As the oncology market enters a new era of growth, targeted therapies across several modalities are transforming the treatment paradigm. Amid this shift, radiopharmaceuticals are emerging as a promising therapeutic class – capturing the attention of big pharma companies and investors alike.
While their roots are firmly embedded in the diagnostics sector, radioligand therapies (RLTs) are now making waves across the oncology treatment landscape due to their precision approach. Novartis was the pioneer of this drug class, as the Swiss pharma became the first company to gain US approval for a RLT with Lutathera (lutetium Lu 177 dotatate) back in 2018.
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Now, both Lutathera and Novartis’ second approved RLT, Pluvicto (lutetium Lu 177 vipivotide tetraxetan) are blockbuster sellers, with GlobalData forecasting the drugs will bring in $1.1bn and $5.1bn for the company in 2031, respectively.
Other pharma titans such as Eli Lilly, AstraZeneca and Bristol Myers Squibb (BMS) are trying to replicate this success across their own RLT programmes. Between 2023 and 2024, the three companies bought out Point Biopharma, Fusion Pharmaceuticals and RayzeBio, respectively, through multi-billion dollar deals to gain a footing in this niche. Lilly has, however, terminated development of AC-225-PSMA-62 in prostate cancer due to the drug failing to meet efficacy expectations.
The interest in RLTs appears to be mirrored by investors, as venture financing for radiopharmaceuticals reached a record high of $408m in 2023 – a value that continued to grow into 2024 through a 330% venture capital surge in Q1 2024 versus the first quarter of 2023.
Despite the burgeoning success of this modality, companies harnessing their potential in oncology will encounter development, regulatory, reimbursement and commercialisation hurdles that must be overcome, experts tell Pharmaceutical Technology.
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Theranostics turn heads
As diagnostic and therapeutic radiopharmaceutical products garner their own individual successes, attention is turning to how these techniques can be combined to facilitate precise and image-driven treatment in oncology. From this concept, theranostic radiopharmaceuticals were born – which harness a radioligand alongside a radioactive isotope visible through an imaging scan.
By blending diagnostics with treatment, Aryeh Sand, healthcare partner and nuclear medicine specialist at investment firm Solomon Partners, believes that theranostic radiopharmaceuticals have broad potential across numerous solid tumour types. “The neuro-endocrine and prostate cancer markets are just the tip of the iceberg,” he says.
Volker Wagner, CMO of French radiopharmaceutical-focused biotech, Orano Med, echoes Sand’s sentiments, noting that theranostics can enhance confidence when treating patients. “With theranostics, a physician can send a patient for imaging and see the extent of their disease. They can then precisely expose tumours to radiation through the radioactive payload, which will go to the exact locations picked up on the scan,” Wagner comments.
This effect, he says, is mirrored outside of the radiopharmaceutical niche, as non-radioactive companion diagnostics are also gaining strong traction within the oncology segment.
According to Marc Hedrick, CEO of RLT and diagnostics-focused biotech, Plus Therapeutics, theranostics present an opportunity as oncology moves away from the one-size-fits all approach. “They offer value by facilitating better patient selection, the ability to monitor patient response dynamically to adapt therapy, and the potential for superior outcomes with fewer off-target effects,” he says.
RLTs are also differentiated from other toxic payload-based therapies like antibody-drug conjugates (ADCs), adds Dirk Pleimes, CEO of German radiopharma-focused biotech, Pentixapharm. “The unique aspect of radiopharmaceuticals is that they are molecular markers, which allows for strong treatment specificity,” he comments.
Addressing radiopharma regulations
When developing any drug, companies must navigate a plethora of regulatory hurdles to obtain approval. However, the use of a radioisotope within a therapy adds an extra layer of complexity, as both drug and nuclear regulators must share the reins during the evaluation process.
When approaching RLT or theranostic regulations, Wagner notes that dosimetry is one of the most important considerations in the early clinical stage and beyond. “Dosimetry gives a sense of whether an administered dose is the right one to treat the cancer. It also helps determine a radiopharmaceutical’s uptake in healthy organs to discern potential side effects,” he says.
In dosimetry, Wagner believes it is highly beneficial to use a radiotracer with similar or identical chemistry to the therapeutic asset. “If the tracer isotope does not behave like the therapeutic isotope, then sometimes the conclusions drawn from imaging can come with a few caveats,” Wagner adds. This is backed up by a 2024 paper published in Theranostics, which explains that similar chemistry between diagnostic and therapeutic compounds “reduce the likelihood of a significant difference in diagnostic and therapeutic compound biodistributions”.
Hedrick adds that companies should aim to engage with regulators in an early and proactive fashion – considering trial design, endpoints and manufacturing standards. He also stresses the importance of educating payers and providers on how both RLTs and theranostics can improve the efficiency of overall care.
As RLTs continue to gain traction, Pleimes says regulators are becoming “increasingly comfortable” with the drug class, as more cases are brought to them. In his eyes, this is especially true in Europe, China and the US, where radiopharmaceuticals are gaining particular prevalence.
Moving forward, Pleimes believes that extra considerations related to RLT development will be unlikely to delay the approval process, as radiation-specific regulators are better in sync with drug-focused agencies.
Reimbursement hurdles lower
Upon approaching the commercialisation stage, companies must also consider reimbursement, which can be challenging to obtain for diagnostics, notes Pleimes. “Discussions with payers around the actual value that diagnostics can provide are often more difficult than conversations around therapeutics,” he iterates.
This challenge will likely also extrapolate to theranostics, adds Wagner: “Like many other innovative drugs in oncology, reimbursement will be a challenge, considering that their associated development costs are particularly high”.
However, Wagner caveats that the imaging and treatment-based theranostic approach can help discern who would benefit from treatment – potentially sweetening coverage discussions with payers.
Despite this, Pleimes believes that conversations around radiopharmaceutical reimbursement with likely remain a challenge in Europe due to the fragmented landscape – though it may be easy within individual countries within the continent. In the US, however, recent payer policy developments may benefit players in the niche.
CDMOs to drive future radiopharma growth
Alongside regulatory and reimbursement challenges, companies in the radiopharma space must contend with manufacturing and logistical hurdles. This includes factors like raw material availability, shipping complexity and the shortage of professionals with specialist manufacturing and administration expertise.
Sand notes that contract development and manufacturing organisations (CDMOs) could play a key part in navigating these challenges. “CDMOs will likely play an ever-larger role in the clinical development and commercialisation of radiopharmaceuticals, as they will facilitate the expansion of companies without traditional field capabilities,” he says.
In Hedrick’s eyes, CDMO-owned infrastructure will also drive growth in the radiopharmaceutical field, as it can better support “rapid, reliable supply” while allowing the flexibility to scale programmes from early development through commercialisation.
This would contrast with the vertical integration approach pioneers of the modality were required to take due to the lack of service providers with radiopharmaceutical-focused expertise or infrastructure. “We are now seeing that CDMOs are starting to service the radiopharmaceutical field, because they see promise in it,” Pleimes comments.
As CDMOs continue to enter the radiopharmaceutical sector, Pleimes believes it logical that smaller companies avoid vertical integration; instead opting to use established and effective technologies and radionuclides from service providers.
