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.
Eli Lilly TRAILBLAZER-ALZ 2 met primary endpoint, all secondary endpoint
Eli Lilly (LLY) announced results of the TRAILBLAZER-ALZ 2 Phase 3 study showing that donanemab significantly slowed cognitive and functional decline in people with early symptomatic Alzheimer’s disease.
Donanemab met the primary endpoint of change from baseline until 18 months on the integrated Alzheimer’s Disease Rating Scale, or iADRS.
The primary endpoint of iADRS measures cognition and activities of daily living such as managing finances, driving, engaging in hobbies, and conversing about current events.
All secondary endpoints of cognitive and functional decline were also met and showed highly statistically significant clinical benefits with similar magnitude.
Based on these results, Lilly will proceed with global regulatory submissions as quickly as possible and anticipates making a submission to the FDA yet this quarter.
Lilly will work with the FDA and other global regulators to achieve the fastest path to traditional approvals.
TRAILBLAZER-ALZ 2, a randomized, double-blind, placebo-controlled study, evaluated the safety and efficacy of donanemab, an investigational amyloid plaque targeting therapy.
The study enrolled people with early symptomatic Alzheimer’s disease, or AD, which includes mild cognitive impairment, or MCI, and the mild dementia stage of disease, with the confirmed presence of AD neuropathology, and participants completed their course of treatment with donanemab once they reached a prespecified level of amyloid plaque clearance.
Participants in TRAILBLAZER-ALZ 2 were stratified by their level of the brain protein tau, a predictive biomarker for Alzheimer’s disease progression. The primary analysis population for which the study was powered was comprised of people with an intermediate level of tau and clinical symptoms of Alzheimer’s disease.
In this population, the primary endpoint (iADRS) showed 35% slowing of decline, and an important key secondary endpoint showed 36% slowing of decline over 18 months.
Additional pre-specified secondary analyses showed: 47% of participants on donanemab showed no decline on CDR-SB, a key measure of disease severity at 1 year. 52% of participants completed their course of treatment by 1 year and 72% completed by 18 months as a result of achieving plaque clearance.
Participants on donanemab had 40% less decline in ability to perform activities of daily living at 18 months. Participants on donanemab experienced a 39% lower risk of progressing to the next stage of disease compared to placebo.
The study also enrolled a smaller number of people with high levels of tau at baseline, representing a later stage of disease progression.
Because these participants were predicted to progress more quickly and be less responsive to therapy, the target population for the study was the intermediate tau population. The high tau participants were combined with the intermediate tau population in an additional primary analysis of all participants enrolled. In this combined population, donanemab also demonstrated meaningful positive results across all clinical endpoints, with CDR-SB and iADRS showing 29% and 22% slowing of decline, respectively. The incidence of amyloid-related imaging abnormalities was consistent with the TRAILBLAZER-ALZ Phase 2 study.
Infusion-related reactions occurred in 8.7% of participants with most cases mild to moderate in severity. In addition to slowing cognitive and functional decline in TRAILBLAZER-ALZ 2, donanemab produced significant reductions in brain amyloid plaquelevels as early as 6 months after initiating treatment, as observed using amyloid positron emission tomography brain scan, with many patients reaching amyloid levels considered negative for pathology1.
Alzheimer’s disease is a progressive brain disorder that affects memory, thinking, and behavior. According to the World Health Organization (WHO), around 50 million people worldwide have dementia, and the most common cause of dementia is Alzheimer’s disease.
In the United States, it is estimated that around 6 million people have Alzheimer’s disease. This number is expected to increase as the population ages.
The economic effect of Alzheimer’s disease is significant. According to the Alzheimer’s Association, in 2021, the total cost of caring for people with Alzheimer’s and other dementias in the United States is projected to reach $355 billion. This includes the cost of medical care, long-term care, and lost wages for family caregivers. By 2050, these costs are projected to increase to $1.1 trillion.
The economic impact is not just limited to the United States. Alzheimer’s disease has a global economic impact, with estimated costs of around $1 trillion in 2018. As the population ages, the economic impact is expected to increase.
LLY shares are up $25 to $429.15 or 6% on the day. Shares of Biogen (BIIB) which has a competing drug are down $5 to $310.03.
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.
Prometheus announces results for PRA023 in APOLLO-CD Phase 2 study
Prometheus Biosciences (RXDX) reported results from its ARTEMIS-UC Phase 2 and APOLLO-CD Phase 2a studies of PRA023 demonstrating strong efficacy and favorable safety results in both studies.
Based on the totality of the data in these two studies, Prometheus intends to advance PRA023 into Phase 3 studies for ulcerative colitis and Crohn’s disease in 2023.
Results from the APOLLO-CD Phase 2a Study: Prometheus’ Phase 2a APOLLO-CD clinical trial was a 12-week open-label study that enrolled 55 patients with moderate-to-severely active CD with endoscopically active disease who had failed conventional or biologic therapy.
Crohn’s Disease
The study enrolled a highly refractory patient population with 70.9% of patients previously treated with at least one biologic therapy and 52.7% treated with two or more biologic therapies.
The results on the key endpoints were as follows: 26.0% of patients on PRA023 achieved endoscopic response; 49.1% of patients on PRA023 achieved clinical remission; PRA023 was well tolerated in the APOLLO-CD study.
There were no treatment-emergent serious adverse events, adverse events leading to discontinuation, or severe AEs assessed as related to PRA023 by the investigator.
The predictive power of the company’s prespecified genetic markers was validated using an alternative Crohn’s-specific CDx algorithm which showed 45.0% endoscopic response relative to all-comers of 26%.
While the original algorithm provided limited benefit on some of the endpoints, the alternative algorithm demonstrated enhanced performance across both clinical and endoscopic outcomes.
As a result of these positive data, Prometheus plans to advance PRA023 into pivotal development in 2023, following discussions with regulators.
Based upon confidence in its precision approach and speed to market, the company conducted an interim companion diagnostic (CDx) analysis of Cohort 1 to evaluate the effectiveness of the CDx candidate in ARTEMIS-UC. Although from limited patient numbers, data from the subset of patients who tested positive on the CDx in Cohort 1 (N=32) demonstrated a placebo-adjusted clinical remission rate of 37.5%, compared with the placebo-adjusted remission rate of 25.0% for all-comers. The expansion cohort (Cohort 2), which is statistically powered to further assess the treatment effect of PRA023 in CDx+ patients will continue to enroll, and the company expects results in the second quarter of 2023.
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.
Biogen/Eisai’s phase 3 trial of lecanemab in MCI meet primary endpoint
Eisai (ESALY) and Biogen (BIIB) “announced positive topline results from Eisai’s large global Phase 3 confirmatory Clarity AD clinical trial of lecanemab, an investigational anti-amyloid beta protofibril antibody for the treatment of mild cognitive impairment due to Alzheimer’s disease, AD, and mild AD with confirmed presence of amyloid pathology in the brain.
#Lecanemab met the primary endpoint and all key secondary endpoints with highly statistically significant results.
#Eisai will discuss this data with regulatory authorities in the U.S., Japan and Europe with the aim to file for traditional approval in the US and for marketing authorization applications in Japan and Europe by the end of Eisai’s FY2022, which ends March 31, 2023.
Amyloid-related imaging abnormalities
Additionally, Eisai will present the Clarity AD study results on November 29, 2022, at the Clinical Trials on Alzheimer’s Congress, and publish the findings in a peer-reviewed medical journal. Lecanemab treatment met the primary endpoint and reduced clinical decline on the global cognitive and functional scale, CDR-SB, compared with placebo at 18 months by 27%, which represents a treatment difference in the score change of -0.45 in the analysis of Intent-to-treat population.
Starting as early as six months, across all time points, the treatment showed highly statistically significant changes in CDR-SB from baseline compared to placebo.
All key secondary endpoints were also met with highly statistically significant results compared with placebo.
Key secondary endpoints were the change from baseline at 18 months compared with placebo of treatment in amyloid levels in the brain measured by amyloid positron emission tomography, the AD Assessment Scale-cognitive subscale14, AD Composite Score and the AD Cooperative Study-Activities of Daily Living Scale for Mild Cognitive Impairment.”
ESALY last traded at $39.79. BIIB last traded at $197.50.
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.
AbbVie’s cariprazine met primary endpoint in Phase 3 study
AbbVie (ABBV) announced top-line results from two Phase 3 clinical trials, Study 3111-301-001 and Study 3111-302-001, evaluating the efficacy and safety of cariprazine as an adjunctive treatment for patients with major depressive disorder.
Cariprazine, sold under the brand names Vraylar in the United States and Reagila in the European Union, is an atypical antipsychotic which is used in the treatment of schizophrenia, bipolar mania, and bipolar depression.
Drug pipeline looks very promising for Abbvie
In Study 3111-301-001, cariprazine showed a statistically significant change from baseline to week six in the Montgomery-Asberg Depression Rating Scale total score compared with placebo.
The Montgomery–Asberg Depression Rating Scale (MADRS) is a ten-itemdiagnostic questionnaire which psychiatrists use to measure the severity of depressive episodes in patients with mood disorders.
Patients treated with cariprazine at 1.5 mg/day achieved improved MADRS total score at week six compared to placebo.
Patients treated with cariprazine at 3.0 mg/day demonstrated improvement in MADRS total score at week six over placebo but did not meet statistical significance.
In Study 3111-302-001, cariprazine demonstrated numerical improvement in depressive symptoms from baseline to week six in MADRS total score compared with placebo but did not meet its primary endpoint for either the 1.5 mg/day or 3.0 mg/day dose.
In a previously published Phase 2/3 registration-enabling study, RGH-MD-75, patients treated with cariprazine flexible doses of 2.0-4.5 mg/day in addition to ongoing antidepressant therapy met the primary endpoint and achieved improved MADRS total scores at week eight compared to placebo.
Based on the positive results of studies 3111-301-001 and RGH-MD-75, and the totality of data reported, AbbVie intends to submit a supplemental New Drug Application with the U.S. FDA for the expanded use of cariprazine for the adjunctive treatment of MDD.
Separately, AbbVie reported Q3 Global Humira sales of $5.425B up 5.6% on reported basis.
In Q3, Global net revenues from the immunology portfolio were $6.674 billion, an increase of 15.3 percent on a reported basis, or 14.9 percent on an operational basis.
Global Humira net revenues of $5.425 billion increased 5.6 percent on a reported basis, or 5.2 percent on an operational basis.
U.S. Humira net revenues were $4.613 billion, an increase of 10.1 percent.
Internationally, Humira net revenues were $812 million, a decrease of 14.6 percent on a reported basis, or 16.7 percent on an operational basis, due to biosimilar competition.
Treatment for Psoriasis
Global Skyrizi net revenues were $796 million. Global Rinvoq net revenues were $453 million.
Treatment for moderate to severe rheumatoid arthritis
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.
FDA announces ‘first authorization’ for marketing of e-cigarette products
The U.S. Food and Drug Administration announced it has authorized the marketing of three new tobacco products, which it noted marks “the first set of electronic nicotine delivery system products ever to be authorized by the FDA through the Premarket Tobacco Product Application – PMTA – pathway.”
The FDA issued marketing granted orders to R.J. Reynolds Vapor Company, a subsidiary of British American Tobacco, for its Vuse Solo closed ENDS device and accompanying tobacco-flavored e-liquid pods, specifically, Vuse Solo Power Unit, Vuse Replacement Cartridge Original 4.8% G1, and Vuse Replacement Cartridge Original 4.8% G2.
“As the RJR Vapor Company submitted data to the FDA that demonstrated that marketing of these products is appropriate for the protection of public health, today’s authorization allows these products to be legally sold in the U.S.,” the FDA stated.
Solar Powered Vuse
Today, the FDA also issued 10 marketing denial orders for flavored ENDS products submitted under the Vuse Solo brand by RJR.
“Due to potential confidential commercial information issues, the FDA is not publicly disclosing the specific flavored products. These products subject to an MDO for a premarket application may not be introduced or delivered for introduction into interstate commerce. Should any of them already be on the market, they must be removed from the market or risk enforcement. Retailers should contact RJR with any questions about products in their inventory.
The agency is still evaluating the company’s application for menthol-flavored products under the Vuse Solo brand,” the FDA stated.
“Today’s authorizations are an important step toward ensuring all new tobacco products undergo the FDA’s robust, scientific premarket evaluation. The manufacturer’s data demonstrates its tobacco-flavored products could benefit addicted adult smokers who switch to these products – either completely or with a significant reduction in cigarette consumption – by reducing their exposure to harmful chemicals. We must remain vigilant with this authorization and we will monitor the marketing of the products, including whether the company fails to comply with any regulatory requirements or if credible evidence emerges of significant use by individuals who did not previously use a tobacco product, including youth. We will take action as appropriate, including withdrawing the authorization,” said Mitch Zeller, J.D., director of the FDA’s Center for Tobacco Products.
Mitch Zeller, J.D., director of the FDA’s Center for Tobacco Products
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.
NY AG announces $1.1B agreement with big three drug distributors over opioids
New York Attorney General Letitia James announced an agreement with McKesson (MCK), Cardinal Health (CAH), and Amerisource Bergen (ABC) – three of the nation’s largest drug distributors – that will deliver up to $1.1B to New York state to combat the ongoing opioid epidemic.
“The $1.1B agreement is the largest monetary settlement ever negotiated by Attorney General James. The agreement resolves claims made by Attorney General James for the three companies’ role in helping to fuel the opioid epidemic and will remove the three distributors from New York’s ongoing opioid trial, currently underway in Suffolk County State Supreme Court…
Additionally, late last month, Attorney General James announced an agreement with Johnson & Johnson (JNJ) that removed the company from New York’s opioid trial in exchange for up to $230M for the state’s opioid prevention and treatment efforts, as well as it ending the sale of opioids nationwide.
The trial against the three remaining defendants – Endo Health Solutions (ENDP), Teva Pharmaceuticals USA (TEVA), and Allergan Finance (ABBV) – is currently underway and will continue in state court,” the AG stated.
As part of today’s agreement, McKesson, Cardinal Health, and Amerisource Bergen will pay New York state a total of up to $1,179,251,066.68, of which more than $1 billion will go towards abatement.
Payments will start in just two months and will continue over the course of the next 17 years, the AG said.
Opioids are a class of drugs that include the illegal drug heroin, synthetic opioids such as fentanyl, and pain relievers available legally by prescription, such as oxycodone (OxyContin®), hydrocodone (Vicodin®), codeine, morphine, and many others.
Fentanyl and similar compounds like carfentanil are powerful synthetic opioids — 50 to 100 times more potent than morphine. High doses of opioids, especially potent opioids such as fentanyl, can cause breathing to stop completely, which can lead to death.
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.
RAPT Therapeutics, Inc. (RAPT) announced positive topline results from its randomized placebo-controlled Phase 1b clinical trial of RPT193 as monotherapy in 31 patients with moderate-to-severe atopic dermatitis (AD).
Atopic dermatitis (eczema) is a condition that makes your skin red and itchy. It’s common in children but can occur at any age. Atopic dermatitis is long lasting (chronic) and tends to flare periodically. It may be accompanied by asthma or hay fever.
After four weeks of treatment, patients with moderate-to-severe AD who received RPT193 showed a 36.3% improvement from baseline in the Eczema Area and Severity Index (EASI) score, a standard measure of disease severity, compared to 17.0% in the placebo group.
Notably, in the two-week period following the end of treatment, the RPT193 group showed continued improvement and further separation from placebo with a 53.2% improvement in EASI at the six-week time point compared to 9.6% in the placebo group. This continued improvement may be related to RPT193’s mechanism of action, which is upstream of other agents targeting cytokines or signaling pathways.
Emma Guttman-Yassky, M.D., Ph.D., the Waldman Professor of Dermatology and System Chair Department of Dermatology at the Icahn School of Medicine at Mount Sinai, and member of RAPT’s Scientific Advisory Board, added, “I am very excited about these results as they not only demonstrate clinically meaningful improvement after just four weeks of treatment, but also further improvement for two weeks after completion of treatment. This may suggest that this novel mechanism of action targeting CCR4 on Th2 cells could have prolonged, disease-modifying effects, which could differentiate it from other agents. Along with being an oral drug that seems to have promising clinical activity and a well-tolerated safety profile, RPT193 could fill a high unmet medical need for AD patients.”
Cantor Fitzgerald
Cantor Fitzgerald analyst Alethia Young raised the firm’s price target on Rapt Therapeutics to $71 from $51 and reiterates an Overweight rating on the shares. The stock in midday trading is up 110%, or $19.60, to $38.17. This morning’s RPT193 data update “was robust with clear clinical benefit compared to placebo on all exploratory endpoints,” Young tells investors in a research note.
The analyst took the drug’s probability of success in atopic dermatitis to 50% from 25% previously, and increased her peak sales estimates. Young sees a “large unmet need” for a safe and effective oral treatment which is separate from the injectable market, and models peak sales of $4B in atopic dermatitis by 2035. She views Rapt’s risk/benefit profile as potentially best-in-class for an oral treatment.
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.
FDA approves Biogen Alzheimer’s drug, says benefits outweigh risks
The FDA approved Biogen’s (BIIB) Aduhelm to treat patients with Alzheimer’s disease.
“This approval is significant in many ways. Aduhelm is the first novel therapy approved for Alzheimer’s disease since 2003.
Perhaps more significantly, Aduhelm is the first treatment directed at the underlying pathophysiology of Alzheimer’s disease, the presence of amyloid beta plaques in the brain.
The clinical trials for Aduhelm were the first to show that a reduction in these plaques – a hallmark finding in the brain of patients with Alzheimer’s – is expected to lead to a reduction in the clinical decline of this devastating form of dementia,” the FDA said in a statement.
Eli Lilly is a partner with Biogen
It added, “We ultimately decided to use the Accelerated Approval pathway – a pathway intended to provide earlier access to potentially valuable therapies for patients with serious diseases where there is an unmet need, and where there is an expectation of clinical benefit despite some residual uncertainty regarding that benefit.
Brain of an Alzheimer patient
In determining that the application met the requirements for Accelerated Approval, the Agency concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweighed the risks of the therapy.”
The FDA said in its approval statement: “Additionally, FDA is requiring Biogen to conduct a post-approval clinical trial to verify the drug’s clinical benefit. If the drug does not work as intended, we can take steps to remove it from the market. But hopefully, we will see further evidence of benefit in the clinical trial and as greater numbers of people receive Aduhelm. As an agency, we will also continue to work to foster drug development for this catastrophic disease.”
STIFEL
Stifel analyst Paul Matteis reiterates his Buy rating on Biogen shares following the FDA granting accelerated approval of aducanumab, now to be called “Aduhelm,” for the treatment of Alzheimer’s disease.
Approval based on amyloid plaque as a “surrogate” is “definitely unexpected” and appears to be a way for FDA to work around the contentious advisory committee meeting, argues Matteis, who adds that the approval “is a big win.” How investors will risk-adjust revenues that are modeled after completion of a Phase 4 trial and how insurers will treat access for a drug approved based on a biomarker are “highly interesting” questions that will now “be debated at a materially higher stock valuation,” added Matteis. Biogen shares remain halted for trading at midday following news of the FDA approval.
JEFFRIES
Jefferies analyst Andrew Tsai said news of Biogen (BIIB) being granted FDA approval for aducanumab is likely to spark investor enthusiasm across all Alzheimer’s names and he believes the longer-term setup for Athira Pharma (ATHA) looks more attractive now. Given what he views as “the FDA tailwind,” he would buy on strength as he believes the FDA’s aducanumab decision “clearly has a positive readthrough” to Athira, whose Phase I data suggests ATHA-1017 could produce “a profound cognitive benefit” in Phase 2/3 studies expected to read out in 2022, Tsai tells investors.
In that context, he thinks a 25%-50% short-term move for Athira shares “seems reasonable” relative to the company’s current market cap.
Shares of Biogen (BIIB) remain halted while Eli Lilly (LLY), who has an Alzheimer’s disease drug in its pipeline, is up 4% to $210.78 following the news.
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 Covid vaccine granted emergency approval from FDA
The Food and Drug Administration issued an emergency use authorization for the third vaccine for the prevention of coronavirus disease. The FDA has determined that the Janssen COVID-19 Vaccine has met the statutory criteria for issuance of an EUA. The totality of the available data “provides clear evidence that the Janssen COVID-19 Vaccine may be effective in preventing COVID-19,” the agency said in a statement.
#stockwinners
The Janssen COVID-19 Vaccine is manufactured using a specific type of virus called adenovirus type 26. The vaccine uses Ad26 to deliver a piece of the DNA, or genetic material, that is used to make the distinctive “spike” protein of the SARS-CoV-2 virus, the FDA said. While adenoviruses are a group of viruses that are relatively common, Ad26, which can cause cold symptoms and pink eye, has been modified for the vaccine so that it cannot replicate in the human body to cause illness, it added. After a person receives this vaccine, the body can temporarily make the spike protein, which does not cause disease, but triggers the immune system to learn to react defensively, producing an immune response against SARS-CoV-2.
The EUA allows Johnson & Johnson’s (JNJ) Janssen COVID-19 vaccine to be distributed in the U.S for use in individuals 18 years of age and older.
Meanwhile, Johnson & Johnson also announced that the U.S. Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices has recommended its single-shot COVID-19 vaccine.
The ACIP recommendation will be forwarded to the Director of the CDC and the U.S. Department of Health and Human Services for review and adoption.
Johnson & Johnson has begun shipping its COVID-19 vaccine and expects to deliver enough single-shot vaccines by the end of March to enable the full vaccination of more than 20M people in the U.S.
The company plans to deliver 100M single-shot vaccines to the U.S. during the first half of 2021. The U.S. government will manage allocation and distribution of the vaccine in the U.S.
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.
Merck to acquire $1B equity stake in Seattle Genetics as part of collaborations
Seattle Genetics (SGEN) and Merck (MRK) announced two new strategic oncology collaborations.
The companies will globally develop and commercialize Seattle Genetics’ ladiratuzumab vedotin, an investigational antibody-drug conjugate, or ADC, targeting LIV-1, which is currently in phase 2 clinical trials for breast cancer and other solid tumors.
Merck bets heavily on Seattle Genetics, Stockwinners
The collaboration will pursue a broad joint development program evaluating ladiratuzumab vedotin as monotherapy and in combination with Merck’s anti-PD-1 therapy Keytruda in triple-negative breast cancer, hormone receptor-positive breast cancer and other LIV-1-expressing solid tumors.
Under the terms of the agreement, Seattle Genetics will receive a $600M upfront payment and Merck will make a $1B equity investment in 5M shares of Seattle Genetics common stock at a price of $200 per share.
Seattle Genetics scores bug with Merck
In addition, Seattle Genetics is eligible for progress-dependent milestone payments of up to $2.6B.
Separately, Seattle Genetics has granted Merck an exclusive license to commercialize Tukysa, a small molecule tyrosine kinase inhibitor, for the treatment of HER2-positive cancers, in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe.
Seattle Genetics will receive $125M from Merck as an upfront payment and is eligible for progress-dependent milestones of up to $65M.
Under the terms of the agreement, Seattle Genetics and Merck will collaborate and equally share costs on the global development of ladiratuzumab vedotin and other LIV-1-targeting ADCs.
Breast cancer drug Liv-1 is expected to do well
The companies have agreed to jointly develop and share future costs and profits for ladiratuzumab vedotin on a 50:50 basis worldwide. Merck will pay Seattle Genetics $600M upfront and make a $1B equity investment in 5M shares of Seattle Genetics common stock at a price of $200 per share.
In addition, Seattle Genetics will be eligible to receive up to $2.6B in milestone payments, including $850M in development milestones and $1.75B in sales milestones.
The companies will jointly develop and commercialize ladiratuzumab vedotin and equally share profits worldwide.
The companies will co-commercialize in the U.S. and Europe. Seattle Genetics will be responsible for marketing applications for approval in the U.S. and Canada, and will record sales in the U.S., Canada and Europe.
Merck will be responsible for marketing applications for approval in Europe and in countries outside the U.S. and Canada, and will record sales in countries outside the U.S., Europe and Canada. Including the upfront payment, equity investment proceeds and potential milestone payments, Seattle Genetics is eligible to receive up to $4.2B.
Under the terms of the agreement, Merck has been granted exclusive rights to commercialize Tukysa in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe. Seattle Genetics retains commercial rights and will record sales in the U.S., Canada and Europe.
Merck will be responsible for marketing applications for approval in its territory, supported by the positive results from the HER2CLIMB clinical trial.
Merck will also co-fund a portion of the Tukysa global development plan, which encompasses several ongoing and planned trials across HER2-positive cancers, including breast, colorectal, gastric and other cancers set forth in a global product development plan.
Seattle Genetics will continue to lead ongoing Tukysa global development planning and operational execution.
Merck will solely fund and conduct country-specific clinical trials necessary to support anticipated regulatory applications in its territory.
Seattle Genetics will receive from Merck $125M as an upfront payment and is eligible to receive progress-dependent milestones of up to $65M. Seattle Genetics will also receive $85M in prepaid research and development payments to be applied to Merck’s global development funding obligations. In addition, Seattle Genetics would receive tiered royalties on sales of Tukysa in Merck’s territory.
SGEN is up 14.8% to $172.40. MRK is up 1.4% to $85.00.
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.
Intra-Cellular says lumateperone 42 mg met primary endpoint in Study 402
Intra-Cellular Therapies (ITCI) announced positive topline results from its Phase 3 clinical trial evaluating lumateperone as adjunctive therapy to lithium or valproate in the treatment of major depressive episodes associated with Bipolar I or Bipolar II disorder.
Intra-Cellular sharply higher on its bi-polar drug
In Study 402, once daily lumateperone 42 mg met the primary endpoint for improvement in depression as measured by change from baseline versus placebo on the MADRS total score.
Lumateperone 42 mg also met the key secondary endpoint, the CGI-BP-S Depression Score.
The lower lumateperone dose, 28 mg, showed a trend for a dose-related improvement in symptoms of depression but the results did not reach statistical significance.
Lumateperone demonstrated a favorable safety profile and was generally well-tolerated in the trial.
Lumateperone was successful in treating bipolar disorders
The most commonly reported adverse events that were observed at a rate greater than or equal to 5% and at least twice the rate of placebo were somnolence, dizziness, and nausea.
Rates of akathisia, restlessness, extrapyramidal symptoms, and changes in weight were similar to placebo.
This trial, in conjunction with our previously reported positive Phase 3 monotherapy study, Study 404, forms the basis for our sNDA for the treatment of bipolar depression in patients with Bipolar I or II disorder as monotherapy and adjunctive therapy which we expect to submit to the FDA in late 2020 or early 2021.
Study 402 was conducted globally in five countries including in the U.S. A total of 529 patients with moderate to severe major depressive episodes associated with either Bipolar I or Bipolar II disorder were randomized 1:1:1 to lumateperone 42 mg, 28 mg or placebo, while being maintained on lithium or valproate as mood stabilizers.
Lumateperone 42 mg met the primary endpoint by demonstrating a statistically significant improvement compared to placebo at week 6, as measured by change from baseline on the MADRS total score.
In the intent-to-treat study population, the least squares mean reduction from baseline for lumateperone 42 mg was 16.9 points, versus 14.5 points for placebo. Lumateperone 42 mg also met the key secondary endpoint of statistically significant improvement on the CGI-BP-S Depression Score.
Lumateperone 28 mg showed a trend for a dose-related improvement in symptoms of depression.
Though not formally tested against placebo since it did not separate on the primary endpoint, lumateperone 28 mg demonstrated a statistically significant improvement versus placebo on the CGI-BP-S. Lumateperone was generally well-tolerated with a favorable safety profile in the trial.
Adverse events were mostly mild to moderate and similar to those seen in prior studies in bipolar depression and schizophrenia, with no new adverse events observed. These findings provide further evidence supporting lumateperone’s favorable safety and tolerability profile across different patient populations.
Importantly, lumateperone was generally safe and well tolerated in the study, adds the analyst. Hazlett believes that if approved in this indication, lumateperone would be differentiated as a potential first-line treatment option in depressive episodes associated with both bipolar I and bipolar II disorders.
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.
Justice Department files False Claims Act against Teva Pharmaceuticals
The U.S. Attorney’s Office has filed a complaint under the False Claims Act against Teva Pharmaceuticals USA. and Teva Neuroscience (TEVA), the maker of Copaxone, a drug for multiple sclerosis.
The government alleges that Teva conspired with a specialty pharmacy, Advanced Care Scripts, and two purportedly independent foundations, Chronic Disease Fund and The Assistance Fund, to violate the Anti-Kickback Statute and False Claims Act by using the foundations as conduits to subsidize Medicare co-pays for Copaxone, all while steadily raising Copaxone’s price, the Justice Department said in a statement.
Teva Pharma. is accused of offering kickbacks
The government alleges that, from 2006 through at least 2015, Teva paid the two foundations well over $300M with the intent and understanding that the foundations would use Teva’s money to cover the Medicare co-pays of patients taking Copaxone. During the same period, Teva raised the price of Copaxone from approximately $17,000 per year to over $73,000 per year, according to the DOJ.
Copaxone is used to treat multiple sclerosis
Copaxone is a brand-name prescription drug. It’s approved to treat certain forms of multiple sclerosis (MS) in adults.
Chronic Disease Fund is accused of being part of the scheme
With MS, the immune system mistakenly attacks the nerves. The damaged nerves then have trouble communicating with brain. This condition can cause a wide variety of symptoms, such as muscle weakness and fatigue (lack of energy).
The Assistance Fund is accused of being part of the price kickback
Copaxone contains the active drug glatiramer acetate. It’s a disease-modifying therapy for MS. Copaxone helps to stop the immune system from attacking the nerves. The drug can reduce the number of MS relapses and also slow worsening of the disease.
Copaxone comes as a solution that’s given by subcutaneous injection (an injection under your skin).
Teva Pharmaceutical Industries Limited is an Israeli pharmaceutical company. The firm develops, manufactures, markets, and distributes generic medicines, specialty medicines, and biopharmaceutical products in North America, Europe, and internationally. It is one of the largest generic drugmakers in the world.
Shares of Teva are down 9% to $10.52 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.
