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  • Ultragenyx Rare Disease Voucher Sales Secure $413M for More Potential Pipeline Building

    Ultragenyx Rare Disease Voucher Sales Secure $413M for More Potential Pipeline Building

    Ultragenyx recently received two separate FDA approvals for gene therapies in the span of a month, each regulatory nod accompanied by a voucher the company could use to speed up the future review of another rare disease drug. In the past week, Ultragenyx has reached separate deals to sell each regulatory fast pass.

    The Novato, California-based biotech announced Wednesday a definitive agreement to sell a voucher for $203 million, following a deal last week to sell a voucher for $210 million. The identities of both voucher buyers were undisclosed and the purchases still need to meet closing conditions. But the deals put Ultragenyx in line for non-dilutive capital that the rare disease specialist can use to bolster its drug pipeline.

    Congress created the voucher program to incentivize drug development in rare diseases. Companies awarded a regulatory approval in a rare pediatric disease can receive one of these vouchers, which cuts the standard review time of 10 months down to six. These vouchers may be sold, and many voucher recipients opt to sell them to big pharmaceutical companies. Demand for these regulatory fast passes has pushed prices higher; most of the vouchers sold this year fetched prices of $200 million or more.

    Ultragenyx received a voucher alongside the late August approval of Gengylcos, a gene therapy for a rare inherited enzyme deficiency called glycogen storage disease type Ia. Another voucher came from the mid-September FDA nod for Fayuvi, a gene therapy for a different rare inherited enzyme deficiency called Sanfilippo syndrome Type A. Each product is the first approved therapy for its respective ultra-rare indication.

    While the Ultragenyx gene therapies carry multi-million-dollar price tags, neither product is expected to become a big revenue driver. Much of the company’s future financial expectations had rested on apazunersen, which was in development for Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. Last month, Ultragenyx reported this antisense oligonucleotide drug failed its Phase 3 test. The company said the disappointing trial outcome would lead to a corporate restructuring and expense reductions.

    The Angelman drug came from an acquisition. Ultragenyx’s two gene therapies also came from business deals. The capital from the voucher sales could be applied toward another deal for a different rare disease therapeutic candidate. While the company made clear its intent to sell both vouchers, it has not said how it will use the proceeds. As of the end of June, Ultragenyx reported its cash position was $436 million. The company is expected to report third quarter 2026 financial results in November.

    Ultragenyx previously received vouchers for rare disease drug approvals in 2017 and 2018. In a prepared statement, Chief Financial Officer Howard Horn noted that the Sanfilippo gene therapy benefited from capital generated by a previous voucher sale, “demonstrating the important role the [priority review voucher] program plays in helping companies develop transformative therapies for rare disease patients.”

    In a Wednesday research note, William Blair analyst Lachlan Hanbury-Brown said that even though Ultragenyx is launching two new gene therapies, the company’s pipeline lacks any clinical catalysts to drive value for the company’s stock in the next year. But the sale of the two vouchers significantly bolsters the biotech’s balance sheet with non-dilutive capital.

    “We look forward to hearing how management plans to strategically deploy this additional capital, as well as details on the company’s expense reductions plans, which we believe will help achieve [generally accepted accounting principles] profitability in 2027,” Hanbury-Brown said.

    Photo: CaptureTheWorld, Getty Images

  • What Corporate Venture Funds Get Wrong about Strategic Value

    What Corporate Venture Funds Get Wrong about Strategic Value

    A venture fund’s objective is to invest capital into early-stage companies to fuel growth, increasing the probability that they are acquired or reach public markets. That’s how an “exit” is achieved, with the invested capital returned at a risk-adjusted profit. Corporate venture capital (CVC) differs from orthodox venture capital because, alongside financial returns, a CVC’s goal is often to create strategic value for a core business.

    Delivering this strategic value is much easier said than done, and therefore not well addressed by most CVC funds. 

    Outlined below are four distinct practices that optimize strategic and financial value. 

    Define strategic value alongside financial return

    Strategic value comes in many forms. Pharma corporate venture should develop investments that might generate a valuable new target, drug, or vaccine candidate, improve the probability of pipeline success, reduce the cost of R&D, accelerate clinical trials — and more through the entire value chain. It is not necessary to acquire such businesses outright, but it is necessary to cultivate them into standardized, scaled solutions for scientists, regulators, clinicians, patients, and payors.

    Assessing the value of an asset — financial and/or strategic — is as much art as science. Financial value can be estimated using discounted cash flow analysis, which relies on pro forma projections and assumptions about future market pricing and multiples, or through comparable-company analysis. Quantifying strategic value is much more difficult. One derivative indicator of strategic value is whether the core CVC business engages with the portfolio company, through piloting, partnering, licensing, and/or purchasing products or services. A CVC that can’t generate this type of engagement is just delivering capital without a strategic return. 

    Lead internal collaborations that power sourcing and diligence

    Placing relevant strategic investments requires deep familiarity with the parent company’s objectives and priorities. This means dedicated internal workstreams with business leaders and subject matter experts who help refine and prioritize use cases and understand unmet needs. In-house CVC resources also help with pipeline building and technical, scientific, and clinical due diligence. Proper engagement is time-consuming and requires operational, process, and communication excellence — skills anathema to the normal concerns of most venture investors.

    Many benefits accrue from generating that upfront buy-in. Deep collaboration raises the CVC’s probability of succeeding in highly scientific and technical markets. It also instills early business awareness and engagement, so portfolio companies develop faster pilots and larger partnerships, executable in a shorter timeframe.

    Drive leverage through syndication

    Most CVC investors are equipped to lead negotiations and price rounds, but there’s a strong case for prioritizing syndicate creation. 

    Syndication provides leverage through agile, coordinated activity on negotiating terms, dividing or delegating board activities, and considering future mergers and acquisitions. It also extends financial leverage, allowing a fund to cover more of the market and back a broader range of strategic interest areas. Joining syndicated deals priced by expert financial investors tends to produce a fair market price. A CVC can always walk away when pricing runs ahead of what its internal modeling supports. 

    Even with reduced board involvement, a fund can stay engaged through shareholder and information rights — quarterly financials, board materials, regular check-ins — whether it holds a board or observer seat or is simply an shareholder. 

    Occasionally, a CVC will need to play a more significant company-building role, In such cases it can place a convertible note or lead a round outright — but always with an eye toward delegating responsibility over time. The correct level of involvement is a case-by-case decision, considering the strategic relevance of the platform, the stage of the company, and the experience of its board and management team.

    Deliver the payoff 

    None of this works as one single move, but as a pattern: sourcing shaped by internal collaboration, pricing and development sharpened by syndication, and continued engagement all the way to exit. Funds that operate this way tend to see their strategic and financial returns reinforce each other over time rather than trade off against one another.

    The key lies in recruiting and training leaders who roll up their sleeves and work seamlessly with internal partners. When portfolio investments help a business develop better therapies or reach patients faster, they deliver strategic value — and ultimately value to patients and providers.

    Photo: Hong Li, Getty Images

    David M. Rubin

    David M. Rubin, Ph.D., is a Managing Director in Merck GHIF, where he leads the drug discovery and precision medicine practice. Dr. Rubin ran and helped build life sciences companies in the USA, UK, Denmark, Hungary, and India. He was awarded National Institutes of Health and American Cancer Society Fellowships to support his research at Harvard Medical School on ubiquitin-dependent protein degradation. He received a B.A. in biology from SUNY Binghamton and a Ph.D. from Temple University. Junk Birds, his collection of previously published short stories, was published in 2025.

    This post appears through the MedCity Influencers program. Anyone can publish their perspective on business and innovation in healthcare on MedCity News through MedCity Influencers. Click here to find out how.

  • Asahi Kasei Acquisition Pays Off With FDA Approval for Novel Herpes Drug

    Asahi Kasei Acquisition Pays Off With FDA Approval for Novel Herpes Drug

    Patients with lesions caused by herpes simplex virus have a new FDA-approved treatment option, an oral antiviral drug from Asahi Kasei.

    The late Friday regulatory decision covers the treatment of adults whose lesions do not respond to the three oral antivirals that make up the current standard of case. Asahi Kasei’s once-daily pill, known in development as pritelivir, will be commercialized under the brand name Hovilpri.

    In people with healthy immune systems, herpes simplex virus (HSV) infection is often asymptomatic or causes mild disease. But in people with weakened immune symptoms — those who have undergone a transplant procedure, or patients with HIV, cancer, or autoimmune disorders — HSV infection can occur more frequently and with greater severity. While oral antivirals are available to treat HSV, viral mutations can lead to resistance to these older drugs. There are alternative treatment options, but they require intravenous dosing.

    Hovilpri gives patients the choice of an oral drug with a different mechanism of action. This small molecule is designed to inhibit a complex of enzymes key to the synthesis of viral DNA. The drug was developed to treat both types of HSV, HSV-1 and HSV-2.

    The regulatory submission for Hovilpri was based on the results of a Phase 3 study that showed statistically significant superiority in lesion healing up to day 28 compared to the physician’s choice of treatment. The most common adverse reaction reported for the study drug was headache.

    “Today’s approval delivers the first FDA-approved treatment for refractory mucocutaneous HSV lesions with a novel mechanism of action in nearly 30 years and marks an important advancement for immunocompromised adults,” Stacy Wheeler, CEO of Asahi Kasei Therapeutics in the U.S. and Europe, said in a prepared statement. “Hovilpri offers a new oral treatment option for adult patients with a persistent, difficult-to-treat disease.”

    Hovilpri was originally developed by AiCuris Anti-Infective Cures, a Germany-based biopharmaceutical company acquired by Asahi Kasei for €780 million (about $919 million). In the February acquisition announcement, Tokyo-based Asahi Kasei said the deal strengthens the company’s presence in severe infectious disease. It added that HSV patients addressed by Hovilpri are commonly seen in transplant centers and specialized hospital settings where the company has established commercial relationships and infrastructure.

    Asahi Kasei has been expanding its portfolio and pipeline via business deals. In 2020, the company closed the 8.3 billion Danish krone (about $1.3 billion) acquisition of Veloxis Pharmaceuticals, a company that develops and sells therapies for organ transplant patients. The Japanese company’s 11.8 billion Swedish krona (about $1.18 billion) acquisition of Calliditas Pharmaceuticals in 2024 brought a portfolio that includes Tarpeyo, which has FDA approval for treating the rare kidney disorder immunoglobulin A nephropathy.

    The Aicuris acquisition brought more than Hovilpri. Asahi Kasei gains royalty income from Prevymis, a cytomegalovirus drug whose global rights Aicuris had licensed to Merck. The Japanese company also gets an experimental antisense oligonucleotide that Aicuris has advanced to Phase 1 testing as a treatment for BK virus infection in kidney transplant patients.

    Photo by Asahi Kasei

  • What Life Sciences Commercial Teams Can Learn from Digital Health

    What Life Sciences Commercial Teams Can Learn from Digital Health

    A cursory glance across job descriptions at most major pharmaceutical companies will, if one is being unkind, look essentially identical to the 2010s: the same divisional divide of analytics, field, launch, CRM, and content, to name just a few, with essential requirements listing a relevant degree and a set number of years in the subdivision. Meanwhile, AI threatens to upend the commercial ecosystem as we know it, if we let it. There’s no shortage of investment in commercial AI: budgets exist, and pilots launch.  What’s missing is a redefinition of the realm of commercial possibility. R&D teams talk about AI in terms of what’s possible, where Commercial teams mostly talk about it in terms of what’s faster: faster call planning, faster reporting, faster content approval. In the pharmaceutical industry, we’re not pondering what the Commercial function could become, only what it could do quicker. I believe that the pharmaceutical industry could learn several vital lessons from the digital health teams they are so frequently adjacent to, or in partnerships with.

    Today

    As it stands, commercial teams still remain largely siloed, despite a decades-long effort to the contrary: the SWAT teams who swoop in for launch, the marketing teams who focus on a “360-degree view of the customer” and pore over agency data on tactic performance, the sales teams who battle daily for two to five minutes with target HCPs, the digital teams optimizing Veeva or Salesforce and wrangling unwieldy data, and the market access teams negotiating, lobbying, and analyzing. Each function is a separate thread in the complex ball of yarn that is a Commercial team in 2026.  Each trying to grapple with the pace of change driven by AI for specific, single threaded needs. For example, a rep getting handed a new “AI-driven next best action” tool but it’s bolted onto the same static quarterly system that existed before, and doesn’t address any fundamental underlying model improvements.

    Opportunities for Pharma

    The real question: if we designed a pharma commercial function from scratch today, would it look anything like this? My hypothesis is no, and that it would look much closer to the world of digital health.

    Digital health and Pharma remain distant cousins in the family tree of life sciences and health. Where digital health can focus on specific interventions and inventions, Pharma, Big Pharma at least, has the unenviable task of spreading its approach across indications, therapeutic areas, and ultimately entire patient and HCP populations. The result is that digital health companies have grown up leaner, with commercial functions built as a specific, consolidated, and usually much smaller function than in pharma. 

    What pharma commercial could borrow from digital health isn’t complicated or novel, but it isn’t obvious in the day to day of a big pharma juggernaut. I once heard that in the beginning days of the internet, the NY Times typed in the full newspaper – just digitizing the physical copy. This is an exact analogy for how most of big pharma is using agentic AI now: focused on re-creation of existing ways of working and increasing efficiencies.  

    The less obvious side to the speed and efficiency coin is the idea of changing the actual operating rhythm of the portfolio: shipping concepts to production in days rather than months or years. A digital health team ships a feature in weeks based on a usage pattern; a pharma commercial team can spend a quarter just getting requirements gathered. Or radical focus: a digital health commercial function is built around a handful of clear outcomes, not a dozen sub-functions each optimizing their own area. Pairing radical focus with pharma’s strength in regulatory & compliance creates the opportunity to expedite patient outcomes in a way before unseen. Finally, digital health companies build with partners from day one because they have to, while pharma often plans in-house first and partner only once internal options are exhausted, organizational fatigue has taken grip, or the “build” is ready to get going.

    Making It a reality

    What would it look like if Pharma embraced a digital health style way of working? Potentially, it would be by replacing the sprawl of sub-divisional dashboards and systems with one shared data layer, a single source of truth on HCP engagement, patient journeys, tactic performance, and pipeline, with access rights layered on top rather than entirely separate systems per function. Instead of a field force, maybe it’s a cognitive command center AI that operates across functions instead of within one, flagging a stalled account to market access, content, and the field team simultaneously instead of routing sequentially through three approval chains. And rethinking the field model itself: rather than dispersing reps to chase five-minute HCP windows on a static cycle, that same investment could fund fewer, richer touchpoints, twice-yearly events, and deeper partnerships with different, AI native platforms with distribution & reach like OpenEvidence that HCPs are already choosing to use on their own terms.

    None of this requires more commercial budget than pharma is already spending. It requires spending it on reimagining commercial as a function instead of speeding up an existing function. Digital health and pharma have plenty to learn from, and with, each other, and AI is the first real forcing function either industry has had to do it. The Pharma commercial teams that treat this as a chance to redesign the model, not just streamline it, will be the ones that change what’s possible for HCPs and patients. The remainder will only benefit from greater day to day efficiency.

    Source: champpixs, Getty Images

    Suzy Jackson

    Suzy Jackson is a Digital Health & Commercial Pharma subject matter expert and former Digital Health Chief Commercial Officer at RVO Health, as well as former Head of the North America Life Sciences Commercial Consulting team at Accenture. Suzy’s independent advisory focuses on Commercial technology, strategy, Go-To-Market efforts, market entry, partnership shaping, and competitive analysis across Pharma and Consumer Health. Outside of her advisory work, Suzy is an avid scuba diver, traveler, and sits on the Boards of two non-profits focused on women’s rights, legal advocacy, and survivorship from trauma and abuse.

    This post appears through the MedCity Influencers program. Anyone can publish their perspective on business and innovation in healthcare on MedCity News through MedCity Influencers. Click here to find out how.

  • Startup Arboretum Sprouts, Bringing a Precision Medicine Approach to Common Diseases

    Startup Arboretum Sprouts, Bringing a Precision Medicine Approach to Common Diseases

    The career of Anthony Philippakis has tracked with the evolution of molecular medicine. The cardiologist started medical school in 2001, one year after the first draft of the human genome was published. As a PhD student, he studied human genetics. At GV, where Philippakis is currently a general partner, he played a key role in the firm’s investment in Foundation Medicine, a precision medicine company that provides cancer tests and analyzes tumor samples, identifying genetic mutations that inform treatment decisions.

    “My professional career has been following the genome wherever it leads,” he said.

    Philippakis’s journey in genetics has reached its next stop. He’s the co-founder and CEO of Arboretum LifeSciences, a startup firmly planted in both diagnostics and drug development. The company is now out of stealth with a handful of partnerships and the ambition to leverage its genomic analyses into alliances with pharmaceutical companies, each side of its business working toward the shared goal of developing new medicines for common diseases. The Cambridge, Massachusetts-based company has raised $30 million in new funding to execute that mission.

    Arboretum is a molecular information company, a concept that Philippakis said goes back to an idea from Foundation Medicine. While a molecular diagnostics company only does diagnostics, a molecular information company does diagnostics and then uses the data generated from those tests to develop therapies, he explained. This concept first took root in cancer, where it’s easier to identify a particular genetic mutation as the driver of the disease — matching a drug to that genetic signature is fairly straightforward. But in a more common conditions like heart disease, there’s usually no single disease driver. To address such diseases, scientists need genetic approaches that define patients based on many variants rather than a single one.

    Arboretum supplies whole genome sequencing tests that support precision medicine programs at health systems. The company then analyzes data from those tests, using the analyses to guide drug development. The startup’s business model would not have been possible years ago, said Clare Bernard, Arboretum’s president. Alongside the rise of AI capabilities, the cost of genome sequencing has come down, making it easier to sequence larger numbers of patients, she explained. There’s also a growing number of indications where genetic testing is clinically indicated and payer reimbursed.

    Arboretum’s analysis yields a risk score indicating genetic predisposition to a particular disease. Bernard said these scores allow the company to identify disease targets as well as patients who could be a match to a clinical trial. These data can also help Arboretum design a clinical trial or match patients to a trial. These data can become the basis of partnerships with pharmaceutical companies, guiding their drug research. In these instances, Arboretum is not acting as a technology vendor. Philippakis said these deals involve risk sharing where the startup gets some of the financial upside if its analysis leads to a successful drug.

    “When we find one of those opportunities, either we approach a pharmaceutical company and say, ‘You’re developing a drug, we have insight on how to run a smarter trial,’ and do a partnership,” Philippakis said. “Alternatively, there might be an early-stage molecule somewhere in the world where we say, oh, let’s go in-license it because we have a differentiated insight on the development.”

    Arboretum aims to form startups around in-licensed assets. These startups would incubate within Arboretum. Philippakis said his company has “a number of active discussions” and he expects to have more to share in 2027.

    The alliances that are public are with health systems. Advocate Health, Geisinger, Providence Healthcare, and Cardiovascular Associates of America are all partners with Arboretum, using the startup’s genetic tests and creating longitudinal molecular registries. The startup also recently started sequencing its first patients in the United Kingdom, and Philippakis said the company aims to grow internationally.

    From the patient perspective, the patient’s doctor would order the Arboretum test, Bernard said. That could be an oncologist or a cardiologist. In some cases, it’s a primary care physician. The test is a simple cheek swab that can be done at a clinical site. Alternatively, a kit can be sent to the patient’s home. After the test, patients may consent to have their data included in a research biobank to be part of Arboretum’s research.

    “If they’re part of the research study, they can get additional results returned,” Bernard said. “That also gives them the opportunity to participate in clinical trials and gives us greater consent to match them to clinical trials in the future.”

    Arboretum started with $5 million in seed financing from GV and F-Prime. The new financing is a Series A round led by GV, F-Prime, .406 Ventures, Hims & Hers, Amgen and other unnamed healthcare investors. Philippakis estimates the capital will last at least three years. In that time, he aims for Arboretum to blossom to half a million people sequenced and several opportunities in drug development, either startups spun out of the company or partnerships with big pharma.

    The concept of Arboretum started taking shape during the Covid-19 pandemic, when Philippakis and Bernard both worked at the Broad Institute. During long outdoor walks, Philippakis said they talked about the confluence of automation, diagnostics, and biotechnology, as well as the shift of these disciplines to more capital-efficient approaches. The startup’s name is a reference to the Arnold Arboretum, which is near Bernard’s home and where the pair took those walks.

    “We like the metaphor an arboretum grows trees, we’re growing biotech companies,” Bernard said.

    Photo by Flickr user Jason Baker via a Creative Commons license

  • From Distributor to Disruptor: Amazon’s Healthcare Flex

    From Distributor to Disruptor: Amazon’s Healthcare Flex

    Could this be Big Tech’s ultimate healthcare flex? For years, Amazon’s ambitions in this $4 trillion market were focused on distribution: acquire a pharmacy here, a primary care network there, and offer convenience.

    But more recently, rather than deploying capital to expand its fulfillment engine, Amazon exercised its cloud and AI muscle to launch tools that enable it to be a “front door” for consumers and patients. From the looks of it, the e-commerce giant has shifted, from competing for a piece of the healthcare journey to collapsing the entire journey into one company.

    In any case, pharma commercialization teams may need to think about Amazon less as a new distributor and more as a disruptor of how patients, prescribers, and payers connect.

    Analyzing the moves

    Big Tech has upended many industries, but the highly regulated and complex healthcare industry has seemed impervious to their influence. Judging by Amazon’s most recent forays, however, this sector may not represent an innovator’s “white whale” for much longer.

    Amazon has closed the “last mile” between writing a prescription and filling it. At One Medical clinics (Amazon’s primary care venture), pharmacy kiosks now let a patient walk out of an appointment with medication in hand, filled by the same company that just diagnosed them. Its oral GLP-1 management program, a recently introduced offering, takes this even further. 

    This year, One Medical also launched an AI-enabled assistant, with access to test results, medical history, and more. The AI agent, dubbed Health AI, can help book medical appointments or provide medical information, such as an explanation of lab results. This clearly strengthens One Medical’s abilities as a provider and can help in enhancing the patient experience all the way through drug dispensing.

    More critically to pharma, Amazon’s Health AI also automatically applies manufacturer coupons and copay assistance programs for eligible patients. On the HCP side, Amazon’s integration with DoseSpot and eNavvi places patient net out-of-pocket at the point of prescribing.

    Health AI subsequently moved beyond the One Medical app, embedding directly into Amazon.com and its mobile app, marking the first time the tool has been available at scale to general consumers, and further enmeshing the patient experience into the overall Amazon environment. These offerings are available to anyone, regardless of being a One Medical member.

    4 potential shake-ups

    The result: Screening, prescribing, fulfillment, and ongoing renewal all happen inside one Amazon-run loop. No outside touchpoint is required.

    That loop doesn’t just change where a patient enters the system and picks up a prescription. It changes the nature of a healthcare “provider,” how care is delivered (including drug fulfillment), and who sees the full picture of a patient’s care. 

    In other words, this shift isn’t just a single disruption; it’s four separate ones. Here’s the “where” and “how” of those potential shake-ups:

    • The HCP relationship: Who manages a condition over time, not just who writes the first script?
    • Channel and patient experience: Who owns the point of fill, and how seamless is the drug-fulfillment experience?
    • Price transparency and patient OOP: How much of a role does real-world price play, for both prescribers and patients, and how do coupon and copay assistance programs need to adapt?
    • Data visibility: Does the full patient journey, from diagnosis to refill, sit outside the purview of traditional pharma data sources, or is it somehow still captured? 

    The four categories aren’t equally mature. And that has real implications for where pharma commercialization teams focus first. 

    Where pharma should focus

    The first two of the above categories are already live with the GLP-1 program. That is, ongoing management of a chronic condition now sits inside Amazon’s own clinical protocol, not a patient’s existing PCP or specialist. And prescribing, fulfillment, and refill take place in a channel pharma doesn’t traditionally call upon.

    GLP-1 is a favorable starting point for this model because it’s heavily consumer-driven and manageable in primary care, so the loop doesn’t need specialist judgment to run. And the GLP-1 cash-pay models give Amazon room to control pricing directly. Moreover, weight-loss management is a long-term, recurring relationship, which is exactly the kind of retention Amazon’s model is built for. 

    Beyond the cash market, Amazon’s ability to automatically apply coupons and copay assistance offers the opportunity for accelerated product uptake, accompanied by significant downside risks to product gross-to-net.  

    If your brand touches a condition that could be managed this way, GLP-1 isn’t a case study to read with curiosity. It’s a preview. Teams should be asking if and how this loop affects prescribing and fulfillment for their therapeutic area; that’s the immediate gap to close. 

    Conditions that depend on specialist diagnosis, complex or in-clinic administration, or those with reimbursement complexity, are a much harder fit for this model (at least for now). The therapeutic areas to watch first are protocol-friendly, primary-care-managed, chronic, and with a cash-pay market underneath.

    Payers and data

    Amazon’s impact on the payer space is at an earlier stage, but the signals are worth tracking closely. Amazon has already become the exclusive home delivery pharmacy for select PBM partners, including its own employee health plan. While that’s a narrow footprint today, whether it stays narrow or expands to further erode the traditional PBM mail-order business is the open question. Every new deal is a data point worth logging.

    Data visibility is the slowest mover of the four categories, and possibly the hardest to predict. Cash-pay markets already have data holes. Adherence and outcomes in these markets have always been harder to capture, since the transaction doesn’t run through the claims and pharmacy systems pharma is used to watching. 

    The bigger exposure is what happens if this extends into broader chronic disease management. Even losing part of a patient’s journey, say the fulfillment and refill half, is enough to break the continuity pharma relies on for adherence programs and tracking the various outcome measures of marketed treatments. 

    Other industries offer a useful, if uncomfortable, reference point. Retail went through its own version of this: Vertically integrated fulfillment that started as a convenience play eventually became infrastructure that competitors couldn’t easily match on price or speed. 

    That pattern is worth keeping in mind, not as a prediction of exactly how this plays out in life sciences, but as a reminder that the “early and narrow” phase doesn’t stay that way indefinitely.

    None of this is a call to panic. It’s a case for watching the right signals. The clearest one: which therapeutic area Amazon’s loop expands into next.

    Photo: undefined undefined, Getty Images

    Adrienne Lovink

    As CEO, Adrienne Lovink is leading Beghou’s next phase of growth and building on its reputation as a trusted commercialization partner in life sciences. She is focused on expanding into new areas of client demand; advancing the firm’s data, AI, analytics, and technology innovation; and investing in its people and culture. Adrienne has over 25 years of experience partnering with pharmaceutical and medical device companies to advance commercialization, market access, forecasting, digital health, real-world data, and advanced analytics, including as a Partner at Trinity Life Sciences and Global Head of Real-World Data and Advanced Analytics at DRG / Clarivate.

    This post appears through the MedCity Influencers program. Anyone can publish their perspective on business and innovation in healthcare on MedCity News through MedCity Influencers. Click here to find out how.

  • Forbion Bucks VC Fundraising Trends, Reeling In €2.3B for Two New Funds

    Forbion Bucks VC Fundraising Trends, Reeling In €2.3B for Two New Funds

    Forbion, already one of Europe’s largest life sciences venture capital firms, now has more investment firepower with €2.3 billion (about $2.6 billion) raised for two new funds.

    The fundraise is the largest in Forbion’s 20-year history, the Naarden, Netherlands-based firm said Tuesday. It comes in a market generally characterized by a shortage of capital, particularly in Europe.

    This year through the third quarter, VC firms globally have raised $142.1 billion total, according to a new report from Pitchbook. VC fundraising is on pace to top $200 billion for the full year, which would mark a strong rebound from 2025. But the report also shows more money going into fewer funds.

    Furthermore, there’s geographic imbalance in the fundraising. About two thirds of this year’s financial commitments to date are for firms in North America. Pitchbook said that despite this year’s global fundraising already surpassing 2025 figures, Europe is on pace for its lowest fundraising total since 2019, excluding last year.

    The imbalance is also evident in tallies of VC investments. The number of deals and the deal values are up year over year, but that reflects strong activity in North America, the report said. Every other region in the world is on pace for a decline in deal count year over year, which Pitchbook attributes to the lack of liquidity across global markets.

    Forbion said it has already completed the first investments from its two new funds, Forbion Growth Opportunities Fund IV and Forbion Ventures Fund VIII. The firm added that it expects its new capital will support investments in about 30 portfolio companies. In the announcement, Forbion co-founder and Managing Partner Sander Slootweg said the capital enables the firm to invest in “the most exciting biotech companies.”

    “We will continue to focus on building and scaling those companies that develop the most paradigm-shifting treatments, addressing true unmet needs and delivering societal impact,” Slootweg said.

    Forbion’s investments this year to date include participation in the $130 million Series A financing of North Carolina-based migraine drug developer Slate Medicines (now on track to go public in a reverse merger) and the $125 million Series A financing of immunology biotech Coultreon Biopharma, based in Belgium.

    Forbion said its new capital comes from existing investors as well as new ones. Disclosed investors include MN, PGGM, KfW Capital, Kauffman Foundation, and Eli Lilly and Company. With the capital raised for the two new funds, Forbion said it has €7.5 billion (about $8.5 billion) in assets under management spread across 13 funds, making it Europe’s largest dedicated life sciences VC firm.

    Image: mustafaU, via Getty Images

  • Shionogi Adds to Its Rare Disease Strategy With $2B IntraBio Acquisition

    Shionogi Adds to Its Rare Disease Strategy With $2B IntraBio Acquisition

    The growth strategy of Shionogi & Co. is picking up a new piece through the $2 billion acquisition of IntraBio, a biotech company that brings a drug approved in two rare indications along with experience and expertise that complement the Japanese firm’s pipeline of drugs for rare neurological diseases.

    The main IntraBio asset in the deal announced Monday is levacetylleucine, brand name Aqneursa. The FDA initially approved this drug in 2024 as a treatment for the neurological manifestations of Niemann-Pick disease type C, a rare inherited enzyme deficiency. It’s one of just two FDA-approved therapies for this disorder. Last month, the FDA expanded Aqneursa’s label to include ataxia-telangiectasia (A-T), making it the first approved therapy for this rare neurodegenerative disorder.

    Osaka, Japan-based Shionogi established a commercial presence in rare disease through a business deal earlier this year, the $2.5 billion acquisition of global rights to Radicava, an amyotrophic lateral sclerosis drug developed by Tanabe Pharma. Shionogi’s other rare disease drug candidates are also from business deals.

    S-606001, currently in mid-stage clinical development for the rare enzyme deficiency Pompe disease, was licensed from Maze Therapeutics in 2024. Zatolmilast came from Shionogi’s 2020 Tetra Therapeutics acquisition. In May, Shionogi disclosed that this oral small molecule failed two Phase 3 tests in Fragile X syndrome, a genetic disorder that leads to intellectual disability and developmental delays. A separate mid-stage study is still evaluating the molecule in Jordan syndrome, a rare neurodevelopmental disorder.

    Privately held IntraBio was founded in 2015. Its scientific founders hailed from the University of Oxford and the University of Munich, where they discovered and developed small molecules that modulate lysosomal function and intracellular calcium signaling. Initially based in Oxford, U.K., IntraBio relocated to Austin, Texas, in 2024.

    Aqneursa is a modified form of L-leucine, an essential amino acid sourced from food that the body needs for building and repairing muscle and for enabling cells to produce energy. IntraBio’s version of the amino acid is modified in a way intended to help it cross membranes, including the blood-brain barrier. But Aqneursa’s label states that the drug’s mechanism of action for treating Niemann Pick and A-T is unknown. The company has said this drug has potential applications in a range of rare and common neurological disorders. According to Shionogi, Aqneursa generated $67.8 million in revenue in fiscal 2025.

    Shionogi’s largest source of revenue is royalties from HIV drugs marketed by ViiV Healthcare, a company majority owned by GSK. Earlier this year, Shionogi increased its minority ownership stake in ViiV from 10% to 21.7%. Those HIV medicines are part of a broader Shionogi business strategy with a 2030 target date for achieving growth goals. Another goal in this strategy is entering the rare disease business overseas.

    Beyond Aqneursa’s FDA approvals, the drug also received European Commission approval early this year for Niemann Pick disease type C. An additional European regulatory review is ongoing in A-T. In a prepared statement, Shionogi CEO Isao Teshirogi said the IntraBio acquisition shows his company’s commitment to building a global business in rare disease.

    “Bringing Aqneursa to Shionogi after our acquisition of Radicava will deepen our commitment to rare disease communities, expand our capabilities and strengthen our portfolio as we advance future innovation for patients with significant unmet needs,” he said.

    The IntraBio acquisition still needs to clear regulatory reviews. Shionogi said it expects the transaction will close by the end of this year.

    Photo: Soichiro Koriyama/Bloomberg, via Getty Images