Your Daily Pharma Scoop: Alnylam Update, Malinckrodt's Adcom Failure, Anika's Drug Recall


Analysis focus: ALNY

Alnylam (NASDAQ:ALNY), a leading RNAi therapeutics company, reported earnings last week. It beat on EPS, which at -$1.22 beat by $0.25. However, its revenue, despite rising 15% y/y, stood at $21.9mn, $8.25mn short of consensus. So, the company must have managed to cut costs elsewhere to have been able to do better on the EPS front. The stock showed slightly positive improvement on the news. However, there are a number of upcoming catalysts that will probably help the stock even further. These are – one, FDA approval of patisiran in August; topline data from a Phase 3 study of givosiran in September; and a Phase 3 study of lumasiran to launch mid-year.

Lumasiran is an RNAi therapeutic targeting primary hyperoxaluria type 1 (PH1), an inherited disorder characterized by the lack of a particular liver enzyme that causes oxalate to accumulate in the body leading to kidney stones. Alnylam just reached an agreement with the FDA as to the trial design.

A successful trial will lead to an NDA in early 2020. The drug reduces oxalate by reducing an enzyme called glycolate oxidase. The drug has Breakthrough Therapy status in the U.S. and PRIME status in Europe. Topline results are expected in the second half of 2019.

Alnylam has a number of ongoing programs and has about $1.6bn in cash on hand. It has advanced two more programs into phase 3 – Inclisiran in ORION-9, -10, and -11 Studies, and Fitusiran in the ATLAS Program. The company has enrolled 30 subjects in the givosiran phase 3 trial, and is back on track to produce interim data by September.

Alnylam is trying to make therapeutic use of the Nobel-prize winning science of RNA interference. Craig Mello, one of the founders of Alnylam, jointly received the 2006 Nobel for their discovery of the technology. RNAi is a selective regulation of gene expression by using small interfering RNA (siRNA) molecules that works “upstream” of modern medicine in that it can “nip the disease in the bud,” so to say. This has the potential to block the production of disease-causing proteins, or “knockdown”. This approach can be taken to, in theory, the most undruggable proteins, so there’s potential for a real medical breakthrough in what ALNY is pursuing. Patisiran will be the proof of concept of their technology, whose PDUFA, as we said, is in August, on the 11th. This is an event of major importance.

Stocks in News: Analysis of MNK, ANIK

Ad Com thumbs down on Mallinckrodt’s stannsoporfin

Discussion: The FDA Ad Com did not approve stannsoporfin for elevated blood bilirubin in neonates in its present form on a 21-3 vote. The committee said there is not sufficient evidence to support effectiveness. This drastically reduces MNK’s late stage pipeline.

Anika down 28% on Q1 miss and product recall

Discussion: Anika Therapeutics (NASDAQ:ANIK) performed poorly in Q1, revenue being down 9%, as a result of a shortfall of $1.1mn related to a voluntary recall of HYALOFAST, HYALOGRAFT-C and HYALOMATRIX. This issue will constrain the company for the rest of the year, and will have an impact on its bottomline.

In other news

Celyad’s (NASDAQ:CYAD) CYAD-01 has dosing underway in two early stage studies, and both are showing decent safety profile. One study is for patients with colorectal cancer (NYSEMKT:CRC) with possibly resectable liver metastases. The other is for CRC patients with unresectable liver metastases. Completion are 2021 and 2020 respectively.

Ritter Pharma (NASDAQ:RTTR) will begin two pivotal phase 3 trials assessing lead candidate RP-G28 in patients with lactose intolerance (LI) using Medpace as its CRO.

Regeneron (NASDAQ:REGN) posted Q1 results, with total revenue of $1.5bn, with EYLEA being the key driver. Sanofi collaboration revenue was $189mn, while Bayer was $248mn.

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.

Editor’s Note: This article covers one or more stocks trading at less than $1 per share and/or with less than a $100 million market cap. Please be aware of the risks associated with these stocks.



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