Bio Roundup: CRISPR Babies, Blood Diseases, Big Cancer Nod & More

Xconomy National — 

This was one of those weeks when the world seemed to slip into a new era with no going back. A Chinese researcher, He Jiankui, claimed he helped bring to life two genetically engineered babies, twin girls with a gene disabled to make them immune to HIV infection. Even though the technology He used—in vitro fertilization, CRISPR-Cas9 gene editing, deep sequencing—has been in place for some time to attempt the creation of a gene-altered human, the scientific world hoped for three years that self-restraint would be the watchword until there was rock-solid consensus of safety, efficacy, and justified use for critical medical needs.

He’s work (if it actually happened; there has been no independent verification) did not meet any of these criteria. It was, shall we say, not well received.

The #CRISPRbabies story is moving fast. Here’s a Twitter thread dedicated to capturing the news flow. For the rest of the week’s news, stay right here. It’s roundup time.


—He Jiankui first made his stunning claim of CRISPR-altered babies on the eve of a major gene-editing conference in Hong Kong, then spoke at the conference two days later, saying he was proud of his work and hinting that another CRISPR baby could be on the way.

The presentation was the world’s first chance to see He’s data, hear his explanations, and to ask him questions. Two leading geneticists became his first-ever interviewers. He’s answers, plus the data he presented, drew major concerns about sloppy and potentially dangerous science, ethics and consent, and much more. It also drew worldwide condemnation and a Chinese national investigation.

—At the close of the Hong Kong summit, the organizing committee issued a statement that He’s work was “irresponsible and failed to conform with international norms.”

—Xconomy previewed the annual American Society of Hematology conference, which starts today in San Diego, with a look at some trends and important presentations in six different disease areas: leukemia, lymphoma, multiple myeloma, sickle cell, beta-thalassemia, and hemophilia.

—The federal agency known as CMS that oversees Medicare and Medicaid filled out some details in the Trump adminstration’s previously announced plan to lower high drug prices, and the backlash was swift. CMS will take feedback on the proposal through January.

—Genomics powerhouse WuXi NextCode is acquiring a privately held Irish company that just received a contract to build a health and genomics database of 400,000 volunteers, about 10 percent of Ireland’s population. The company, Genomics Medicine Ireland, will receive up to $400 million from the Irish government and others. No acquisition price was disclosed.


—Bristol-Myers Squibb (NYSE: BMY) suffered another lung-cancer setback when its immunotherapies nivolumab (Opdivo) and ipilimumab (Yervoy) could not do better than placebo in a big Phase 3 study called Checkmate 451 for post-chemotherapy patients. It’s the second recent failure for Bristol in small cell lung cancer, and the bad news could force the FDA to reevaluate an earlier accelerated approval, writes Jacob Plieth of Vantage.

—U.K. biotech Vectura said it would stop working on its asthma drug VR475 after a Phase 3 failure.

—Alkermes (NYSE: ALKS) said its experimental drug ALKS 3831 in a Phase 3 trial led to less weight gain for schizophrenia patients than the staple antipsychotic olanzapine. Alkermes will file for approval of the drug next year, but it’s unclear if doctors will prescribe ALKS 3831 over cheaper generics.

—Vertex Pharmaceuticals (NASDAQ: VRTX) reported success for the first pair of Phase 3 studies of its triple-drug combinations intended to expand cystic fibrosis treatment to vastly more patients. Next year, the company will select which three-drug cocktail it will submit for FDA approval.

—The FDA placed a clinical hold on a mid-stage study of a Zafgen (NASDAQ: ZFGN) diabetes drug. The company did not mention safety problems. It said the FDA halted the study as a precaution because the drug works in a similar way to an earlier Zafgen obesity drug that was linked to deadly blood clots in two patients.


—In a big step for gene-based cancer treatment, the FDA approved larotrectinib (Vitrakvi) from Loxo Oncology (NASDAQ: LOXO), marking the agency’s second OK for a drug that targets a tumor’s DNA fingerprint, no matter where in the body that cancer is found. Larotrectinib treats solid tumors that have an NTRK fusion, which occurs in up to 1 percent of solid tumors, including cancers of the kidney, bladder, stomach, and lung.

—The FDA also approved gilteritinib (Xospata) from Astellas Pharma for acute myeloid leukemia patients who haven’t responded to other treatments and whose cancer has an FLT3 mutation. Some 25 to 30 percent of AML patients have an FLT3 mutation.

—The FDA approved amifampridine (Firdapse), a Catalyst Pharmaceuticals (NASDAQ: CPRX) drug for the rare autoimmune disease Lambert-Eaton myasthenic syndrome.


—Moderna inched closer to what could be the biggest biotech IPO ever. In a new filing, Moderna said it plans to raise as much as $600 million by selling more than 21.7 million shares at $22 and $24 apiece to investors and another 3.3 million to underwriters. The document revealed that founding investor Flagship Pioneering owns 18.1 percent of Moderna, and CEO Stephane Bancel holds 9.4 percent. Partner AstraZeneca is next with a 7.9 percent stake.

—Roivant Sciences launched another “vant” subsidiary, Aruvant Sciences, to develop gene therapies for blood diseases. The lead experimental product, which comes from Cincinnati Children’s Hospital Medical Center, is for sickle cell disease and beta-thalassemia; early Phase 1/2 data will be discussed at ASH on Monday.

—Atlanta-based Antios Pharmaceuticals raised a $25 million Series A round to develop a drug for hepatitis B and potentially hepatitis D. Antios was formed by former leaders of Pharmasset and Idenix, which were both bought out for their hepatitis C drugs.

—Seven months after its lead drug flopped in clinical testing, Edge Therapeutics (NASDAQ: EDGE) agreed to merge with privately-held cancer drug maker PDS Biotechnology. PDS shareholders will get 70 percent of the combined company, and Edge backers 30 percent.

—-Arsanis (NASDAQ: ASNS), also reeling from a trial failure, will merge with X4 Pharmaceuticals of Cambridge, MA. X4 is developing drugs for cancer and rare diseases and is run by Genzyme vet Paula Ragan.

—San Diego-based ViaCyte raised $80 million in Series D financing to continue clinical tests of its stem cell-based diabetes therapy.

Roche is acquiring Jecure Therapeutics in hopes that the startup’s preclinical research leads to new drugs that treat the fatty liver disease known as NASH.

—Redwood City, CA-based Auris Health raised $220 million to support commercialization of its robot, which helps surgeons diagnose and treat lung conditions.

—Rheostat Therapeutics, which is developing neurodegenerative disease therapies based on the cellular process for breaking down and recycling waste, raised a $23 million Series A round to advance toward clinical trials.

—Last week, PellePharm of San Francisco landed $70 million upfront from Denmark’s LEO Pharma to support late-stage clinical testing of a drug for a rare skin cancer.

Ben Fidler and Frank Vinluan contributed to this report.