News briefing: Intellia hits clinic with CRISPR therapy; Vifor continues to spend, paying up to $340M for DGF program
Another CRISPR therapy has entered the clinic. Intellia Therapeutics, the Cambridge gene editing biotech, announced today that they dosed their first patient with a therapy meant to treat transthyretin amyloidosis, a rare genetic disease caused by a mutation in a liver protein.
Although a handful of CRISPR therapies have already entered the clinic at Intellia and elsewhere, the new study is notable for being just the second study to inject CRISPR directly into a patient. Other approaches, such as those meant to treat sickle cell anemia, involve taking cells out of a patient, editing them, and then infusing them back in.
Editing cells in vivo, by contrast, is more difficult, as it’s dependent on the ability to target to CRISPR to the right part of the body. The liver, though, is one of the easiest areas to target for any genetic therapy. The eye is also a relatively straightforward target, and Editas tested an in vivo CRISPR treatment for blindness earlier this year, marking the first time the gene therapy tool was used directly in humans.
Intellia has plenty of competition for transthyretin amyloidosis. In the past few years, Pfizer, Ionis and Alnylam have each developed drugs proven to ameliorate the condition. Intellia, however, is gunning for a cure. — Jason Mast
Vifor continues to spend, paying up to $340M for DGF program
A few weeks after shelling out $440 million to market a pruritus drug in the US, Vifor is wheeling and dealing once again.
The Swiss company has agreed to license and commercialize a program from Angion Biomedica for the treatment of delayed graft function (DGF) and cardiac surgery-associated acute kidney injury. Angion’s experimental drug, dubbed ANG-3777, will net the biotech up to $80 million in cash, equity investment and clinical milestones.
Angion can also receive up to $260 million in regulatory milestones, including if and when the drug scores approval in the US and EU, as well as up to 40% in royalties on sales. Vifor’s license will apply globally except in China, Taiwan, Hong Kong and Macau for all of ANG-3777’s nephrology indications.
While Vifor will handle the marketing for all territories where the drug could reach approval, Angion will continue to run the clinical development programs. The companies will share responsibilities for regulatory filings.
The companies say that ANG-3777 was engineered to mimic the biological activity of the hepatocyte growth factor, activating critical pathways in the body’s organ repair process after an acute injury. — Max Gelman
Inmagene snags $21M Series B, bringing total raise to $40 million
Shanghai-based Inmagene reeled in a $21 million Series B to fuel its mission of in-licensing and developing drugs in China.
The latest round, led by Vertex Ventures China, brings Inmagene’s total raise up to $40 million. According to the company, two “engines” drive its pipeline: in-licensing drugs from other countries that “fit China” to conduct global clinical trials, and conducting drug development. The company’s lead candidate, IMG-20, was created in partnership with Affibody and MedImmune, an AstraZeneca subsidiary. The candidate, which showed positive safety and efficacy results in trials with psoriasis patients, is about to enter global registration trials, according to Inmagene.
“We are grateful to Vertex, Panacea, Kunlun, SCVC and other investors for their strong support,” Inmagene chairman and CEO Jonathan Wang said in a statement. “This financing should help strengthen Inmagene’s leading position in immunology drug development in China.” — Nicole DeFeudis
Day after taking poison pill, Aptevo soars on early glance at bispecific data
Seattle-based Aptevo Therapeutics has adopted a stockholder rights plan — commonly called a poison pill on the Street — to fend off a potential hostile takeover.
The move comes in the wake of Tang Capital Partners’ disclosure that it now owns 54% of the biotech. Soon afterwards, Aptevo also touted a second complete remission reported from the Phase I trial of its CD123xCD3 bispecific in acute myeloid leukemia, sending shares $APVO up 115.70% to $51.12.
Under the rights plan, shareholders were given one right for each share they own, which would become exercisable only if a person or a group acquires ownership of 10% or more in a transaction not approved by Aptevo’s board. The aim is to let stockholders purchase shares at discount, so as to effectively dilute the ownership of the hostile party.
Without naming names, Aptevo said it’s looking to protect the company and its shareholders from third parties that are not acting in their best interests. — Amber Tong