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Five Key Precision Medicine Players With Products in the Market

Precision medicines are gaining increasing traction in healthcare. Here are five companies driving the field with recent approvals in RNA therapeutics,…

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Precision medicines are gaining increasing traction in healthcare. Here are five companies driving the field with recent approvals in RNA therapeutics, cell and gene therapies, and more.

Medicines have traditionally been given to patients in a one-size-fits-all approach, with many patients failing to benefit and experiencing side effects. This challenge is leading the field to shift toward precision medicine, where treatments are tailored to specific disease or patient subgroups to increase their effectiveness.

Precision medicine was once limited to the realm of science fiction but now more than a quarter of all new therapeutics approved since 2015 have a precision or personalized component, according to the Personalized Medicine Coalition. This covers a wide range of modalities, including small molecules, antibodies and cell and gene therapies. It also provides treatment alternatives for people with cancer in addition to rare conditions such as spinal muscular atrophy (SMA), Duchenne muscular dystrophy and hemophilia.

The market for precision medicines was valued at around $29.1 billion in 2023 and is predicted to grow to $50.2 billion by 2028, according to a report by MarketsandMarkets. This growth is expected to be fuelled by the increasing presence of targeted therapeutics in the market. It is also flourishing as high-throughput DNA sequencing and artificial intelligence (AI)-driven analytics become more accessible to help physicians identify which patients can benefit the most from a treatment.

The lightning growth of precision medicine in healthcare is attracting the attention of investors, particularly in oncology and in drug discovery. In a recent survey, more than half of life sciences investors were interested in opportunities in precision medicine along with AI and cell and gene therapy. And a plethora of big pharma companies are building up their precision medicine expertise, including Novartis, Pfizer, Roche, Johnson & Johnson, and more.

The last couple of years alone has seen some monumental approvals by the U.S. Food and Drug Administration (FDA) in precision medicine. Here are five players that have made a big impact on precision medicine based on recent approvals in the field.

 

1. Alnylam Pharmaceuticals
Founded: 2002 | Headquarters: Cambridge, Massachusetts, U.S.
FDA approval: Amvuttra, 2022

Alnylam focuses its research and development on drugs based on RNA. The name is derived from Alnilam, the center star in the Orion’s belt constellation, and also means a “string of pearls” in Arabic, which is a reference to the nucleotide strands that make up RNA.

In particular, Alnylam develops therapeutics based on a type of RNA called small interfering (si)RNAs. The drugs, delivered into patients via lipid nanoparticles, selectively silence RNAs that would normally be transcribed into proteins in the cell. This makes them prime candidates for taking on rare diseases caused by mutations in vital proteins.

After many ups and downs over its development, Alnylam achieved its first FDA drug approval in 2018, namely of Onpattro (patisiran) for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR).

Over the following years, Alnylam received subsequent FDA green lights for other products Givlaari (givosiran) and Oxlumo (lumasiran), with the latest approval being for Amvuttra (vutrisiran) for the treatment of polyneuropathy of hATTR.

Amvuttra is designed to be administered subcutaneously once every three months, potentially making it more comfortable for patients than Onpattro, which is administered via intravenous infusion once every three weeks. Amvuttra was approved based on results from a Phase III trial where it beat the placebo group at reducing disease severity.

Alnylam is now testing Amvuttra with a biannual regimen and as a treatment for patients with ATTR amyloidosis with cardiomyopathy. Among other candidates, the firm is also developing the drug fitusiran in Phase III for the treatment of hemophilia.

 

2. Biogen
Founded: 1978 | Headquarters: Cambridge, Massachusetts, U.S.
FDA approval: Qalsody, 2023

biogen logo

Biogen began its life as Biotechnology Geneva and evolved into its final form following a number of mergers over the years.

The company has had a rollercoaster history. Over the last decade, it has hit the headlines with highlights such as the approval of the SMA therapy Spinraza (nusersin) in 2016 and the controversial accelerated approval of its antibody treatment Aduhelm (aducanumab) in 2021, despite lacking evidence of its clinical benefit in patients with Alzheimer’s disease.

The accelerated approval of Biogen’s candidate Qalsody (tofersen) this year marked the fourth approved treatment for patients with the rare disease amyotrophic lateral sclerosis (ALS). The antisense oligonucleotide drug, product of a collaboration between Biogen and Ionis Pharmaceuticals, is the first approved therapy to block mutated forms of SOD1 that occur in rare cases of ALS.

The approval decision was based on Qalsody’s ability to reduce plasma levels of neurofilament light chain (NfL), an emerging biomarker of neurodegeneration. The company also noted trends of improvement in disease symptoms in the treatment group, but they were not statistically significant. Therefore, Biogen will still need to back up the clinical benefit in confirmatory trials going forward.

In addition to its approved products, Biogen is developing a pipeline of treatments for conditions including systemic lupus erythematosus, acute ischemic stroke and essential tremor.

 

3. BioMarin
Founded: 1997 | Headquarters: Novato, California, U.S.
FDA approval: Rocktavian, 2023

BioMarin focuses on the development of treatments for rare diseases underserved by current treatments. One example is Aldurazyme, which the company says was inspired by the experience of a father wanting to develop a treatment for his son with the condition mucopolysaccharidosis I.

The firm has also marketed treatments for other rare diseases including Maroteaux–Lamy syndrome, Lambert–Eaton myasthenic syndrome, Morquio A syndrome and more.

BioMarin’s latest approval by the FDA was the gene therapy Rocktavian in 2023. The therapy is designed to tackle hemophilia A, a bleeding disorder caused by a mutation in a gene encoding the protein factor VIII (FVIII).

The standard treatment for hemophilia A is a lifelong preventative therapy involving regular injections of healthy FVIII. In contrast, Rocktavian is a one-off injection providing a functional copy of FVIII, reducing the amount of prophylactic treatment the patient needs.

Rocktavian’s first approval was by the European Medicines Agency (EMA) in 2022. The regulators’ decisions were based on data from a phase 3 trial where those receiving the gene therapy saw a drop in their annualized bleeding rate while receiving FVIII prophlaxis.

BioMarin sees approximately 2,500 patients that could be eligible for Rocktavian in the U.S. According to Clarivate, Rocktavian has the potential to become a blockbuster.

Rocktavian’s EU approval also coincided with the FDA greenlight of Hemgenix, the first gene therapy for hemophilia B developed by uniQure and CSL Behring.

 

4. Johnson & Johnson
Founded: 1886 | Headquarters: New Brunswick, New Jersey, U.S.
FDA approval: Carvykti, 2022

CAR-T cell therapies have caught the imagination of the biotech and pharma industry for their potential to effectively cure forms of blood cancer in some patients. The therapies involve extracting a patient’s immune T cells, engineering them in a lab to hunt down cancer cells and then reinfusing them into the patient.

The first CAR-T therapies—Novartis’ Kymriah and Gilead’s Yescarta—were approved by the FDA in 2017 as treatments of the blood cancers B-cell precursor acute lymphoblastic leukemia and large B-cell lymphoma respectively. They were approved for use only in patients whose cancers had relapsed after multiple lines of cancer therapy.

In 2017, the Janssen Pharmaceutical Companies of Johnson & Johnson teamed up with the Chinese company Legend Biotech to get their own candidate in the CAR-T race. The partnership paid off in 2022 when Carvykti (ciltacabtagene autoleucel) gained FDA market approval for the fifth-line treatment of multiple myeloma.

Of the current CAR-T therapies in the market, Carvykti has the most in common with Abecma, which was developed by Bristol-Myers Squibb. Abecma was approved by the FDA in 2021, also for the fifth-line treatment of multiple myeloma. The main differences between the two are that they bind slightly differently to their target, BCMA, and Carvykti has a lower recommended dose.

As with all approved CAR-T therapy developers, Janssen is striving to have Carvykti approved for earlier stages of blood cancer treatment, which will allow the therapy to reach more patients.

 

5. Novartis
Founded: 1996 | Headquarters: Basel, Switzerland
FDA approval: Pluvicto, 2022

Novartis was born as a result of a merger between the companies Ciba-Geigy and Sandoz. The name is derived from the Latin phrase “novae artes,” or “new skills,” which the company says embodies its commitment to research and development in new medicines.

The big pharma company has a wide range of products on the market, and one recent approval, that of Pluvicto (Lutetium (177Lu) vipivotide tetraxetan) in 2022, adds weight to the emerging field of radiopharmaceuticals: drugs that carry a radiotherapy agent directly to a tumor.

Novartis’s strong interest in radiopharmaceuticals led it to acquire key developers of these therapies, including Advanced Accelerator Applications (AAA) in 2017 and Endocyte in 2018. The takeover of AAA gained Novartis access to the approved radiopharmaceutical Lutathera for the treatment of neuroendocrine tumors. Meanwhile, the incorporation of Endocyte let Novartis snap up what would later be Pluvicto, which is indicated for the treatment for a type of metastatic prostate cancer.

Radiopharmaceutical drugs provide a more targeted approach to cancer treatment than regular radiotherapy, which typically involves beaming radiation into a wide area in the patient’s body. The compounds zoom in on cancer cells expressing a particular target—PSMA in the case of Pluvicto—and blast them at close range. In parallel with Pluvicto, the FDA greenlit a complementary diagnostic imaging agent, Locametz, to identify patients that were likely to benefit from the therapy.

Radiopharmaceuticals are a hot commodity in oncology right now and other companies working in this field include Bayer and ITM Isotope Technologies Munich along with startups such as Rayze Bio and Aktis Oncology.

 

Jonathan Smith is a freelance science journalist based in the U.K. and Spain. He previously worked in Berlin as reporter and news editor at Labiotech, a website covering the biotech industry. Prior to this, he completed a PhD in behavioral neurobiology at the University of Leicester and freelanced for the U.K. organizations Research Media and Society of Experimental Biology. He has also written for medwireNews, Biopharma Reporter and Outsourcing Pharma.

The post Five Key Precision Medicine Players With Products in the Market appeared first on Inside Precision Medicine.

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Four burning questions about the future of the $16.5B Novo-Catalent deal

To build or to buy? That’s a classic question for pharma boardrooms, and Novo Nordisk is going with both.
Beyond spending billions of dollars to expand…

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To build or to buy? That’s a classic question for pharma boardrooms, and Novo Nordisk is going with both.

Beyond spending billions of dollars to expand its own production capacity for its weight loss drugs, the Danish drugmaker said Monday it will pay $11 billion to acquire three manufacturing plants from Catalent. It’s part of a broader $16.5 billion deal with Novo Holdings, the investment arm of the pharma’s parent group, which agreed to acquire the contract manufacturer and take it private.

It’s a big deal for all parties, with potential ripple effects across the biotech ecosystem. Here’s a look at some of the most pressing questions to watch after Monday’s announcement.

Why did Novo do this?

Novo Holdings isn’t the most obvious buyer for Catalent, particularly after last year’s on-and-off M&A interest from the serial acquirer Danaher. But the deal could benefit both Novo Holdings and Novo Nordisk.

Novo Nordisk’s biggest challenge has been simply making enough of the weight loss drug Wegovy and diabetes therapy Ozempic. On last week’s earnings call, Novo Nordisk CEO Lars Fruergaard Jørgensen said the company isn’t constrained by capital in its efforts to boost manufacturing. Rather, the main challenge is the limited amount of capabilities out there, he said.

“Most pharmaceutical companies in the world would be shopping among the same manufacturers,” he said. “There’s not an unlimited amount of machinery and people to build it.”

While Novo was already one of Catalent’s major customers, the manufacturer has been hamstrung by its own balance sheet. With roughly $5 billion in debt on its books, it’s had to juggle paying down debt with sufficiently investing in its facilities. That’s been particularly challenging in keeping pace with soaring demand for GLP-1 drugs.

Novo, on the other hand, has the balance sheet to funnel as much money as needed into the plants in Italy, Belgium, and Indiana. It’s also struggled to make enough of its popular GLP-1 drugs to meet their soaring demand, with documented shortages of both Ozempic and Wegovy.

The impact won’t be immediate. The parties expect the deal to close near the end of 2024. Novo Nordisk said it expects the three new sites to “gradually increase Novo Nordisk’s filling capacity from 2026 and onwards.”

As for the rest of Catalent — nearly 50 other sites employing thousands of workers — Novo Holdings will take control. The group previously acquired Altasciences in 2021 and Ritedose in 2022, so the Catalent deal builds on a core investing interest in biopharma services, Novo Holdings CEO Kasim Kutay told Endpoints News.

Kasim Kutay

When asked about possible site closures or layoffs, Kutay said the team hasn’t thought about that.

“That’s not our track record. Our track record is to invest in quality businesses and help them grow,” he said. “There’s always stuff to do with any asset you own, but we haven’t bought this company to do some of the stuff you’re talking about.”

What does it mean for Catalent’s customers? 

Until the deal closes, Catalent will operate as a standalone business. After it closes, Novo Nordisk said it will honor its customer obligations at the three sites, a spokesperson said. But they didn’t answer a question about what happens when those contracts expire.

The wrinkle is the long-term future of the three plants that Novo Nordisk is paying for. Those sites don’t exclusively pump out Wegovy, but that could be the logical long-term aim for the Danish drugmaker.

The ideal scenario is that pricing and timelines remain the same for customers, said Nicole Paulk, CEO of the gene therapy startup Siren Biotechnology.

Nicole Paulk

“The name of the group that you’re going to send your check to is now going to be Novo Holdings instead of Catalent, but otherwise everything remains the same,” Paulk told Endpoints. “That’s the best-case scenario.”

In a worst case, Paulk said she feared the new owners could wind up closing sites or laying off Catalent groups. That could create some uncertainty for customers looking for a long-term manufacturing partner.

Are shareholders and regulators happy? 

The pandemic was a wild ride for Catalent’s stock, with shares surging from about $40 to $140 and then crashing back to earth. The $63.50 share price for the takeover is a happy ending depending on the investor.

On that point, the investing giant Elliott Investment Management is satisfied. Marc Steinberg, a partner at Elliott, called the agreement “an outstanding outcome” that “clearly maximizes value for Catalent stockholders” in a statement.

Elliott helped kick off a strategic review last August that culminated in the sale agreement. Compared to Catalent’s stock price before that review started, the deal pays a nearly 40% premium.

Alessandro Maselli

But this is hardly a victory lap for CEO Alessandro Maselli, who took over in July 2022 when Catalent’s stock price was north of $100. Novo’s takeover is a tacit acknowledgment that Maselli could never fully right the ship, as operational problems plagued the company throughout 2023 while it was limited by its debt.

Additional regulatory filings in the next few weeks could give insight into just how competitive the sale process was. William Blair analysts said they don’t expect a competing bidder “given the organic investments already being pursued at other leading CDMOs and the breadth and scale of Catalent’s operations.”

The Blair analysts also noted the companies likely “expect to spend some time educating relevant government agencies” about the deal, given the lengthy closing timeline. Given Novo Nordisk’s ascent — it’s now one of Europe’s most valuable companies — paired with the limited number of large contract manufacturers, antitrust regulators could be interested in taking a close look.

Are Catalent’s problems finally a thing of the past?

Catalent ran into a mix of financial and operational problems over the past year that played no small part in attracting the interest of an activist like Elliott.

Now with a deal in place, how quickly can Novo rectify those problems? Some of the challenges were driven by the demands of being a publicly traded company, like failing to meet investors’ revenue expectations or even filing earnings reports on time.

But Catalent also struggled with its business at times, with a range of manufacturing delays, inspection reports and occasionally writing down acquisitions that didn’t pan out. Novo’s deep pockets will go a long way to a turnaround, but only the future will tell if all these issues are fixed.

Kutay said his team is excited by the opportunity and was satisfied with the due diligence it did on the company.

“We believe we’re buying a strong company with a good management team and good prospects,” Kutay said. “If that wasn’t the case, I don’t think we’d be here.”

Amber Tong and Reynald Castañeda contributed reporting.

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Petrina Kamya, Ph.D., Head of AI Platforms at Insilico Medicine, presents at BIO CEO & Investor Conference

Petrina Kamya, PhD, Head of AI Platforms and President of Insilico Medicine Canada, will present at the BIO CEO & Investor Conference happening Feb….

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Petrina Kamya, PhD, Head of AI Platforms and President of Insilico Medicine Canada, will present at the BIO CEO & Investor Conference happening Feb. 26-27 at the New York Marriott Marquis in New York City. Dr. Kamya will speak as part of the panel “AI within Biopharma: Separating Value from Hype,” on Feb. 27, 1pm ET along with Michael Nally, CEO of Generate: Biomedicines and Liz Schwarzbach, PhD, CBO of BigHat Biosciences.

Credit: Insilico Medicine

Petrina Kamya, PhD, Head of AI Platforms and President of Insilico Medicine Canada, will present at the BIO CEO & Investor Conference happening Feb. 26-27 at the New York Marriott Marquis in New York City. Dr. Kamya will speak as part of the panel “AI within Biopharma: Separating Value from Hype,” on Feb. 27, 1pm ET along with Michael Nally, CEO of Generate: Biomedicines and Liz Schwarzbach, PhD, CBO of BigHat Biosciences.

The session will look at how the latest artificial intelligence (AI) tools – including generative AI and large language models – are currently being used to advance the discovery and design of new drugs, and which technologies are still in development. 

The BIO CEO & Investor Conference brings together over 1,000 attendees and more than 700 companies across industry and institutional investment to discuss the future investment landscape of biotechnology. Sessions focus on topics such as therapeutic advancements, market outlook, and policy priorities.

Insilico Medicine is a leading, clinical stage AI-driven drug discovery company that has raised over $400m in investments since it was founded in 2014. Dr. Kamya leads the development of the Company’s end-to-end generative AI platform, Pharma.AI from Insilico’s AI R&D Center in Montreal. Using modern machine learning techniques in the context of chemistry and biology, the platform has driven the discovery and design of 30+ new therapies, with five in clinical stages – for cancer, fibrosis, inflammatory bowel disease (IBD), and COVID-19. The Company’s lead drug, for the chronic, rare lung condition idiopathic pulmonary fibrosis, is the first AI-designed drug for an AI-discovered target to reach Phase II clinical trials with patients. Nine of the top 20 pharmaceutical companies have used Insilico’s AI platform to advance their programs, and the Company has a number of major strategic licensing deals around its AI-designed therapeutic assets, including with Sanofi, Exelixis and Menarini. 

 

About Insilico Medicine

Insilico Medicine, a global clinical stage biotechnology company powered by generative AI, is connecting biology, chemistry, and clinical trials analysis using next-generation AI systems. The company has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques for novel target discovery and the generation of novel molecular structures with desired properties. Insilico Medicine is developing breakthrough solutions to discover and develop innovative drugs for cancer, fibrosis, immunity, central nervous system diseases, infectious diseases, autoimmune diseases, and aging-related diseases. www.insilico.com 


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Another country is getting ready to launch a visa for digital nomads

Early reports are saying Japan will soon have a digital nomad visa for high-earning foreigners.

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Over the last decade, the explosion of remote work that came as a result of improved technology and the pandemic has allowed an increasing number of people to become digital nomads. 

When looked at more broadly as anyone not required to come into a fixed office but instead moves between different locations such as the home and the coffee shop, the latest estimate shows that there were more than 35 million such workers in the world by the end of 2023 while over half of those come from the United States.

Related: There is a new list of cities that are best for digital nomads

While remote work has also allowed many to move to cheaper places and travel around the world while still bringing in income, working outside of one's home country requires either dual citizenship or work authorization — the global shift toward remote work has pushed many countries to launch specific digital nomad visas to boost their economies and bring in new residents.

Japan is a very popular destination for U.S. tourists. 

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This popular vacation destination will soon have a nomad visa

Spain, Portugal, Indonesia, Malaysia, Costa Rica, Brazil, Latvia and Malta are some of the countries currently offering specific visas for foreigners who want to live there while bringing in income from abroad.

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With the exception of a few, Asian countries generally have stricter immigration laws and were much slower to launch these types of visas that some of the countries with weaker economies had as far back as 2015. As first reported by the Japan Times, the country's Immigration Services Agency ended up making the leap toward a visa for those who can earn more than ¥10 million ($68,300 USD) with income from another country.

The Japanese government has not yet worked out the specifics of how long the visa will be valid for or how much it will cost — public comment on the proposal is being accepted throughout next week. 

That said, early reports say the visa will be shorter than the typical digital nomad option that allows foreigners to live in a country for several years. The visa will reportedly be valid for six months or slightly longer but still no more than a year — along with the ability to work, this allows some to stay beyond the 90-day tourist period typically afforded to those from countries with visa-free agreements.

'Not be given a residence card of residence certificate'

While one will be able to reapply for the visa after the time runs out, this can only be done by exiting the country and being away for six months before coming back again — becoming a permanent resident on the pathway to citizenship is an entirely different process with much more strict requirements.

"Those living in Japan with the digital nomad visa will not be given a residence card or a residence certificate, which provide access to certain government benefits," reports the news outlet. "The visa cannot be renewed and must be reapplied for, with this only possible six months after leaving the countr

The visa will reportedly start in March and also allow holders to bring their spouses and families with them. To start using the visa, holders will also need to purchase private health insurance from their home country while taxes on any money one earns will also need to be paid through one's home country.

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