Below are the top biotechnology stocks to watch over the coming days and weeks. Keep in mind that these stocks do NOT meet the stringent requirements for stock picks in the Premium service.
Biotech stocks and IBB is lagging behind the S&P 500 which means there’s more value to be found in IBB right now.
Biotech stocks overshot the performance of the S&P 500 in early 2019 but have been consolidating since mid-January while the S&P 500 has continued to rise.
If we go back to the December 24, 2018 low, biotech stocks are leading the S&P 500.
Top 6 Biotech Stocks To Watch
Back on December 26, 2018, Acer Therapeutics announced that the FDA had accepted for review Acer’s New Drug Application (NDA) for EDSIVO™ for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. The FDA also granted a priority review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. Priority review is a designation granted by the FDA to accelerate the review process for drugs that offer a significant improvement in treatment or provide treatment where no satisfactory alternative therapy exists.
“The acceptance of our NDA for EDSIVO™ is an important step in our efforts to help patients with vEDS, who suffer with a devastating disease that currently has no approved treatment,” said William Andrews, M.D., FACP, Chief Medical Officer of Acer. “We have had the honor of learning about the significant challenges of living with vEDS directly from patients and their families. This has in large part driven the hard work, passion and complete dedication that our small team has given to this effort, and we will continue to do so as the FDA reviews our NDA for EDSIVO™. We are excited about the possibility of making EDSIVO™ available in the U.S. for patients in the near future.”
“We continue to accelerate our pre-commercial activities supporting the potential U.S. launch of EDSIVO™ for the treatment of vEDS if it is approved by the FDA,” said Chris Schelling, CEO and Founder of Acer. “Additionally, we are working diligently on advancing and expanding our pipeline with the goal of bringing multiple products to patients with serious rare diseases over the next several years.”
Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. vEDS is the most severe subtype where patients suffer from life threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. The average mortality is 51 years of age. An Acer-commissioned patient-finder study phenotypically identified 4,169 vEDS patients in the U.S. from an analysis of a commercially available patient claims database with data of approximately 190 million unique patient lives. Based on that information, Acer estimates the prevalence of phenotypically-defined vEDS in the U.S. could be greater than 1 in 45,000. Currently, there are no FDA-approved therapies for vEDS. Acer is advancing EDSIVO™ (celiprolol), a new chemical entity (NCE), for the treatment of vEDS based on a randomized controlled clinical study of celiprolol(1). FDA granted a priority review of the EDSIVO™ NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. EDSIVO™ received FDA Orphan Drug Designation for the potential treatment of vEDS in 2015.
ACER stock does not present a decent entry at the moment. Prices have been extended too far to the upside lately. For a good entry it is better to wait for a consolidation.
Vyleesi (Bremelanotide) for female sexual dysfunction (FSD). The PDUFA date is extended to June 23, 2019.
“During 2018, we increased our financial guidance three times and today announced that we achieved our financial objectives for the full year of 2018,” said Ted Myles, AMAG’s chief financial officer. “We also completed the transformation of our balance sheet, and with nearly $400 million of cash on hand and approximately $20 million in near-term debt, we are well positioned to advance our portfolio of novel products.”
“2019 will be a year of investment and we are in the fortunate position that our cash flow generating products and our balance sheet can support the development of our future products; each of which has significant revenue and growth prospects because they provide innovative therapeutic solutions to patients with unmet medical needs,” added Mr. Myles.
AMAG stock presents an ok setup. Prices have been consolidating lately. There is a resistance zone just above the current price starting at $12.65. Right above this resistance zone may be a good entry.
BHV-0223 for Amyotrophic lateral sclerosis (ALS). NDA acceptance announced November 25, 2018 but a PDUFA date was not given. The estimated PDUFA date is sometime in July 2019.
Back on November 26, 2018, Biohaven Pharmaceutical announced that its 505(b)(2) new drug application (NDA) for BHV-0223, an investigational sublingual form of riluzole for the treatment of people living with amyotrophic lateral sclerosis (ALS), had been accepted for review by the FDA. Biohaven previously announced that the FDA granted orphan drug designation for BHV-0223 in ALS. BHV-0223 is a novel, lower dose formulation of riluzole designed to be placed under the tongue where it dissolves in seconds and is absorbed sublingually. The active ingredient, riluzole, is the only FDA-approved treatment to extend tracheostomy-free survival in people living with ALS.
“We are extremely proud to announce the FDA acceptance for review of our 505(b)(2) NDA submission of BHV-0223, as it represents Biohaven’s first NDA. If approved, we believe BHV-0223 would provide an important advancement in treatment for people living with ALS, including the large number of patients who have difficulty swallowing tablets or liquids,” said Robert Berman, M.D., Chief Medical Officer of Biohaven. “We are committed to designing new treatment options for life-altering neurological diseases and believe that our sublingual, orally-dissolving tablet formulation of riluzole can provide a meaningful alternative for people living with ALS.”
ALS is a progressive neurodegenerative motor neuron disease affecting nerve cells in the brain and the spinal cord in which patients experience muscle weakness, trouble swallowing and muscle atrophy that ultimately progresses to paralysis, impaired breathing and death. More than 80% of people living with ALS experience dysphagia, which is difficulty swallowing, during their disease course.
The 505(b)(2) NDA submission is supported by data from multiple studies and clinical trials evaluating the bioequivalence of BHV-0223 to riluzole oral tablets (Rilutek®), as well as the safety and tolerability of BHV-0223. Key findings from the clinical program showed that sublingual administration of BHV-0223 (40 mg) achieved similar blood exposures to orally ingested Rilutek (50 mg). In these studies and trials, BHV-0223 was generally well tolerated. Patients also reported the sublingual formulation easy to use in clinical trials in which BHV-0223 was administered to patients with ALS and dysphagia.
BHV-0223 is a sublingually administered, orally dissolving tablet (ODT) that makes use of the unique Zydis® ODT fast-dissolve technology. It is being developed under an exclusive worldwide agreement with Catalent. While riluzole is FDA-approved for ALS, existing formulations consist only of an oral tablet taken with water or an oral suspension (thickened liquid). BHV-0223 is designed to be placed under the tongue where it dissolves in seconds and is absorbed by the vasculature, thereby eliminating the need for swallowing.
ALS is a progressive, neurodegenerative motor neuron disease that affects nerve cells in the brain and the spinal cord. The disease belongs to a group of disorders known as motor neuron diseases, which are characterized by the gradual degeneration and death of motor neurons. ALS affects approximately 20,000 individuals in the United States. Since its FDA’s approval in 1995, riluzole remains the only approved drug demonstrated to enhance survival and/or time to tracheostomy.
More than 80% of people living with ALS experience dysphagia at some point in their disease course. Even mild or moderate dysphagia can lead to difficulty managing small oral boluses such as tablets through the swallowing process. The impaired ability to swallow safely puts people with ALS at risk of aspiration and its potentially life-threatening consequences such as aspiration pneumonia.
BHVN stock does not present a decent entry opportunity at the moment. Rumors circulated on Friday that the company is up for sale and a potential suitor has emerged. Prices have been extended too far to the upside. For a nice entry it is better to wait for a consolidation which likely isn’t coming because of the buyout rumors circulating.
HTX-011 for post operative pain. The PDUFA date under priority review is set for April 30, 2019.
Back on December 31, 2018, Heron Therapeutics announced that the FDA has accepted the new drug application (NDA) for Heron’s investigational agent, HTX-011, and has granted it a Priority Review designation. HTX-011 is a long-acting, extended-release formulation of the local anesthetic bupivacaine in a fixed-dose combination with the anti-inflammatory meloxicam for the management of postoperative pain. HTX-011 is the first and only dual-action fixed-combination product specifically designed to address both postoperative pain and inflammation in a single administration at the surgical site. The NDA for HTX-011, which was submitted on October 30, 2018, comprises data from five Phase 2 clinical trials and two Phase 3 clinical trials that included over 1,000 patients undergoing five different surgical procedures. The FDA also indicated that it is not currently planning to hold an advisory committee meeting to discuss this application. The FDA set a Prescription Drug User Fee Act goal date of April 30, 2019.
“We are pleased to receive Priority Review designation for the HTX-011 NDA,” said Barry D. Quart, Pharm.D., Chief Executive Officer of Heron. “We believe that HTX-011 could have a considerable impact on the lives of patients by significantly reducing the proportion of patients who experience severe pain and receive opioids after surgery, especially at discharge. We look forward to continuing to work closely with the FDA during the review process with the goal of bringing this important product to patients in 2019.”
The FDA had previously granted Breakthrough Therapy designation for HTX-011 based on the results of Phase 2 studies and two completed Phase 3 studies, which showed that HTX-011 produced significant reductions in both pain intensity and the need for opioids through 72 hours post-surgery compared to placebo and bupivacaine solution, the standard-of-care. The FDA has now granted Priority Review designation to the NDA for HTX-011. Priority Review designation is for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment or prevention of serious conditions. HTX-011 is the first and only non-opioid, long acting local anesthetic to demonstrate in Phase 3 studies a statistically significant reduction in severe pain and an increase in the number of patients who require no opioids for 72 hours postoperatively versus bupivacaine solution, the standard-of-care. The overall safety profile of HTX-011, administered locally into the surgical site without a needle, was similar to that of the well-established safety profile of bupivacaine solution, without evidence of meloxicam-related toxicities.
“Despite ongoing efforts to prevent opioid abuse, patients continue to receive large quantities of opioids for postsurgical pain,” said Jay Redan, M.D., FACS, Medical Director of Minimally-Invasive General Surgery at Florida Hospital Celebration Health. “There is a significant need for safe, effective and non-addictive options that can decrease opioid exposure and improve the patient recovery experience, as well as make an impact on the opioid epidemic by significantly reducing the amount of opioids necessary to take home for pain management.”
HTX-011, which utilizes Heron’s proprietary Biochronomer® drug delivery technology, is an investigational, long-acting, extended-release formulation of the local anesthetic bupivacaine in a fixed-dose combination with the anti-inflammatory meloxicam for the management of postoperative pain. By delivering sustained levels of both a potent anesthetic and a local anti-inflammatory agent directly to the site of tissue injury, HTX-011 was designed to deliver superior pain relief while reducing the need for systemically administered pain medications such as opioids, which carry the risk of harmful side effects, abuse and addiction. HTX-011 has been shown to reduce pain significantly better than placebo or bupivacaine alone in five diverse surgical models: hernia repair, abdominoplasty, bunionectomy, total knee arthroplasty and breast augmentation. HTX-011 was granted Fast Track designation from the FDA in the fourth quarter of 2017 and Breakthrough Therapy designation in the second quarter of 2018. Heron submitted an NDA to the FDA for HTX-011 in October of 2018 and received Priority Review designation in December 2018. The FDA set a Prescription Drug User Fee Act goal date of April 30, 2019.
HRTX presents an ok setup opportunity. We see reduced volatility while prices have been consolidating in the most recent period. There is a resistance zone just above the current price starting at $25.44. Right above this resistance zone may be a good entry point. There is a support zone below the current price at $24.20, a stop order could be placed below this zone.
Contepo for complicated urinary tract infections (cUTI). The PDUFA date under priority review is set for April 30, 2019.
Back on January 14, 2019, Nabriva Therapeutics announced that the FDA had updated the PDUFA (Prescription Drug User Fee Act) goal date for the completion of the FDA’s review of the CONTEPO New Drug Application (NDA) from June 30, 2019 to April 30, 2019. The two-month acceleration is due to a clarification of the classification and subsequent expedited review period for the CONTEPO NDA submitted in October 2018. In addition to priority review, CONTEPO has been granted Qualified Infectious Disease Product (QIDP) and Fast Track designations by the FDA for the treatment of several serious infections, including cUTI.
Urinary tract infections (UTIs) are a significant health problem in both the community- and hospital-based treatment settings. It is estimated that 150 million UTIs occur yearly worldwide, accounting for $6 billion in health care expenditures, according to the American Urological Association. Patients with complicated urinary tract infections (cUTIs), which includes acute pyelonephritis (kidney infection), have pyuria and bacteria in their urine, in association with features such as fever, chills, malaise or flank/back pain, in the setting of a functional or anatomical abnormality of the urinary tract or a history of catheterization. Patients who fail to respond to an initial course of antibiotics may develop a cUTI, which occurs when the bacteria are embedded in the bladder wall or ascend to the kidneys, where they can multiply more slowly and are much harder to address with antibiotics. In most cases, cUTIs occur following treatment for a normal UTI because antibiotics were given too late, for too short a period of time, at too low of a dose course or the wrong antibiotic was used and did not provide adequate spectrum of coverage. An estimated three million cases of cUTIs are treated in the hospital setting in the United States each year for Gram-negative infections. Enterobacteriaceae are the most common pathogens causing cUTIs and, currently, widespread antibiotic resistance limits the effective treatment options for cUTI. Ineffectively managed cUTI can lead to increased treatment failure rates, recurrence of infection, increased re-hospitalization, and increased morbidity and mortality.
CONTEPO™ (fosfomycin for injection, previously referred to as ZTI-01 and ZOLYD) is a novel, potentially first-in-class in the United States, intravenous investigational antibiotic with a broad spectrum of Gram-negative and Gram-positive activity, including activity against most contemporary multi-drug resistant (MDR) strains such as ESBL-producing Enterobacteriaceae. Intravenous (I.V.) fosfomycin has been approved for a number of indications and utilized for over 45 years in Europe to treat a variety of infections, including cUTIs and other serious bacterial infections. CONTEPO utilizes a new dosing approach, originally developed by Zavante (which Nabriva Therapeutics acquired), to optimize its pharmacokinetics and pharmacodynamics. Nabriva Therapeutics believes these attributes, along with the positive clinical experience worldwide, support CONTEPO as a first-line treatment for cUTIs, including acute pyelonephritis, suspected to be caused by MDR pathogens. At least 20 percent of cUTIs are caused by MDR bacteria and limited treatment options are available in the U.S. In addition, non-clinical data have shown that CONTEPO acts in combination with certain other antibiotics to improve bacterial killing.
NBRV does present a nice setup opportunity. Prices have been consolidating lately. A pullback is taking place, which may present a nice opportunity for an entry. There is a very little resistance above the current price.
New formulation of Thiola (tiopronin) for cystinuria. The PDUFA date is set for June 30, 2019.
Back on November 12, 2018, Retrophin announced that the FDA had accepted for review the New Drug Application (NDA) for a new formulation of Thiola (tiopronin) in the treatment of cystinuria. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2019.
“We know that living with a rare kidney disorder like cystinuria can be a challenge so we have worked with the patient, caregiver and medical communities over the past several years to identify ways we can further enhance Thiola,” said Stephen Aselage, chief executive officer of Retrophin. “The acceptance of this NDA for review is an important milestone as we continue efforts to fulfill our ongoing commitment to support patients with cystinuria and deliver a new, more patient-friendly formulation of Thiola.”
The NDA was filed by the Company’s partner, Mission Pharmacal Company, through the 505(b)(2) regulatory pathway which allows the FDA to reference previous findings of safety and efficacy for an already-approved product, in addition to reviewing findings from further studies of the product.
Thiola (tiopronin) tablets are indicated for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria with urinary cystine >500 mg/day, who are resistant to treatment with conservative measures of high fluid intake, alkali and diet modification, or who have adverse reactions to d-penicillamine.
RTRX does present a good setup opportunity. Prices have been consolidating lately and the volatility has been reduced. There is a resistance zone just above the current price starting at $22.11. Right above this resistance zone may be a good entry point.
Disclosure: I do not hold any position in any stocks mentioned in this article.