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June 18, 2019 Bioscience Notebook

Japan greenlights Alexion’s new blood-disorder drug

PHOTO | Contributed Alexion Pharmaceuticals' research facility at 100 College St.

Alexion Pharmaceuticals Inc. said Tuesday it has won regulatory approval to market its new drug Ultomiris in Japan to patients with an ultra-rare blood disease.

The drug, which received U.S. Food & Drug Administration approval in December, is Alexion’s successor to Soliris, the blockbuster drug that put the Elm City-born company on the map as a biotech powerhouse a decade ago.

The decision by the Japanese Ministry of Health, Labour and Welfare clears the way for physicians in Japan to prescribe Ultomiris to patients suffering from paroxysmal nocturnal hemoglobinuria (PNH), a life-threatening disorder in which a patient’s immune system prematurely destroys red blood cells.

Like Soliris, Ultomiris inhibits c5, an immune system protein overactivated in PNH patients. But it requires fewer infusions than its predecessor, reducing the treatment burden for patients, according to Alexion.

Alexion says the drug has the potential to become the new standard of care for the disease. The company, which has a research facility in New Haven, released data last week showing the drug continued to be safe and effective in PNH patients after one year of using it.

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New Haven neuro drug developer Biohaven Pharmaceuticals Holding Co. Ltd., which is nearing the regulatory finish line on drugs for migraine and ALS, announced Monday it would sell $300 million worth of common shares in a public offering.

Goldman Sachs & Co. LLC and Piper Jaffray & Co. are acting as joint book-running managers.

The news, announced after markets closed Monday, sent the company’s stock plummeting by 29 percent in pre-market trading early Tuesday. Biohaven stock, which trades on the NYSE under the symbol BHVN, opened at $40.48, its lowest price since January.

Biohaven said it would use proceeds from the sale for working capital and to advance the development of its calcitonin gene-related peptide (CGRP) receptor antagonists and glutamate modulating drugs.

The FDA is expected to decide in July whether to approve Biohaven’s Nurtec, a thin-film, orally dissolving formulation of an older drug for amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s Disease.

The company had been heavily rumored this spring to be a candidate for takeover after Bloomberg reported, citing unnamed sources, that the company was in talks about a possible sale.

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New Haven immunotherapy biotech Kleo Pharmaceuticals Inc. announced Tuesday that it has singled out its first drug candidate — a potential treatment for the blood cancer multiple myeloma.

The experimental drug, CD38-ARM, uses technology developed at Yale that can “recruit” antibodies to target multiple myeloma cancer cells for destruction by the body’s immune system. CD38 is a protein linked to multiple myeloma and other blood cancers.

The company expects to advance the treatment into clinical trials next year.

Kleo CEO Douglas Manion said the drug aims to be a safer and more effective therapy than existing anti-CD38 treatments for the disease, a cancer of the white blood cells.

The compound is the first to come out of the company’s recent alliance with Tokyo biopharma PeptiDream Inc. which led Kleo’s $21 million Series B financing round last November. New Haven’s Biohaven Pharmaceuticals also has a 42-percent stake in the company.

Kleo’s ARMs (antibody recruiting molecules) are synthetic compounds that act like biologics (drugs derived from living organisms) but are smaller, more versatile, and faster and cheaper to produce, according to the company.

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New Haven’s Trevi Therapeutics Inc., which went public in May, reported first-quarter 2019 losses of $4.8 million, a slight uptick from $4.1 million a year ago.

The biopharma, which is developing a treatment for chronic itching, said it spent $3.3 million on research and development during the period ended March 31, 2019, up from $2.4 million in the year-ago period.

The company attributed the higher spending to the start of a Phase 2b/3 trial on its lead drug candidate.

General and administrative expenses tallied $1.5 million, up from $800,000 a year ago, which it blamed on increased personnel expenses and consulting and professional fees.

The company said it had $12.9 million in cash on hand as of the end of the first quarter, compared to $7.2 million at the end of 2018.

It netted another $62.6 million in its IPO and concurrent private placement in May.

Founded in 2011, Trevi has developed a new version of an older opioid, nalbuphine ER, which is already FDA-approved to treat pain.

Although it is an opioid, the drug is not considered a controlled substance and the federal government does not classify it as having a high potential for abuse.

Trevi said last week it had started a Phase 2 clinical trial on the drug for a second condition — chronic cough in patients with idiopathic pulmonary fibrosis.

Contact Natalie Missakian at news@newhavenbiz.com

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