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Moderna (MRNA) Up 6% On Upbeat Data From Cancer Jab Study

Shares of Moderna (NASDAQ:MRNA) rose 6.2% on Apr 9 after management announced encouraging data from a portion of an early-stage study on mRNA-4157/V940, its investigational individualized neoantigen therapy, in certain patients with head and neck cancer. The INT is being developed in collaboration with Merck (NYSE:MRK).

This portion of the study evaluated the combination of mRNA-4157 and Merck's blockbuster immuno-oncology drug Keytruda in patients with human papillomavirus negative (HPV-) head and neck squamous cell carcinoma (HNSCC).

Data from the study showed that treatment with the combination not only achieved positive clinical responses in HPV- HNSCC patients but even showed evidence of activation of immune responses. The combined therapy was also found to be safe and well-tolerated by study participants.

Per management, treatment with the combination achieved an objective response rate (ORR) in six out of 22 patients, including two complete responses and four partial ones. The disease control rate was 63.6%. These results were presented at the American Society of Cataract and Refractive Surgery (ASCRS) earlier this week.

Moderna and Merck are already evaluating mRNA-4157 in two separate pivotal late-stage studies in melanoma and non-small cell lung cancer (NSCLC) indications. The companies intend to expand mRNA-4157 across multiple oncology indications and also start more clinical studies throughout this year. Last month, Moderna/Merck posted details on a government website of a phase II/III study evaluating the INT in patients with cutaneous squamous cell carcinoma (CSCC), a form of skin cancer.

Merck and Moderna entered a strategic partnership in 2016 to develop and market mRNA-based therapeutics to treat various types of cancer. Per the terms of the collaboration, the companies will share costs and profits equally.

Year to date, Moderna's shares have gained 12.2% against the industry's 5.4% fall.

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Unlike other therapies that are uniformly designed to treat all patients, the INT aims to bring individualized treatment to cancer patients. MRNA-4157 is tailored for each patient based on the unique mutational signature of a patient's tumor.

As opposed to traditional medications, mRNA-based therapies teach the body how to make a specific protein that can help your immune system prevent or treat certain diseases. The COVID-19 pandemic further demonstrated the significant potential of mRNA-based therapeutics. By way of COVID-19 vaccines, mRNA vaccines have generated immune responses against the virus at record-high levels compared with traditional protein-based and adeno-based vaccines.

Currently, Moderna has only one commercial product, its COVID-19 vaccine, which is currently facing a steep/significant decline in revenues, ever since the pandemic ended. Using the robust cash sales generated during the pandemic years, Moderna has significantly accelerated its pipeline development, which has shown high clinical efficacy across multiple different indications.

Moderna plans to launch 15 new marketed products, including four in rare disease indications, over the next five years. These include vaccines for RSV, CMV and influenza. Management expects potential FDA approval for its RSV vaccine mRNA-1345 in June. If approved, mRNA-1345 could be Moderna's second product launch, whose commercial launch is expected in the third quarter of 2024.

Management believes that the company's vaccine portfolio targets large addressable markets with an estimated total addressable market of around $52 billion for infectious disease vaccines.

Moderna, Inc. Price

Moderna, Inc. PriceModerna, Inc. Quote

Zacks Rank & Key Picks

Moderna currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the overall healthcare sector include ADMA Biologics (NASDAQ:ADMA) and ANI Pharmaceuticals (NASDAQ:ANIP), each sporting a Zacks Rank #1 (Strong Buy).

In the past 60 days, estimates for ADMA Biologics' 2024 earnings per share have risen from 22 cents to 30 cents. During the same period, EPS estimates for 2025 have improved from 32 cents to 50 cents. Year to date, shares of ADMA have surged 35.2%.

Earnings of ADMA Biologics beat estimates in three of the last four quarters while meeting the same on one occasion. ADMA delivered a four-quarter average earnings surprise of 85.00%.

In the past 60 days, estimates for ANI Pharmaceuticals' 2024 EPS have risen from $4.06 to $4.43. Meanwhile, during the same period, EPS estimates for 2025 have improved from $4.80 to $5.04. Year to date, shares of ANIP have risen 21.6%.

Earnings of ANI Pharmaceuticals beat estimates in each of the last four quarters. ANI delivered a four-quarter average earnings surprise of 109.06%.

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5 FDA Decisions To Watch In The Second Quarter

The year started well for the biotechnology industry. Steady dealmaking coupled with a rebound in initial public offerings helped lift investor confidence and, with it, drugmaker stocks.

While that optimism has moderated somewhat since, a series of decisions by the Food and Drug Administration over the next three months could help the sector sustain its upturn. The agency may expand approvals for two cancer cell therapies and clear a third vaccine for respiratory syncytial virus. Sanofi and Regeneron await a decision on a lucrative new use for their top-selling Dupixent, while Geron could see decades of drug research finally pay off.

Here are five FDA decisions to watch in the second quarter.

CAR-T therapies for early multiple myeloma treatment

The FDA this quarter could broaden the use of two cell therapies for multiple myeloma in decisions potentially worth billions of dollars to their respective developers.

By April 5, the agency will decide whether to clear Johnson & Johnson and Legend Biotech's Carvykti for use in myeloma patients as early as after one relapse. That decision could be accompanied by a similar OK for Bristol Myers Squibb's Abecma and open up use of the therapies, which are currently only available in the U.S. For people with more advanced disease.

European regulators have already endorsed earlier use of Abecma and Carvykti based on trial data showing they help people live longer without their disease progressing. But the FDA has appeared less supportive. The agency delayed a decision on Abecma last year and held a March advisory meeting to more closely scrutinize the data for it and Carvykti. In briefing documents, staff scientists flagged an apparent increase in the risk of death early on in the clinical trials of both therapies.

The companies attributed that finding to limitations in the "bridging therapies" study participants could receive while waiting for CAR-T treatment. Panelists were convinced and backed both, agreeing that those issues likely wouldn't occur as frequently in practice.

Nonetheless, the regulatory scrutiny suggests that the FDA might put new safety restrictions on Carvykti and Abecma if it chooses to clear them for earlier use. — Jonathan Gardner

Moderna's vaccine for RSV

COVID-19 vaccine makers Pfizer and Moderna have been facing the downside of their pandemic success, as falling sales have brought tough questions from investors.

Moderna, for its part, aims to follow its COVID shot with several other messenger RNA-based vaccines, including one for respiratory syncytial virus that's now under FDA review.

Respiratory syncytial virus, or RSV, can cause severe illness in infants, older adults and people with compromised immune systems. Two vaccines, from GSK and Pfizer, are already approved to prevent RSV-related disease in adults over the age of 60. Sales of each have been strong, although GSK has established a clear edge.

Moderna argues its vaccine, dubbed mRNA-1345, can compete. The shot is a pre-filled, single-dose vaccine, potentially offering more convenient administration.

In Phase 3 testing, the vaccine was 84% effective at preventing infection with two or more symptoms in older adults. That efficacy does appear to wane over time, however.

At a recent meeting, Centers for Disease Control and Prevention advisers reviewed known safety risks for GSK's and Pfizer's shots, both of which were associated in very rare cases with Guillain-Barré syndrome, or GBS, a neurological condition affecting the nerves. Moderna has not reported any cases of GBS in Phase 3 testing of its shot.

An FDA decision on mRNA-1345 is set for May 12. — Delilah Alvarado

Geron's imetelstat for myelodysplastic syndromes

Drug development is a time-consuming, expensive process. Geron, a biotech founded 34 years ago to pursue stem cell research, knows this well.

The company's long journey may finally reach an end by June 16, when the FDA will decide whether to approve a bone marrow disease drug Geron has been developing for nearly two decades. Called imetelstat, the drug treats myelodysplastic syndromes and some analysts believe it has blockbuster potential.

Over the many years of Geron's research, imetelstat failed studies in multiple cancer types, and the company lost Johnson & Johnson as a partner along the way. But the drug finally succeeded in Phase 3 testing for myelodysplastic syndromes last year. And in March, it secured the endorsement of an FDA advisory panel despite skepticism from agency reviewers.

Geron aims to challenge Bristol Myers Squibb's Reblozyl, first with a clearance in people with "lower-risk" disease and then in more people who have the condition. Stifel analyst Stephen Wiley estimated the drug could generate $2 billion in peak yearly sales, as it could be helpful in people who don't respond well to Reblozyl.

Geron could be a buyout target, too, according to Wiley, though the company has said it plans to sell the drug on its own. — Ben Fidler

Sanofi and Regeneron's Dupixent for COPD

Dupixent is one of the pharmaceutical industry's most widely used and lucrative products. Approved for asthma, eczema and three other inflammatory conditions, the drug earned the companies more than $11 billion in sales last year.

By June 27, the FDA could add another major indication to Dupixent's list of approved uses. The regulator is currently reviewing the injectable medicine as a treatment for a type of chronic obstructive pulmonary disease, or COPD.

If cleared by the FDA, Dupixent would be the first biologic drug for people with moderate-to-severe COPD who also have evidence of what's called Type 2 inflammation. A pair of late-stage studies, which enrolled nearly 1,900 people, showed Dupixent could cut the rate of COPD attacks by about one-third. Analysts therefore expect the FDA to issue an approval and forecast billions of dollars in additional yearly sales. — Ned Pagliarulo

Pfizer's gene therapy for hemophilia B

Some time during the second quarter, the FDA will decide whether to clear a gene therapy from Pfizer for hemophilia B, the less common form of the blood clotting disorder.

Reaching this point has been a long time coming for Pfizer, which licensed the therapy from Spark Therapeutics a decade ago. In subsequent testing, treatment lowered bleeding rates and reduced the need for regular blood transfusions — powerful benefits meant to last many years with a single infusion.

But it's unclear whether Pfizer will find a market if the FDA chooses to approve its therapy. A rival genetic medicine, from CSL Behring and UniQure, won U.S. Approval for hemophilia B in November 2022. However, few patients have been treated to date, sluggish uptake that could reflect the breadth of effective options available.

The story is similar with Roctavian, a gene therapy BioMarin Pharmaceutical sells for the more common "A" form of hemophilia. Only three patients received Roctavian last year: two in Germany and one in the U.S.

Pfizer's therapy, which in January won Canadian approval, could provide another data point. But the company seems to be downplaying its potential. In recent presentations for investors, a once-weekly shot called marstacimab has gotten higher billing. — Ned Pagliarulo

The Zacks Analyst Blog Highlights Bristol Myers, Moderna, 2seventy Bio, Amylyx Pharmaceuticals And Syros Pharmaceuticals

For Immediate Release

Chicago, IL – April 12, 2024 – Zacks.Com announces the list of stocks featured in the Analyst Blog. Every day the Zacks Equity Research analysts discuss the latest news and events impacting stocks and the financial markets. Stocks recently featured in the blog include: Bristol Myers BMY, Moderna MRNA, 2seventy bio TSVT, Amylyx Pharmaceuticals, Inc. AMLX and Syros Pharmaceuticals SYRS.

Here are highlights from Thursday's Analyst Blog:

Biotech Stock Roundup: Bristol Myers, Moderna, etc.

It was a regular week in the biotech sector with a focus on pipeline and regulatory updates. While smaller biotechs were in focus on regulatory updates, bigwigs like Bristol Myers and Moderna gained the spotlight on drug label expansion and pipeline data, respectively.

Recap of the Week's Most Important Stories:

Updates from BMY: Bristol Myers announced that the FDA approved a label expansion for chimeric antigen receptor (CAR) T cell immunotherapy Abecma. The therapy is now approved for the treatment of adult patients with relapsed or refractory multiple myeloma after two or more prior lines of therapy, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 monoclonal antibody.

It is to be noted that Abecma is being jointly developed and commercialized in the United States by Bristol Myers Squibb and 2seventy bio. Outside the United States, BMY assumes the sole responsibility for Abecma's manufacturing and commercialization. The latest approval, granted on Apr 4, was based on results from the KarMMa-3 trial, wherein Abecma tripled progression-free survival compared to standard regimens, with a 51% reduction in risk of disease progression or death and a well-established safety profile.

The expanded approval brings this personalized CAR T cell therapy to more patients with relapsed or refractory multiple myeloma earlier in their treatment journey as a one-time infusion offering meaningful treatment-free intervals when responding to the therapy.

Story continues

An approval was mostly expected as the FDA Oncologic Drugs Advisory Committee recently voted in favor (8:3) of Abecma's benefit/risk profile for patients with triple-class exposed relapsed or refractory multiple myeloma. BMY and TSVT were expecting a decision in December but the FDA delayed and stated that the ODAC will meet to review data supporting the application for Abecma.

Abecma is already approved in the United States for adult patients with triple-class exposed relapsed or refractory multiple myeloma after four or more prior lines of therapy. Abecma was recently approved in Japan, Switzerland and the European Union for adult patients with triple-class exposed relapsed and/or refractory multiple myeloma after two prior lines of therapy.

BMY also announced data from the cohorts of the KRYSTAL-1 study, evaluating the lung cancer drug Krazati (adagrasib) in combination with Erbitux (cetuximab), for the treatment of patients with previously treated KRASG12C-mutated locally advanced or metastatic colorectal cancer (CRC).

The combination of Krazati and cetuximab demonstrated clinically meaningful activity as a targeted treatment option for CRC patients. The combination demonstrated an objective response rate of 34% with a median follow-up of 11.9 months in 94 patients. The median progression-free survival and the median overall survival was 6.9 months and 15.9 months, respectively, in pre-treated patients with KRASG12C-mutated locally advanced or metastatic CRC.

BMY currently has a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

Moderna Up on Vaccine Data: Moderna announced encouraging data from a portion of an early-stage study on mRNA-4157/V940, its investigational individualized neoantigen therapy (INT), in certain patients with head and neck cancer. Its shares gained on the news.

The INT is being developed in collaboration with Merck. This portion of the study evaluated the combination of mRNA-4157 and Merck's blockbuster immuno-oncology drug Keytruda in patients with human papillomavirus negative (HPV-) head and neck squamous cell carcinoma (HNSCC).

Data from the study showed that treatment with the combination not only achieved positive clinical responses in HPV- HNSCC patients but also showed evidence of activation of immune responses. The combined therapy was also found to be safe and well-tolerated by study participants.

Per management, treatment with the combination achieved an objective response rate in six out of 22 patients, including two complete responses and four partial ones. The disease control rate was 63.6%. These results were presented at the American Society of Cataract and Refractive Surgery, earlier this week.

Amylyx Withdraws Drug, Reduces Jobs: Amylyx Pharmaceuticals, Inc. Announced that it will remove its amyotrophic lateral sclerosis (ALS) drug Relyvrio (sodium phenylbutyrate and taurursodiol, also known as AMX0035) from the U.S. Markets and it has already started a process with the FDA on the same. The drug, approved under the brand name Albrioza in Canada, will also be removed from Canadian markets.

While the formal decision to remove the drug from these markets has come recently, the company was contemplating the same following the failure of the late-stage PHOENIX study last month, which led to a massive crash in the company's shares. PHOENIX did not meet its primary endpoint of reaching statistical significance as measured by a change from baseline in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) total score at week 48. The study also failed to achieve statistical significance in secondary endpoints.

Consequently, Relyvrio/Albrioza will no longer be available for new patients. Nevertheless, the patients currently on therapy in the United States and Canada who, in consultation with their physician, wish to continue can be transitioned to a free drug program.

Meanwhile, the PHOENIX Open Label Extension is ongoing. Amylyx will now focus on advancing two key programs investigating its lead candidate, AMX0035, in Wolfram syndrome and progressive supranuclear palsy and AMX0114, an antisense oligonucleotide targeting calpain-2, in ALS.

Concurrently, Amylyx announced a restructuring plan, whereby it will reduce its workforce by approximately 70% and decrease financial commitments outside its priority areas. Amylyx expects to have a cash runway into 2026 by undertaking this plan.

Regulatory Update from Syros: Syros Pharmaceuticals announced that the FDA has granted Fast Track Designation to tamibarotene for the treatment of newly diagnosed acute myeloid leukemia (AML). The designation was granted to tamibarotene, in combination with azacitidine and venetoclax, for the treatment of newly diagnosed AML with RARA overexpression, as detected by an FDA-approved test in adults aged more than 75 years or who have comorbidities that preclude the use of intensive induction chemotherapy. Shares were up on the news.

The Fast Track designation allows therapeutic candidates for priority review and accelerated approval if supported by clinical data.

SYRS is currently developing tamibarotene, an oral, selective, retinoic acid receptor alpha or RARα agonist in combination studies as a frontline treatment of AML and higher-risk myelodysplastic syndrome (HR-MDS). The FDA also granted a Fast Track Designation to tamibarotene for treating HR-MDS.

Performance

The Nasdaq Biotechnology Index has gained 0.41% in the past five trading sessions and Moderna's shares have increased 7.72% during the same time frame. Over the past six months, shares of VRTX have risen 13.87%. (See the last biotech stock roundup here: Biotech Stock Roundup: IRON, VERV Down on Study Updates, BMYs Drug News & More)

What's Next in Biotech?

Stay tuned for pipeline updates.

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