To overcome regulatory hurdles for "N-of-1" medicines, researchers are using an "umbrella clinical trial" strategy. This approach keeps core components like the delivery system constant while only varying the patient-specific guide RNA, potentially allowing the FDA to approve the platform itself, not just a single drug.

A key trend in 2025's drug approvals is that "best-in-class" therapies are distinguished not just by efficacy, but by innovations in formulation and delivery that improve the patient experience. Examples include subcutaneous versions of IV drugs and new delivery methods that expand patient access.

Instead of targeting new biological pathways, Apogee enhances proven antibody therapies by extending their half-life. This shifts the competitive battleground from pure scientific discovery to patient adherence and lifestyle, aiming for quarterly or semi-annual dosing versus the current bi-weekly standard for market leaders.

A $2,000 preventative injection like a PCSK9 inhibitor sounds expensive. However, its cost is likely justified when calculated against the massive societal and individual expense of future medical bills, plus the economic value of additional healthy, productive years.

Protagonist believes its oral IL-23 blocker will not just compete with existing injectables but will capture a new market. They target the over 50% of eligible patients who currently take no therapy due to a dislike of injections or the safety profiles of other oral options, thereby expanding the total addressable market.

Beyond the initial wave of GLP-1s from Novo and Lilly, the next major competitive front in the obesity market will be monthly injectables. Amgen and Pfizer (via its Metsara acquisition) are poised to lead this race, shifting the focus to dosing convenience and long-term adherence.

When asked about complex antibodies like ADCs and bispecifics, GSK's CSO emphasizes that extending a drug's duration is a primary innovation. He highlights a severe asthma treatment dosed just twice a year as a prime example of creating significant patient value before adding further engineering complexity.

Despite being a pill, oral Wegovy requires an empty stomach, only 4oz of water, and a 30-minute post-dose fast. This difficult regimen is a major impediment to its uptake, particularly in the U.S. where patients prioritize the maximum efficacy of injectables over the supposed convenience of a cumbersome pill.

Recent FDA approvals for Milestone's Cardamist nasal spray and J&J's subcutaneous Ribrevent Fastpro highlight a key industry trend: improving patient convenience. These products shift treatment from clinical settings to on-demand, at-home use or reduce administration time, creating value beyond just clinical efficacy.