3 Things In Biotech You Should Learn Today: August 10, 2017

by: Zach Hartman

A Roche Raf inhibitor might help in inflammatory disorders.

Innovation Pharmaceuticals nearing an endgame for an important trial.

Cellectar scores a surprising improvement in early myeloma study.

Welcome to new edition of "3 Things In Biotech You Should Learn Today," a daily digest dedicated to helping you keep up with the recent news in the biotech and pharmaceutical industries.

So let's get right to it!

A Roche Raf inhibitor might help in inflammatory disorders

Roche (OTCQX:RHHBF) has been an important player in the use of targeted therapy for metastatic melanoma. As long as the tumor harbors a mutation in a gene called BRAF, its drug vemurafenib can potentially provide some control.

But BRAF mutations can also be associated with nonmalignant conditions, such as the inflammatory disorder called Erdheim-Chester disease (ECD). This is where the body overproduces a certain kind of white blood cell called a histiocyte, which leads to tissue damage, most notably in the bone.

And nearly half the cases of ECD result from the V600 mutation in BRAF, perhaps making this a suitable target for treatment. This disease was identified in the phase 2 basket study VE-BASKET, and on the basis of results from this trial, RHHBF has submitted a drug application for ECD with BRAF mutation.

Recently, the company announced that the FDA has granted priority review status and breakthrough designation for vemurafenib in this treatment area, giving a clue as to where the agency stands on this agent.

Looking forward: Given that there are few effective treatment options for this rare disorder, it comes as little surprise that the FDA is flooring the pedal on the review of vemurafenib here. And any expansion of the approved label for the agent is welcome in light of growing competition from other Raf inhibitors in the melanoma space.

Innovation Pharmaceuticals nearing an endgame for an important trial

Innovation Pharmaceuticals (OTCQB:IPIX) is developing a novel antimicrobial agent called brilacidin for the prevention of severe oral mucositis, which is where the mucosal linings of the mouth break down due to some injury, leading to increased risk of infection and debilitating pain.

Mucositis is a common complication from local therapy for certain cancers, like head and neck squamous cell carcinoma, where radiation therapy is an important treatment option.

The news with IPIX is that it has completed enrollment of a phase 2 study assessing brilacidin as a preventative therapy for mucositis, and the company anticipates a top-line data readout in the fourth quarter of 2017.

Looking forward: The current standard of care for oral mucositis generally is pointed at alleviating symptoms, with nothing available to address the underlying infections that lead to the complication. Brilacidin looks like it could be an effective option in this line, so it is important for those who are following supportive care to keep up on news like this.

Cellectar scores a surprising improvement in early myeloma study

Here's a company you may not have heard of: Cellectar Biosciences (NASDAQ:CLRB). Currently it is involved in the study of cytotoxic therapies conjugated to phospholipids, which make up an important part of the cell membrane. CLR-131 is a phospholipid ether connected to iodine-131.

The thought process here is that cancer cells will preferentially work the phospholipids into their cell membranes, exposing themselves to the cytotoxic payload. It's an interesting smart bomb-like approach that does not rely on expression of specific molecules the way that approved antibody-drug conjugates do.

But that's a fun science project until we have bona fide trial results. And CLRB delivered here in spades, with some highly encouraging early-stage findings. In its phase 1 trial involving CLR-131 in heavily pretreated patients with multiple myeloma, patients achieved a median overall survival of 22.5 months after a single infusion of the drug.

Looking forward: Considering the patients in this study had received nearly six prior lines of therapy, this is a pretty outstanding result. Consider that in the SIRIUS trial, daratumumab monotherapy led to median overall survival of 17.5 months in heavily pretreated patients. So this early finding is an encouraging sign that CLRB is on to something important in this treatment space.


So there have been some surprises coming down the way, with new indications for older drugs and new treatment strategies showing great promise!

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Regardless, thanks again and have a great day!

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.

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