in a phase 3 clinical trial. The clinical stage biotech company will submit mavacamten for the treatment of obstructive hypertrophic cardiomyopathy (HCM) to the U.S. Food and Drug Administration (FDA) for review early next year. MyoKardia has four other treatments for serious cardiovascular diseases in the pipeline, but mavacamten, as its most advanced candidate, likely will be the main catalyst for share movement for a while to come.
Biotech stocks have soared in 2020 on the back of promising COVID-19 vaccine or treatment data. But the trend for biotechs to surge dramatically is not one limited to only pandemic related stocks. Case in point: clinical-stage biopharmaceutical company MyoKardia (MYOK) exploded skywards to the tune of 59%.The surge followed an announcement that mavacamten, MyoKardia’s experimental obstructive hypertrophic cardiomyopathy (HCM) drug, met all of its primary and secondary endpoints in a Phase 3 clinical trial (EXPLORER-HCM).The promising data has excited both the cardiology community and Wall Street, as if approved, mavacamten could be the turning point for people at risk from the life-threatening heart condition and potentially very profitable for MyoKardia, too.With no effective therapies for HCM currently available, Cowen analyst Ritu Baral estimates mavacamten could reach “peak US and EU sales of $3.7 billion and $2.1 billion by 2028 and 2029, respectively.”Further data from the EXPLORER Phase 3 trial is expected in 2H20 and the company plans on submitting an NDA for FDA approval by 1Q21. If all runs smoothly, mavacamten could hit the market as early as 4Q21.Baral said, “We are thoroughly impressed with the totality (strength of benefit, consistency across endpoints, statistics, safety) of the Ph3 EXPLORER trial data. The composite endpoint was met with a clinically meaningful treatment absolute delta of 20% with more than twice as many placebo patients meeting the composite endpoint (a threshold important to our key opinion leaders)… We see the totality of the data as the best-case outcome for MYOK as they transition focus into launch and commercial efforts.”The 5-star analyst reiterated an Outperform rating on MYOK, while bumping her price target to $145 (from $104). The implication for investors? Upside of a further 30% from current levels. (To watch Baral’s track record, click here)Baral’s take gets the Street’s full backing. All 9 reviews published during the last 3 months recommend MYOK a Buy. Yesterday’s surge, though, appears to have taken the analyst community by surprise, as the average price target is $90.22, and implies a drop of 7% from current levels. Expect some Wall Street readjustments to MyoKardia models shortly. (See MyoKardia stock analysis on TipRanks)Read more: * 3 Biotech Stocks Under $3 With 200%-Plus Upside Potential * 3 Stocks Needham’s Top Analysts Are Raving About
Aiming to ask regulators early next year to approve its treatment for a potentially fatal heart condition, a Peninsula company's stock jumped more than 70% today on news that its drug passed a critical late-stage clinical trial. MyoKardia Inc. (NASDAQ: MYOK)…
We hate to say this but, we told you so. On February 27th we published an article with the title Recession is Imminent: We Need A Travel Ban NOW and predicted a US recession when the S&P 500 Index was trading at the 3150 level. We also told you to short the market and buy […]
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Q4 2019 MyoKardia Inc Earnings Call
MyoKardia shares made an incredible gain on Monday after the company announced positive results from its late-stage pivotal clinical trial of mavacamten.
Investors need to pay close attention to MyoKardia (MYOK) stock based on the movements in the options market lately.
(Bloomberg) -- MyoKardia Inc. jumped on Monday after its experimental treatment for a rare heart condition met its primary goal and all secondary targets in a key study, delivering a win for the now $4.7 billion drugmaker.The late-stage trial, dubbed “Explorer,” showed patients with hypertrophic cardiomyopathy -- a condition where the heart muscle becomes abnormally thick -- who received mavacamten saw a significant benefit over those who got a placebo. The primary endpoint of the study was a composite functional analysis testing the effect of 30 weeks of treatment on both symptoms and cardiac function. Secondary endpoints included a bevy of scores detailing heart functionality.Shares of the South San Francisco, California-based company spiked as much as 71% to an all-time high in New York. MyoKardia stock has more than doubled in the past 12 months even after falling 16% this year through Friday’s close. The closely watched Nasdaq Biotechnology Index, a barometer for investor sentiment in the drug-development space, is up 30% in the past 12 months and traded at the highest level since July 2015.Mavacamten was well tolerated with safety comparable to placebo and consistent with previous studies. Across both the mavacamten and placebo groups, 2% of study participants discontinued treatment, and severe side effects and cardiovascular events were similar in both groups.The drug’s safety has been key for investors. Analysts had expected promising results could lead to more than $3 billion in annual sales by 2027. While the initial data for the trial show promise, investors will want to see more detailed results from the pivotal program. MyoKardia will submit the data to a professional meeting this year and expects to file a marketing application with U.S. regulators in the first three months of 2021.Cytokinetics Inc., a company studying its Amgen Inc.-partnered drug for chronic heart failure, soared as much as 25% to a decade-high. The duo announced Friday morning that U.S. regulators granted Fast Track designation for the medicine ahead of late-stage results expected later this year.“Overall, these data hit our home-run scenario” as the safety profile of mavacamten came in better than expected, wrote JPMorgan analyst Anupam Rama.(Updates with share movement throughout, adds Cytokinetics move in the sixth paragraph and JPMorgan commentary in seventh.)For more articles like this, please visit us at bloomberg.comSubscribe now to stay ahead with the most trusted business news source.©2020 Bloomberg L.P.
Aiming to ask regulators early next year to approve its treatment for a potentially fatal heart condition, a Peninsula company's stock jumped more than 70% today on news that its drug passed a critical late-stage clinical trial. MyoKardia Inc. (NASDAQ: MYOK)…
Here's a roundup of top developments in the biotech space over the last 24 hours.Scaling The Peaks (Biotech Stocks Hitting 52-week Highs May 14) * Allogene Therapeutics Inc (NASDAQ: ALLO)(reacted to ASCO abstract) * Altimmune Inc (NASDAQ: ALT)(announced first…
BRISBANE, Calif., May 06, 2020 -- MyoKardia, Inc. (Nasdaq: MYOK), today reported financial results for the first quarter ended March 31, 2020. “Our team has continued to drive.
Q1 2019 MyoKardia Inc Earnings Call
MyoKardia, Inc. (MYOK), a clinical-stage biopharmaceutical company discovering and developing targeted therapies for the treatment of serious cardiovascular diseases, today announced that it has commenced a proposed underwritten public offering to sell $450.0 million in shares of its common stock. MyoKardia expects to grant the underwriters a 30-day option to purchase up to $67.5 million in additional shares of common stock at the public offering price, less the underwriting discount. All shares of common stock will be offered by MyoKardia.
Companies In The News Are: