3 Things In Biotech You Should Learn Today: November 5, 2017

Summary

• Ionis takes its swing in Europe.
• Aduro attacks myeloma.
• Sanofi marks another grave in the fight against IPF.

Welcome to another edition of "3 Things In Biotech You Should Learn Today," a daily digest dedicated to helping you keep pace with the fast-moving world of pharmaceutical and biotechnology research.

Ionis takes its swing in Europe

Company: Ionis Pharmaceuticals (IONS)

Therapy: Inotersen, an antisense oligonucleotide targeting the transthyretin (TTR) gene

Disease: TTR-related familial amyloidosis

News: IONS announced that it has submitted a marketing authorization application to the EMA, seeking approval for inotersen. This application is supported by results from the NEURO-TTR study, which demonstrated significant improvement for inotersen compared with placebo in a number of meaningful endpoints relating to function in patients with neuropathy related to familial amyloidosis. This application will be evaluated under the Accelerated Assessment Program.

Looking forward: That's one good way to build quickly on good news. Just yesterday we covered the strong data presented by IONS, and now this? Moreover, the company intimated that it would be submitting to the FDA very soon, as early as next week. So this is one potential way to get an advantage over patisiran, which appears to be more favorably tolerated at this time: get approved sooner? We'll see. It's possible that safety issues could sink this application, but we're talking about an unambiguously effective therapy in an area of critical unmet need for patients. So I like its chances of approval.

Aduro attacks myeloma

Company: Aduro Biotech (ADRO)

Therapy: BION-1301, an anti-APRIL monoclonal antibody

Disease: Multiple myeloma

News: ADRO announced in its press release for its third-quarter filing that the FDA has granted approval to move forward with clinical study of BION-1301, an anti-APRIL monoclonal antibody that has demonstrated encouraging preclinical efficacy in multiple myeloma. APRIL is a ligand for BCMA, which has grown in importance as a hot new therapeutic target in myeloma studies.

Looking forward: ADRO takes an interesting risk-hedging approach to cancer, as it has a diverse pipeline that extends well outside of its flagship listeria-based treatment approach. This is good and bad, as the listeria-based immunotherapy is rather risky as a new therapeutic modality, with ADRO being unable to demonstrate clear efficacy so far (though hopefully this will change). Other programs help mitigate this risk, but it also creates a drag on funds, increasing the R&D burn rate and requiring outside sources of cash. The recent emergence of BCMA as a target for multiple myeloma is highly exciting, as well, and one that I am watching with significant interest, as we're going to see a fair number of therapies targeting this protein somehow come out in the next few years.

Sanofi marks another grave in the fight against IPF

Company: Sanofi (SNY)

Therapy: SAR156597, a bispecific IL-4/IL-13 antibody

Disease: Idiopathic pulmonary fibrosis (IPF)

News: SNY announced that the development of SAR156597 in IPF has been suspended in its Q3 earnings release. It is unknown at this time why development has been terminated since no presentations or announcements of clinical trial results have been made.

Looking forward: Very unfortunate news. I know I have quite a few readers here interested in developments in the IPF space, which is clearly quite difficult to treat. Intuitively, one would hope that the link between pro-inflammatory cytokines like IL-13 and IPF would yield efficacy in clinical study. Unfortunately, the reality is often more complicated than that. Hopefully, we'll get a better read on what the problem with this therapy was in the future. For now, patients have to rely on just two approved therapy options for IPF.

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Analyst’s Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.