Vyjuvek is designed to treat the genetic root cause of DEB
Krystal Biotech (KRYS) announced the U.S. Food and Drug Administration has approved Vyjuvek for the treatment of patients six months of age or older with dystrophic epidermolysis bullosa, or DEB.
DEB is a rare disease that appears at birth or during the first few years of life, and lasts a lifetime. Prognosis is variable, but tends to be serious. Life expectancy is 50 years, and the disease brings with it complications related to infections, nutrition and neoplastic complications.
“Vyjuvek is designed to address the genetic root cause of DEB by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional COL7 protein expression with redosing.
Vyjuvek is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for the treatment of DEB, both recessive and dominant, that can be administered by a healthcare professional in either a healthcare professional setting or in the home,” the company stated.
With this approval, the FDA issued the Company a Rare Pediatric Disease Priority Review Voucher, or PRV, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review, the company noted.
“Vyjuvek is expected to be available in the United States in the third quarter of 2023, and the company will begin the promotion of Vyjuvek immediately.
Outside of the US, the European Medicines Agency has granted Vyjuvek orphan drug designation and PRIME – PRIority MEdicines – eligibility for the treatment of DEB.
The Company anticipates starting the official Marketing Authorization Application procedure in the second half of 2023 with a potential approval in 2024.
The Company is also working with the Pharmaceuticals and Medical Devices Agency in Japan to study Vyjuvek and seek approval for potential launch in 2025,” the company added.
KRYS is up 8.7% to $95.00 per share on heavy trading volume.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
bluebird bio (BLUE) announced the U.S. Food and Drug Administration has approved Zynteglo, also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta-thalassemia in adult and pediatric patients who require regular red blood cell transfusions.
“Due to the complex nature of gene therapy, Zynteglo will be available exclusively at Qualified Treatment Centers, which are carefully selected based on their expertise in relevant areas such as stem cell transplantation, cell and gene therapy, and beta-thalassemia; and receive specialized training to administer Zynteglo.
Information on bluebird’s QTC network, as well as personalized support focused on the needs of each patient throughout their treatment journey and information on insurance coverage and access will be available through bluebird’s patient support program,” the company stated.
“The FDA approval of Zynteglo offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation, and unlocks new possibilities in their daily lives.
Current thalassemia treatment
After more than a decade of research and clinical development, and through the perseverance of clinicians, patients, and their families, the approval of Zynteglo marks a watershed moment for the field of gene therapy.
As the first ex-vivo lentiviral vector gene therapy approved in the U.S. for the treatment of people with beta-thalassemia, we are ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care,” said Andrew Obenshain, CEO of bluebird bio.
Expensive Treatment
The lifetime cost of medical care for a patient with transfusion-dependent beta-thalassemia can reach up to $6.4M in the U.S. and the average total health care cost per patient per year is 23 times higher than the general population. bluebird estimates that there are approximately 1,300-1,500 individuals with transfusion-dependent beta-thalassemia in the U.S.
” bluebird has set the wholesale acquisition cost of Zynteglo in the U.S. at $2.8M, in “recognition of its robust and sustained clinical benefit demonstrated in clinical studies and its potential to alleviate a lifetime of health care costs associated with regular RBC transfusions and iron management.” Tom Klima, chief commercial and operating officer of bluebird bio, said.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Jazz Pharmaceuticals announces $5B revenue target for 2025
Jazz Pharmaceuticals (JAZZ) announced its Vision 2025 to deliver sustainable growth and enhanced value.
Vision 2025 includes the following expectations: 1) generating $5B in revenue in 2025; 2) approval of at least five additional novel products by the end of the decade; and 3) realizing a 5% adjusted operating margin improvement from 2021 to 2025, driven by operational excellence.
The company also confirmed that it expects to meet its previously announced 2021 revenue guidance range of $3.02B-$3.1B and its net product sales guidance for neuroscience and oncology.
Jazz ended 2021 demonstrating executional excellence across its business, including launching five key products in 2020 and 2021, integrating the GW Pharmaceuticals business, making progress towards its deleveraging target and initiating multiple potentially registrational clinical trials.
The company remains on track to deliver revenue diversification, with at least 65% of 2022 net product revenue from newly launched or acquired products, driving sustainable growth and enhanced shareholder value.
“Building on our track record of strong execution and guided by our patient-centric approach, Jazz is setting forth its Vision 2025 to deliver meaningful treatment options to patients, a great place to work for employees and significant value to shareholders,” said Bruce Cozadd, chairman and CEO of Jazz Pharmaceuticals.
“Jazz’s leadership in sleep and rare epilepsy with Xywav and Epidiolex, respectively, coupled with promising new oncology products like Zepzelca and Rylaze, have led to the rapid transformation of our revenue base.
We are further poised to enter new disease areas with serious unmet patient need and substantial market potential, including movement disorders and PTSD, with our mid-to late-stage assets nabiximols, suvecaltamide and JZP150.
We expect our continued operational excellence to drive a five-percentage point improvement in our adjusted operating margin from 2021 to 2025, and we are confident in our ability to continue to leverage strategic capital allocation to grow our business.”
In Monday’s trading shares of JAZZ are up $10.49 to $144.91.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Aytu, Neos Therapeutics enter all-stock merger agreement
Aytu BioScience (AYTU) and Neos Therapeutics (NEOS) announced that they have entered into a definitive merger agreement pursuant to which Neos will merge with a wholly owned subsidiary of Aytu in an all-stock transaction.
Upon the effectiveness of the merger, Neos stockholders will be entitled to receive 0.1088 shares of common stock of Aytu for each share of Neos common stock held, after taking into account the one-for-ten reverse split of Aytu’s common stock that was effected on December 8.
The transaction will result in Neos stockholders owning approximately 30% of the fully diluted common shares of Aytu.
The all-stock transaction is valued, on a fully diluted basis, at approximately $44.9M based on the 10-day volume weighted average price of Aytu stock for the period ended December 9.
The combined entity will have an increased footprint in the prescription pediatric market, an established multi-brand ADHD portfolio addressing the $8.5B ADHD market and combined revenue scale.
For the 12-month period ending September 30, Neos generated $57M in revenues. On a combined pro-forma basis for this same period, Aytu and Neos’ aggregate net revenue is over $100M.
In addition, this merger facilitates operational and commercial synergies that can be harnessed to accelerate the path to profitability for the combined entity, with estimated annualized cost synergies of approximately $15M beginning FY22.
The combined company will be led by Josh Disbrow, CEO of Aytu and will be headquartered in Englewood, Colorado.
The board of the combined company will consist of six members designated by Aytu and two members designated by Neos, including Neos CEO and director Jerry McLaughlin and Neos director Beth Hecht.
The merger is currently expected to close by Q2 of 2021, subject to certain approvals by both Aytu and Neos stockholders and the satisfaction of other customary closing conditions.
As part of the transaction, Aytu has agreed to provide Neos with access to up to $5M cash for working capital needs for the period prior to the closing of the merger.
In addition, upon closing of the merger, $15M in principal of Neos’s existing senior secured debt facility with affiliates of Deerfield Management will be repaid, and Deerfield has agreed to allow the remaining debt under the facility to remain outstanding with the combined company following the merger.
Indebtedness under Neos’s existing ABL agreement with Encina Business Credit will also remain outstanding.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Bristol-Myers to acquire MyoKardia for $225.00 per share in cash
Bristol-Myers (BMY) will buy MyoKardia (MYOK) for $225 a share in cash, or $13.1B. MyoKardia’s lead pipeline drug, code-named mavacamten, treats a chronic heart condition that can cause irregular heart rhythms in some patients and even death. Bristol plans to ask U.S. health regulators next year to approve the drug, Bristol CEO Giovanni Caforio says.
MYOK sold for $13.1B
The transaction was unanimously approved by both the Bristol Myers Squibb and MyoKardia Boards of Directors and is anticipated to close during the fourth quarter. Bristol Myers Squibb expects to finance the acquisition with a combination of cash and debt.
The transaction is expected to add a significant growth driver during the medium- to long-term.
Bristol-Myers goes shopping, Stockwinners
It is expected to be minimally dilutive to Bristol Myers Squibb’s non-GAAP EPS in 2021 and 2022 and accretive beginning in 2023. Bristol Myers Squibb reaffirms its existing 2021 non-GAAP EPS guidance range.
MyoKardia, Inc. discovers, develops, and commercializes targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. Its lead product candidate is mavacamten, an orally administered small molecule, which is in Phase III clinical trial that is designed to reduce left ventricular contractility to alleviate the functional consequences and symptoms of obstructive hypertrophic cardiomyopathy (HCM) and prevent or reverse HCM progression, as well as in Phase II clinical trial for non-obstructive HCM.
The company also develops MYK-491, an orally-administered small molecule, which is in Phase IIa clinical trial that is designed to restore normal cardiac muscle contractility in the diseased dilated cardiomyopathy (DCM) heart. Its preclinical programs include MYK-224, a HCM-targeting candidate that is designed to reduce excess cardiac contractility and enhance diastolic function; LUS-1, which is used to counteract a muscle abnormality that results in impaired relaxation of the left ventricle; and ACT-1 targeting genetic DCM due to sarcomeric mutations and impaired calcium regulation.
MYOK closed at $139.60, it last traded at $221.00. BMY closed at $58.72.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Johnson & Johnson to acquire Momenta Pharmaceuticals for $52.50/shr
Johnson & Johnson (JNJ) announced it has entered into a definitive agreement to acquire Momenta Pharmaceuticals (MNTA) in an all cash transaction for approximately $6.5 billion.
The transaction will include full global rights to nipocalimab (M281), a clinically validated, potentially best-in-class anti-FcRn antibody.
Momenta sold for $6.5B, Stockwinners
Nipocalimab gives Janssen the “opportunity to reach significantly more patients by pursuing indications across many autoimmune diseases with substantial unmet medical need in maternal-fetal disorders, neuro-inflammatory disorders, rheumatology, dermatology and autoimmune hematology.
Nipocalimab recently received a rare pediatric disease designation from the U.S. Food and Drug Administration.
Momenta’s expertise in FcRn mechanisms is especially important for nipocalimab as it supports and accelerates the development of a medicine designed to target a number of autoantibody-driven conditions across several of Janssen’s established therapeutic areas.
Janssen expects nipocalimab to contribute to its goals of achieving above-market growth over the mid and long term.
In addition to Momenta’s employees and lead asset nipocalimab, Janssen will acquire Momenta’s pipeline of clinical and preclinical assets.
Under the terms of the transaction, which was approved by the Boards of Directors of both companies, Vigor Sub, Inc. (Merger Sub), a newly formed wholly-owned subsidiary of Johnson & Johnson (the Company), will commence a tender offer to purchase all outstanding shares of Momenta for $52.50 per share.
The closing of the offer is conditioned on the tender of a majority of the outstanding shares of Momenta’s common stock on a fully diluted basis, as well as clearance under the Hart-Scott-Rodino Antitrust Improvements Act and other customary closing conditions.
The merger will be effected as soon as practicable after the closing of the tender offer. The transaction is expected to close in the second half of 2020.
While the closing of the transaction is expected to be modestly dilutive, the Company is maintaining its current 2020 Adjusted EPSi guidance range.
Looking ahead, the costs associated with the development of Momenta’s portfolio are expected to be incremental to planned R&D investment levels over the next few years given the value creation potential of our current portfolio, and thus expect this incremental investment in R&D to have an EPS impact worth approximately 10c-15c in 2021.
Shares of MNTA are up 69% to $52.14 in morning trading.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Gilead submits NDA to FDA for Veklury for COVID-19 treatment
Gilead Sciences (GILD) announced that it has submitted a New Drug Application to the U.S. Food and Drug Administration for Veklury® (remdesivir), an investigational antiviral for the treatment of patients with COVID-19.
Gilead files for NDA for Veklury
#Veklury is currently available in the U.S. under an Emergency Use Authorization for the treatment of hospitalized patients with severe COVID-19.
The filing is the final tier of the rolling NDA submission that was initiated on April 8, 2020.
The filing is supported by data from two randomized, open-label, multi-center Phase 3 clinical studies of Veklury conducted by Gilead and the Phase 3 randomized, placebo-controlled study of Veklury conducted by the National Institute of Allergy and Infectious Diseases.
These studies demonstrated that treatment with Veklury led to faster time to recovery compared with placebo and that a 5-day or 10-day treatment duration led to similar clinical improvement.
Across studies, Veklury was generally well-tolerated in both the 5-day and 10-day treatment groups, with no new safety signals identified. Veklury has been approved by multiple regulatory authorities around the world, including in the European Union and Japan.
In countries where Veklury has not been approved, including the United States, Veklury is an investigational drug and the safety and efficacy of remdesivir have not been established.
Veklury has not been approved by the U.S. Food and Drug Administration (FDA) for any use.
In the United States, the FDA granted Veklury an Emergency Use Authorization (EUA) for the treatment of hospitalized patients with severe COVID-19. This authorization is temporary and may be revoked, and it does not take the place of the formal new drug application submission, review and approval process.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Shares of Fulgent Genetics (FLGT) have surged during Wednesday’s session after the company reported second quarter results after the market close last night.
The company noted that it had “record” figures for quarterly revenue, billable tests, adjusted earnings, and adjusted EBITDA, as it has commercially launched several tests for COVID-19 since March 2020.
EARNINGS
In its latest report, Fulgent Genetics reported Q2 adjusted EPS of 17c on revenue of $17.3M, compared to analysts’ estimates of (5c) and $10.05M.
The company delivered 180,513 billable tests in the quarter, an increase of 1003% year-over-year, and gross margin for the quarter improved roughly eight percentage points from the previous quarter and roughly 81% year-over-year.
Commenting on the results, chairman and CEO Ming Hsieh said, “The global COVID-19 pandemic has tested who we are as a company, and now more than ever we have demonstrated that Fulgent is a technology company with a proprietary platform built for massive scale.
Our technology is the cornerstone for all facets of our business, including cloud computing, pipeline services, record management, web portal services, clinical workflow, sequencing as a service and automated lab services.
Covid-19 Test Kit
Our second quarter results illustrate how we quickly applied our technology to the needs of today, organically developing and launching multiple tests to detect COVID-19 with Emergency Use Authorization from the FDA, including an at-home test offered through Picture Genetics, our patient-initiated product.
These offerings have attracted major new customer accounts, resulting in an inflection point in our business and outlook.”
GUIDANCE:
On Fulgent’s quarterly conference call, CFO Paul Kim said that based on the “explosive” demand the company is seeing from the market and the quality of its customers, he continues to see the upward trajectory and transformation of the business continuing for the balance of 2020.
Fulgent now projects test volumes for 2020 to be over 1.3M, which translates to into over $120M in revenues. Kim added that he sees adjusted net income of $25M, or about $1.00 per share in FY20.
Analysts had expected the company to report FY20 EPS of 12c on revenue of $45.02M.
Analysts Comments:
Following the results, Piper Sandler analyst Steven Mah maintained an Overweight rating on Fulgent Genetics and raised his price target on the stock to $68 from $31.
Mah, who continues to believe COVID-19 testing is durable for the coming years, said he is encouraged by Fulgent’s early success in test volume, adding that his higher revenue estimates are due to increased COVID-19 expectations.
PRICE ACTION:
In afternoon trading, Fulgent Genetics shares are up nearly 23%, trading at $36.46.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Exact Sciences receives revised EUA for COVID-19 test
A letter to Exact Sciences Laboratories (EXAS), dated August 3, posted to the site of the FDA states:
“On May 22, 2020, based on your request, the Food and Drug Administration issued a letter determining that your product met the criteria for issuance under section 564(c) of the Act to be eligible for authorization under the March 31, 2020, Emergency Use Authorization – EUA – for Molecular-based Laboratory Developed Tests for Detection of Nucleic Acid from SARS-CoV-2 for the qualitative detection of nucleic acid from SARS-CoV-2 in respiratory specimens collected from individuals suspected of COVID-19 by their healthcare provider…
On July 17, 2020, FDA received a request from you to revise the Scope of Authorization, and thus the test’s intended use as originally specified by the High Complexity LDT Umbrella EUA, to include self-collection of nasal swab specimens that are self-collected at home or in a healthcare setting by individuals using an authorized home-collection kit specified in this EUA’s authorized labeling when determined to be appropriate by a healthcare provider, and to specify that testing is limited to Exact Sciences Laboratories at two locations..
Having concluded that the criteria for issuance of this authorization under Section 564(c) of the Act are met, I am authorizing the emergency use of your product, as described in the Scope of Authorization of this letter (Section II), subject to the terms of this authorization.”
Exact Sciences is known for it’s Cologuard, colon cancer detection test
The COVID-19 test is offered through US physicians and authorized healthcare providers. The test is intended for use with patients who meet the CDC’s current guidance for evaluation of COVID-19 infection.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
IMV Inc. provides update on progress of COVID-19 vaccine program
IMV Inc. (IMV) provided further details on the company’s progress in developing its candidate vaccine to prevent COVID-19 infection in response to the global health threat posed by the novel coronavirus.
The company said, “We are working closely with regulatory agencies and our collaborators to initiate clinical studies as quickly as possible.
IMV updates it’s progress on Covid-19
The design of the phase 1 clinical study, agreed with Health Canada, is a randomized controlled study, assessing the safety and immunogenicity of DPX-COVID-19, in 84 healthy adults across two age cohorts: (1) adults between 18-55 years old inclusive and (2) 56 and above. Two dose levels of DPX-COVID-19 will be tested (25 undefined or 50 undefined).
We are pleased that Health Canada has welcomed the design of a phase 1 trial that includes this vulnerable population.
The rapid progress in target selection, the vaccine formulation, manufacturing and preclinical results so far not only demonstrate the potential of our delivery platform, but also build on our previously reported clinical data from a similarly designed vaccine against RSV, the respiratory syncytial virus.
Clinical results have shown our DPX-based vaccine against RSV demonstrated a unique ability to generate safe and long-lasting immune responses in older adults.
IMV’s candidate vaccine, DPX-COVID-19, is based on IMV’s first-in-class delivery platform that generates targeted and sustained immune response in vivo.
Fully synthetic, the vaccine candidate is designed to focus the immune response on the weaknesses of the virus with the goal to optimize safety and efficacy: DPX-COVID-19 is a formulation of the DPX delivery platform with four complementary peptide antigens that were selected for their high immunogenicity and ability to bind non-overlapping areas on the virus spike and impact its infective function in preclinical studies, Importantly, our selected targets are located outside of the 614 mutation which, according to recent research, has been demonstrated to increase the virus’ ability to infect cells in vitro and suggested to potentially reduce vaccine-induced immunity.
We believe our vaccine candidate would retain its potential efficacy independently from current/future mutations of the virus at this site, Areas on the virus spike identified as potentially responsible for vaccine-enhanced disease4 have been excluded from our target selection to minimize safety risk.
Since the Company announced the selection of its candidate vaccine on May 21st, the Company has made significant progress.
Preclinical studies have demonstrated the capacity of DPX-COVID-19 to induce strong immunogenicity including the binding on target to the spike protein and viral neutralization, The Company has completed the current good manufacturing practice (“cGMP”) formulation and manufacturing process development for DPX-COVID-19, and Multiple batches have been successfully produced at IMV.”
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Monopar, NorthStar file provisional patent for development, use of RITs
Monopar Therapeutics (MNPR) and NorthStar Medical Radioisotopes announced that a provisional patent application entitled “Precision Radioimmunotherapeutic Targeting of the Urokinase Plasminogen Activator Receptor for Treatment of Severe COVID-19 Disease” has been filed with the U.S. Patent and Trademark Office.
The patent application offers hope for Covid-19 patients, Stockwinners
This application covers novel compositions and uses of cytotoxic radioisotopes attached to antibodies that bind to uPAR, thereby creating precision targeted radiotherapeutics for the treatment of severe COVID-19 and other respiratory diseases.
Advanced COVID-19 patients frequently develop severe, life-threatening, pulmonary inflammation as a result of a viral induced cytokine storm.
The development of this cytokine storm is associated with a high rate of mortality in severe COVID-19 patients, even with oxygen support and mechanical ventilation.
A severe immune reaction in which the body releases too many cytokines into the blood too quickly. Cytokines play an important role in normal immune responses, but having a large amount of them released in the body all at once can be harmful. A cytokine storm can occur as a result of an infection, autoimmune condition, or other disease. It may also occur after treatment with some types of immunotherapy.
Signs and symptoms include high fever, inflammation (redness and swelling), and severe fatigue and nausea. Sometimes, a cytokine storm may be severe or life threatening and lead to multiple organ failure. Also called hypercytokinemia.
uPRITs have been designed with the goal of selectively destroying the aberrantly activated white blood cells responsible for causing the cytokine storm.
If successful, healthy tissue would be spared in the process as the uPAR target is primarily only present on this unique class of white blood cells and not in healthy tissue.
The co-inventors of the provisional patent application are James Harvey, Chief Scientific Officer of NorthStar, and Andrew P. Mazar, Chief Scientific Officer of Monopar.
If granted, the patent would offer exclusivity to Monopar and NorthStar for the development and potential use of uPRITs in the treatment of severe COVID-19 and other respiratory diseases.
This provisional patent application leverages the therapeutic radioisotope expertise of NorthStar and the translational expertise of Monopar to create a novel, targeted radioimmunotherapeutic.
Radioimmunotherapy uses an antibody labeled with a radionuclide to deliver cytotoxic radiation to a target cell. In cancer therapy, an antibody with specificity for a tumor-associated antigen is used to deliver a lethal dose of radiation to the tumor cells.
On June 16, 2020, Monopar and NorthStar announced a 50/50 collaboration to couple Monopar’s MNPR-101 uPAR targeting monoclonal antibody to a therapeutic radioisotope provided by NorthStar.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Novavax sinks after White House omits as COVID vaccine finalist
The New York Times reports that Moderna (MRNA) is among the five finalists selected by the Trump administration as the most likely to produce a vaccine for the coronavirus.
Moderna (MRNA) is one of the finalists
By narrowing the field, the White House is betting it can identify the most promising vaccines at an early stage, speed along the process of determining which will work and ensure that the winner or winners can be quickly manufactured in large quanities, the Times said.
Pfizer is also one of the finalists
The announcement of the decision will be made at the White House in the next few weeks, government officials said.
AstraZeneca is one of the five companies.
Dr. Anthony S. Fauci, the federal government’s top epidemiologist and director of the National Institute of Allergy and Infectious Diseases, hinted at the coming action on Tuesday when he told a medical seminar that “by the beginning of 2021 we hope to have a couple of hundred million doses.”
Johnson & Johnson is also a finalist for Covid 19 vaccine
Finalists
A White House announcement is expected in the new few weeks. In addition to Moderna, the winners are AstraZeneca (AZN), Johnson & Johnson (JNJ), Merck (MRK) and Pfizer (PFE), according the Times.
Merck is the final company on the list.
B. Riley FBR
B. Riley FBR analyst Mayank Mamtani views the selloff today in shares of Novavax on the New York Times report that the company was not selected as a finalist for the White House’s “Operation Warp Speed” as overdone.
Mamtani reiterates a Buy rating on Novavax. NVAX closed down 11% to $44.25.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Bristol Myers Squibb (BMY) reported positive ulcerative colitis treatment data. The report sent shares of Arena Pharmaceuticals (ARNA) higher.
Arena shares jump of Bristol Myers data, Stockwinners
The study tested Bristol Myers’ Zeposia in patients with moderate to severe ulcerative colitis. At weeks 10 and 52, the experimental ulcerative colitis treatment induced clinical remission and maintained remission, respectively.
Zeposia belongs to a class of oral drugs known as sphingosine-1-phosphate receptor modules, or S1P. This is the first time an S1P drug has shown strong effectiveness in ulcerative colitis treatment, suggesting these drugs could beat out another class called janus kinase inhibitors, or JAKs.
Arena is also testing out an S1P drug, etrasimod.
Etrasimod is a next-generation, once-daily, oral, highly selective sphingosine 1-phosphate (S1P) receptor modulator discovered by Arena, and designed for optimized pharmacology and engagement of S1P receptor 1, 4 and 5 which may lead to an improved efficacy and safety profile.
Etrasimod provides systemic and local effects on specific immune cell types and has the potential to treat multiple immune-mediated inflammatory diseases including ulcerative colitis, Crohn’s disease, and atopic dermatitis.
Etrasimod is an investigational compound that is not approved for any use in any country.
Canter Comments
Cantor Fitzgerald analyst Alethia Young raised the firm’s price target on Arena Pharmaceuticals (ARNA) to $88 from $68 and reiterates an Overweight rating on the shares.
Bristol Meyers’ (BMY) positive topline data this morning from the pivotal study of its sphingosine-1-phosphate ozanimod in ulcerative colitis is a “positive readthrough” to Arena’s etrasimod ulcerative colitis program, Young tells investors in a research note.
The analyst increased her probability of success to 80% from 75% and is now “very confident” in the Phase 3 success of etrasimod.
#Young also thinks that #S1P1 as a class is shaping up to be a “big commercial opportunity” and increased her peak sales to $3B.
She thinks Arena shares will be up at least 10% today on Bristol’s news given that ozanimod and etrasimod are both S1P1s and this is the first positive Phase 3 readout for the class in ulcerative colitis, says the analyst.
Credit Suisse
Credit Suisse analyst Martin Auster raised the firm’s price target on Arena Pharmaceuticals (ARNA) to $87 from $77 and keeps an Outperform rating on the shares after Bristol-Myers (BMY) announced that S1P modulator ozanimod met its primary endpoint of clinical remission in the Phase 3 “True North” study of adults with moderate to severe ulcerative colitis.
Auster views ozanimod’s topline success as de-risking for the S1P class and sees positive read-through for Arena’s etrasimod, he tells investors. He has increased his view on the odds of success for etrasimod in ulcerative colitis following Bristol’s report, the analyst added.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Iterum says Phase 3 trial of sulopenem did not meet primary endpoint
Iterum Therapeutics (ITRM) announced that sulopenem did not achieve statistical non-inferiority relative to ertapenem in its SUlopenem for Resistant Enterobacteriaceae 2 clinical trial in complicated urinary tract infection.
Iterum shares collapse following it’s drug failure
The primary U.S. Food and Drug Administration endpoint was overall clinical and microbiologic response on Day 21 in the micro-MITT population as evaluated using a 10% non-inferiority margin.
The randomized, multi-center, double-blind SURE 2 clinical trial enrolled 1,395 patients to measure the efficacy, tolerability, and safety of IV and oral sulopenem for the treatment of cUTI in adults.
Patients were randomized to receive either IV sulopenem once daily for a minimum of five days followed by oral sulopenem twice daily to complete seven to ten days of treatment, or IV ertapenem once daily for a minimum of five days followed by either oral ciprofloxacin or, for quinolone resistant isolates, amoxicillin-clavulanate twice daily.
Responder rates at the test of cure visit for sulopenem were 67.8% and for ertapenem were 73.9% with a difference of -6.1%.
The difference in response rates was driven almost entirely by higher rates of asymptomatic bacteriuria on sulopenem relative to ertapenem, only evident at the test of cure visit; the rates of patients receiving additional antibiotics or with residual cUTI symptoms was similar.
Clinical response at the test of cure in the Modified Intent to Treat patient population and Clinically Evaluable patient population sulopenem vs ertapenem: 0.4% was similar.
The outcome at other secondary endpoints was also similar, including the overall response at the end of therapy visit at Day 10.
Strategic Alternatives
Based on the trial results, Iterum Therapeutics is evaluating its corporate, strategic and financial alternatives with the goal of maximizing value for its stakeholders while prudently managing its remaining resources.
These alternatives could potentially include the licensing, sale or divestiture of the company’s assets or proprietary technologies, a sale of the company, a merger or other business combination, another strategic transaction involving the company, restructuring activities, winding down of operations, dissolving and liquidating assets or seeking protection under bankruptcy laws.
This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.
Axsome Therapeutics announces AXS-07 met primary, secondary endpoints
Axsome Therapeutics (AXSM) announced that AXS-07, Axsome’s novel, oral, multi-mechanistic investigational medicine for the acute treatment of migraine, met the two regulatory co-primary endpoints and significantly improved migraine pain and most bothersome symptoms as compared to placebo in the MOMENTUM Phase 3 trial.
AXS-07 also met the key secondary endpoint, demonstrating statistically significant superiority to the active comparator rizatriptan on sustained freedom from migraine pain.
MOMENTUM was a randomized, double-blind, placebo- and active-controlled trial which enrolled only patients with a history of inadequate response to prior acute migraine treatments, assessed using the Migraine Treatment Optimization Questionnaire, or mTOQ-4.
A total of 1,594 patients were randomized in a 2:2:2:1 ratio to AXS-07, rizatriptan, MoSEIC, meloxicam, or placebo, to treat a single migraine attack of moderate or severe intensity.
In addition to a history of inadequate response, enrolled patients exhibited a high rate of characteristics that are strongly associated with poor treatment outcomes including cutaneous allodynia, severe migraine pain intensity, obesity and morning migraine.
The study was conducted pursuant to a FDA special protocol assessment, or SPA.
AXS-07 met the two regulatory co-primary endpoints by demonstrating, with high statistical significance, a greater percentage of patients as compared to placebo achieving pain freedom and absence of most bothersome symptom two hours after dosing.
Superiority of AXS-07 to rizatriptan and MoSEIC meloxicam was established as specified in the SPA, by demonstration of a greater percentage of AXS-07 patients achieving sustained pain freedom from two to 24 hours after dosing, compared to rizatriptan and MoSEIC meloxicam, as well as to placebo.
The positive results on both co-primary endpoints along with the demonstration of component contribution support the filing of an NDA for AXS-07 in the acute treatment of migraine.
AXS-07 provided greater and more sustained migraine pain relief compared to placebo and rizatriptan, which translated to a significant reduction in rescue medication use for AXS-07 compared to placebo and rizatriptan.
The percentage of patients experiencing sustained pain relief from two to 24 hours after dosing was 53.3% for AXS-07, compared to 33.5% for placebo and 43.9% for rizatriptan.
Sustained pain relief from two to 48 hours was also experienced by a statistically significantly greater proportion of AXS-07 patients, compared to placebo and rizatriptan patients.
Rescue medication was used by 23.0% of AXS-07 patients, compared to 43.5% of placebo and 34.7% of rizatriptan patients. AXS-07 provided rapid relief of migraine pain with the percentage of patients achieving pain relief with AXS-07 being numerically greater than with rizatriptan at every time point measured starting at 15 minutes, and statistically significantly greater than with rizatriptan by 60 minutes.
The proportions of patients experiencing pain relief 1.5 hours after dosing were 60.5% for AXS-07 compared to 52.5% for rizatriptan and 48.3% for placebo.
AXS-07 was statistically significantly superior to rizatriptan on several other secondary endpoints including Patient Global Impression of Change and return to normal functioning at 24 hours.
AXS-07 was safe and well tolerated in the trial.
The most commonly reported adverse events with AXS-07 were nausea, dizziness and somnolence, none of which occurred at a rate greater than placebo or greater than 3%.
There was one serious adverse event in the AXS-07 arm which was deemed by the investigator not to be related to study drug.
The MOMENTUM study was conducted pursuant to an SPA with the FDA.
The SPA provides agreement that the overall MOMENTUM trial design and planned analysis adequately address objectives that, if met, will support the regulatory submission for approval of AXS-07 for the indication of acute treatment of migraine in adults with or without aura.
Based on FDA feedback, Axsome believes that MOMENTUM will be the only efficacy trial required to support an NDA filing for AXS-07 for the acute treatment of migraine.
Axsome plans to file the NDA in the second half of 2020. AXS-07 is a novel, oral, rapidly absorbed, multi-mechanistic investigational medicine for the acute treatment of migraine, consisting of MoSEIC meloxicam and rizatriptan.
AXS-07 is thought to act by inhibiting CGRP release, reversing CGRP-mediated vasodilation, and inhibiting neuro-inflammation, pain signal transmission, and central sensitization.
Axsome’s MoSEIC technology significantly increases the speed of absorption of the meloxicam component after oral administration while maintaining a long plasma half-life.
AXS-07 is covered by 21 issued U.S. and international patents providing protection out to 2036, and Axsome maintains worldwide rights.
Detailed study results, including additional secondary endpoints, will be submitted for presentation at upcoming medical meetings and for publication.
AXS-07 is also being evaluated in the INTERCEPT Phase 3 trial which is a randomized, double-blind, placebo-controlled study evaluating the early treatment of migraine with AXS-07.
In contrast to the ongoing MOMENTUM trial in which patients with a history of inadequate response treated migraine attacks once they have become of moderate or severe intensity, in the INTERCEPT trial, patients are to administer AXS-07 at the earliest sign of migraine pain.
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