XORTX Pharma Corp. spent four years quietly assembling the patent estate behind XRx-008, a formulation of oxypurinol aimed at the treatment of rare autosomal dominant polycystic kidney disease (ADPKD), before completing a reverse merger in January to become XORTX Therapeutics Inc. The lead program, now in pre-IND, is positioned to move directly into phase II in expectation of a 505(b)(2) filing, with a mechanism targeting the "disease axis" among obesity, blood pressure and insulin resistance, explained Allen Davidoff, president and CEO of the Calgary, Alberta-based firm.

Although the body of research is growing, therapeutic efforts to curb or reverse hearing loss fall far short of those in the more prominent sensory impairment of vision loss. Several dozen drugs are approved to treat visual disorders such as macular degeneration and glaucoma that can lead to blindness. In contrast, not a single drug is approved to treat or prevent hearing loss.

Not to be confused with a round-the-clock operation, Twentyeight-Seven Therapeutics Inc. (28-7) managed its time over the past two years by quietly assembling a team of scientific luminaries focused on the task of modulating non-coding RNA (ncRNA) as the platform to discover and develop small molecules that increase levels of the tumor suppressor microRNA (miRNA), Let-7, in cancer indications. Those efforts resonated with a stable of investors, including seed investor MPM Capital, which co-led with Novartis Venture Fund 28-7's $65 million series A. Others in the syndicate included JJDC Inc., Vertex Ventures HC, Longwood Fund, and Astellas Venture Management.

Fulcrum Therapeutics Inc. began to assemble its series B round in March, with the goal of advancing its lead program – a small molecule described as a double homeobox protein 4, or DUX4, inhibitor – through IND filing and into the clinic to treat facioscapulohumeral muscular dystrophy (FSHD). The rare genetic neuromuscular disease is characterized by progressive skeletal muscle weakness due to the death of muscle cells and tissue, leading four of five patients to lose their ability to walk by the time they reach early adulthood.

When it comes to sensory functions, the eyes have been the focus of much attention from researchers and drug developers, resulting in last year's breakthrough for Spark Therapeutics Inc. with the FDA nod for gene therapy Luxturna (voretigene neparvovec-rzyl) to treat children and adults with confirmed biallelic RPE65 mutation-associated retinal dystrophy, an ultra-rare disease that leads to vision loss and, sometimes, blindness. 

Emergent Biosolutions Inc. positioned its pick-up of Adapt Pharma Ltd. as an opportunity to diversify and expand the company's presence in medical countermeasures and threats to public health by adding Adapt's flagship product, Narcan (naloxone HCI), developed by Lightlake Therapeutics Inc. and still the only nasal form of naloxone approved by the FDA for emergency treatment of known or suspected opioid overdose. (See BioWorld Today, Nov. 20, 2015.)

Unlike some of its peers, Anavex Life Sciences Corp. has toiled along the tortoise path to drug development with its lead candidate, the oral sigma-1 receptor (S1R) agonist ANAVEX-2-73, in early Alzheimer's disease (AD). For more than a decade, the New York-based company has bet that compounds for AD based on its discovery platform will have competitive advantages over candidates based on the amyloid beta approach, which hypothesizes that plaques in the brain cause the disease. (See BioWorld Today, May 6, 2008.)

Tetraphase Pharmaceuticals Inc. turned the commercial corner with FDA approval of Xerava (eravacycline) to treat complicated intra-abdominal infections, or cIAI.

Bluebird Bio Inc. and privately held Gritstone Oncology Inc. said they plan to collaborate in a broad cancer cell therapy R&D and commercialization pact involving 10 tumor-specific targets across multiple tumor types and, in certain cases, T-cell receptors (TCRs) directed to those targets.

Following its first FDA approval, for Inveltys (loteprednol etabonate ophthalmic suspension 1 percent) to treat inflammation and pain following ocular surgery, developer Kala Pharmaceuticals Inc. said it plans to hire a specialty sales force of about 60 reps to target 80 percent of likely prescribers in preparation to launch the ophthalmic suspension product early next year.