Are Cell & Gene Therapy Programs a better bet for Big Pharma?

This came across my desk, thought it was some quality information...

BigPharma is heading to a sales cliff in the next 4 years. Leading into 2028, aseries of blockbuster drugs are coming off patent protection, otherwise knownas Loss of Exclusivity (LOE) – the scale of this potential loss insales is over $200B.

Theexpectation is that Big Pharma will be out there to protect its revenuestreams, which will be done through acquisition, a strategy not unusual for anylarge company.

Whatare the targets?

BigPharma has not been good at early-stage development and uses a strategy thatrelies on the acquisition of key technologies and platforms that are close tocommercialisation. The choices are:

1. Cell and Gene Therapy Drugs: The commercialisation stage is Phase 2, and after ~120 patients
2. Small Molecule Drugs: Commercialisation at Phase 3 after trialling on hundreds (or more) patients over multiple years.

BigPharma likes to control the last stage of development. They are happy forlate-stage risk, so the acquisition sweet spots are at Phase 1 for Cell andGene Therapy Drugs and Phase 2 for Small Molecule Drugs.

Opensthe questions for the contrary position.

1. if you have a Cell Therapy at the end of Phase 1 and it is not licensed – is it wanted by the market?
2. if you have a Small Molecule therapy at the end of Phase 2 and it is not licensed – is it wanted by the market?

Big Pharma can see value in Celland Gene Therapy

Lookingat pipelines, management commentary and asset acquisition of key pharma players,here are some thoughts on how this exclusivity protection may play out, and whyCell and Gene Therapy has a strong chance of being the main target. You willsee in the first table below a few Big Pharma may need to play catch up, andthat is generally expensive!

ForBig Pharma, taking earlier targets to solve the LOE problem is unsuitable andwill not solve the problem (long lead times, blowing past 2028), so for Celland Gene Therapy to be a target, they must be trialled in humans.

While most of the Cell and GeneTherapy drug development has been in CAR-T’s, this data is expected to also besimilar for CAR-NK and CAR-iNKT drugs under development.

1. CAR-T/TCR therapies for blood cancers are 3X as likely to be approved when entering Phase I as the average oncology drug
2. CAR-T/TCR are over 2X as likely as the average haematological oncology drug.
3. Orphan gene therapies are 2 – 3.5X as likely to be approved when entering Phase I as the average drug in clinical trials, outperforming in every clinical development phase.
4. Orphan gene therapies are 2X as likely to be approved when entering Phase I as the average drug in similar therapeutic areas*, outperforming in every phase.

Below is a table of how much BigPharma have at risk, and what they have done about it, except for Novo Nordisk,J&J and Abbvie who are yet to play.

Transactions in the Cell andGene Therapy sector (Novo Nordisk, J&J and Abbvie still missing on sub $1bndeals)