Biotech General Discussion

• NASH is a liver disease that is one of the most challenging diseases for finding effective therapeutics. There still are no approved drugs to treat NASH after years and billions of dollars of research.

• NASH was a high flying indication for biopharma company valuations and investors around 5 years ago when many companies entered the space.

• See Table 1 for smid-cap biopharmas that currently have NASH assets. Included is info on trial stage, mechanism of action (MoA), targets, and hedge funds holding each company.

Table 1. Smid-cap biopharmas with NASH trials.

• Since 2018, because of difficulties in running trials for NASH and difficulties getting positive results that convince the FDA that a drug should be approved, NASH company valuations have plummeted.

• However, there has been some recent positive results, and a number of major catalysts readouts coming in the next 12 months, including in the coming days, that have rekindled hope and interest in NASH for investors.

• We highlight some of those key NASH companies and readouts in this post.

MDGL:

• In May 2018 MDGL reported impressive Phase 2 data from their MGL-3196 trial

• Their stock skyrocketed afterwards (+145%) but then decreased rapidly; now down 78% since this Phase 2 data

• Likely because of overall negative sentiment toward NASH and the length of time and difficulties with running clinical trials for this disease

FIG. 1 MDGL 5 yr stock chart

VKTX:

• VKTX has seen a similar trend in their stock

  • Both VKTX and MDGL assets are THRb agonists

  • VKTX saw a spike in 2018 (specifically in May 2018 with MDGL and in Sept 2018 when positive Ph2 NAFLD data was presented) but has fallen sharply from those peak valuations

FIG. 2 VKTX 5 yr stock chart

• After these impressive Phase 2 data reports for NASH/NAFLD, the market realized that Phase 3 NASH data is a long ways away

  • MGDL, VKTX, and other NASH stocks have languished since 2019. See FIG. 1 and FIG. 2.

  • MDGL down 78% and VKTX down 63% since May 31, 2018

• See Table 2 for moves in some NASH companies since YE 2019.

Table 2. Moves of major smid-cap NASH companies since end of 2019

ICPT:

• In the race for NASH approval, leading the pack is ICPT, who is about to resubmit their reanalyzed and twice positive Ph3 data, this time with much more safety data and new consensus readout data.

FIG. 3 ICPT 5 yr stock chart

• See Table 3 to see how ICPT compares to other NASH companies with upcoming readouts.

Table 3. Smid-cap biopharmas with upcoming NASH readout

AKRO

• Recently there is a NASH resurgence partly brought on by AKRO data from September that has continued to spark some resurgence in NASH

  • Sept AKRO data on 9/13/22 showed both the 28mg and 50mg doses of “Efruxifermin” were statistically significant in primary and secondary endpoints in Phase 2b HARMONY study, especially in a composite endpoint that showed that in many patients the drug lowered fat and reduced fibrosis, the two hallmarks of NASH

  • AKRO stock moved 137% upon release of this data!

FIG. 4 AKRO lifetime chart showing recent resurgence

• Exciting NASH readouts are imminent and will be coming for the upcoming quarters (see Table 3)

• MDGL Ph3 data is due this month and should arrive any day.

  • MDGL is a BPIQ Big Mover for December and we are anxiously awaiting the data

• MDGL had great Ph2 data, see Table 4 for a comparison of Ph2 and Ph3 design

Table 4. Phase 2 and Phase 3 trial design and data for MDGL asset

• MDGL made some changes to the design for Ph3 to try to increase their chances of success in this larger patient population

• Ph3 trial used a screening algorithm to help confirm that "NASH" patients have NASH

  • NASH was proven in patients by biopsy within 24 weeks before patient randomization and needed a fibrosis stage of 1A, 1B, 2, or 3 and NAS of at least 4

  • 80% patients confirmed NASH on liver biopsy.

• Exciting later stage trial readouts are coming in the next year

  • ICPT resubmitting for approval this month based on reanalysis of phase 3 data using a consensus read, which again was positive.

  • ETNB reading out Ph2b ENLIVEN data in Q1 '23

    • MOA the same as AKRO (FGF21 analogs)

  • VKTX Ph2b VOYAGE data expected in Q2 ‘23

  • TERN readout expected at the end of Q4

    • More selective THRB agonist than MDGL or VKTX

See our subscriber's article for additional info on these assets and trials including MoA, targets, and which hedge funds held the companies at the end of Q3.

History: Published 12/14/22

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