5:48 PM
Oct 05, 2018
 |  BioCentury  |  Finance

Seeking validation

4Q18 offers glimpse of key data and launch trajectories in large markets

Editor's Note: This article was updated on Oct 08, 2018 at 11:20 AM PDT

The final stretch of the year offers clinical catalysts and early launch data that could validate investments in hot immuno-oncology targets and large untapped markets.

Late-stage readouts from Vertex Pharmaceuticals Inc. in cystic fibrosis and Biogen Inc. in Alzheimer’s disease will take the spotlight in 4Q18.

Buysiders will also be looking to a slew of annual conferences, including the American Society of Hematology (ASH) meeting in December, for additional clinical data on therapies targeting IL-2 and tumor necrosis factor receptor superfamily member 17 (BCMA; TNFRSF17; CD269).

Others have their eyes on launches for large markets such as migraine, or that feature novel modalities, such as RNAi.

ClearBridge Investments’ Marshall Gordon said the quarter is “more tilted towards continuing execution and launches than data.”

Big caps, big risks

Late-stage data from Vertex and Biogen will take the clinical spotlight in 4Q.

Vertex is evaluating two different triple combinations -- VX-445 or VX-659 plus tezacaftor and Kalydeco ivacaftor -- across four Phase III trials. The regimens have the potential to address about 90% of the cystic fibrosis market.

The company expects data from the two Phase III studies of VX-659 in combination with tezacaftor and Kalydeco late this year.

In a Phase II study, once-daily 400 mg VX-659 plus tezacaftor and Kalydeco improved mean percent predicted FEV1 from baseline to day 29 by 13.3 percentage points vs. 0.3 percentage points for placebo.

“The triple combo probably has to hit,” according to Kai Brüning of apo Asset Management, who said Phase III success is baked into Vertex’s current share price.

Ailsa Craig of International Biotechnology Trust and Ivo Staijen of HBM Partners agreed that Vertex’s readout has more downside than upside potential. “The bar for them is high now. They have to hit that same level of efficacy for everybody to be content,” said Staijen.

“They have to hit that same level of efficacy for everybody to be content.”

Ivo Staijen, HBM

Both Phase III trials of VX-445 plus tezacaftor and Kalydeco are expected to read out in 1Q19.

VX-445 and VX-659 are next-generation correctors of cystic fibrosis transmembrane conductance regulator (CFTR), tezacaftor is a CFTR corrector and Kalydeco is a CFTR potentiator.

Staijen is also watching Galapagos N.V. in the CF space. The biotech expects data this year from the Phase I FALCON trial of its triple combination comprising late binding CFTR corrector GLPG2737, early binding CFTR corrector GLPG2222 and CFTR potentiator GLPG2451.

“It can spoil the party a bit if they show interesting initial triple data of their own,” said Staijen.

In AD, Biogen plans to present additional analyses from its Phase II BAN2401 trial this month.

“That’s a big binary event. So everyone is stressing going into that,” said Craig.

In July, Biogen reported data from the trial showing that the anti-β amyloid therapy slowed cognitive decline by as much as 30%. However, inconsistencies in the dose response and an imbalance of high-risk apolipoprotein E ε 4 (APOE4)-positive patients across the treatment and placebo arms appeared to spook investors.

“There was a big debate regarding the imbalance in the baseline characteristic of the highest dose, and that will be certainly clarified during the presentations in late October,” said IBT’s Marek Poszepczynski.

Staijen said the “amount of enthusiasm” that Biogen and partner Eisai Co. Ltd. have conveyed so far “suggests they expect a robust reception of the full data.”

Conference season

Investors are also awaiting clinical updates from conferences including the Society for Immunotherapy of Cancer (SITC) and the American Association for the Study of...

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