{"id":378486,"date":"2023-12-11T09:26:53","date_gmt":"2023-12-11T14:26:53","guid":{"rendered":"https:\/\/platohealth.ai\/the-biotech-news-you-missed-from-the-weekend\/"},"modified":"2023-12-12T03:30:53","modified_gmt":"2023-12-12T08:30:53","slug":"the-biotech-news-you-missed-from-the-weekend","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/the-biotech-news-you-missed-from-the-weekend\/","title":{"rendered":"The biotech news you missed from the weekend","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"
<\/div>\n

Want to stay on top of the science and politics driving biotech today?\u00a0Sign up<\/a>\u00a0to get our biotech newsletter in your inbox.<\/em><\/p>\n

Hello from ASH! Writing this Readout from a press room at the annual hematology confab here in San Diego. Today\u2019s edition is chockfull of Vertex content, plus some extras from ASH and elsewhere.<\/p>\n

\n

advertisement<\/p>\n<\/div>\n

Vertex\u2019s Joshua Boger is colorful<\/h2>\n

Joshua Boger, founder of Vertex Pharmaceuticals, still finds the company\u2019s new\u00a0sickle cell approval<\/a>\u00a0flabbergasting:\u00a0\u201cI would have lost a house in a bar bet if I had bet that Vertex would be the first company to bring a CRISPR drug to the world,\u201d he told STAT. In a wide-ranging interview, Boger \u2014 who stepped down from his helm in 2017 after 28 years there \u2014 spoke about his work at Vertex, how he imagines biotech\u2019s future, and what he thinks of Elon Musk. (Spoiler: He\u2019s \u201cextremely weird. He just doesn\u2019t connect with people.\u201d)<\/p>\n

As for biotech, despite the generally gloomy environment, Boger has \u201cnever been more excited by the scientific and medical possibilities.\u201d He also considers it to be \u201cmathematical lunacy to blame health care costs on drug prices.\u201d<\/p>\n

Read more.<\/a><\/p>\n

\n

advertisement<\/p>\n<\/div>\n

CAR-T drugs effective in autoimmune disease<\/h2>\n

Two-and-a-half years after receiving a CAR-T infusion, 15 patients with autoimmune diseases remain in complete remission, new data presented at ASH show. This includes nine people with lupus, four with systemic sclerosis, and three with inflammatory myositis.<\/p>\n

CAR-T cells were initially engineered to fight B cell cancers, but they are now being explored as targeted weapons against the malfunctioning B cells in autoimmune diseases. Researchers at the University Hospital Erlangen in Germany tested this out first in a young patient with lupus: \u201cShe had no treatment options, and it became clear if she didn\u2019t receive any treatment she would be dead in a few months,\u201d the study\u2019s lead author told STAT. \u201cShe couldn\u2019t sleep laying down because her heart was failing. She couldn\u2019t walk more than 10 meters because her lungs were failing.\u201d<\/p>\n

The CAR-T approach here was risky, because it wasn\u2019t clear if the lupus was driven by B cells or T cells, \u201cso nobody knew if we were going to kill that patient or help her,\u201d he said. \u201cThe patient in the end was super brave.\u201d<\/p>\n

Now, the patient is back to a normal life, and able to jog five times a week. The subsequent 14 patients treated with this approach have seen similar results, and some have seen some reversal of the organ damage inflicted by their respective autoimmune diseases.<\/p>\n

Read more.<\/a><\/p>\n

The backstory to Vertex\u2019s experimental non-opioid painkiller<\/h2>\n

The quest for non-opioid painkillers has been pretty bleak \u2014 until now, perhaps. Early next year, Vertex Pharmaceuticals will release late-stage trial data of a drug that blocks pain-sensing neurons from signaling the brain. The drug\u2019s being tested in post-surgical pain, as well as in diabetic patients with chronic nerve pain. If the studies are successful, Vertex is looking to win approval for the small molecule drug, dubbed VX-548, to treat moderate to severe acute pain. Analysts think it has blockbuster potential, with estimates that sales might reach as much as $5 billion by 2030.<\/p>\n

Vertex has been developing this experimental drug for decades. It\u2019s a long, winding story that involves Nobel Prize-winning proteins that glow and a 10-year-old Pakistani street performer who felt no pain.<\/p>\n

Read more.<\/a><\/p>\n

ICER lays out cases of price hikes without justification<\/h2>\n

Last year, costs spiked for eight common medicines without any valid rationale \u2014 leading to $1.2 billion in extra costs to patients and insurers. AbbVie is responsible for the biggest spike: The wholesale cost for its blockbuster inflammation drug Humira rose 7.1% \u2014 leading to an additional $386 million spent on this drug, according to a new ICER report. Second in line was Johnson & Johnson\u2019s multiple myeloma drug Darzalex, whose wholesale cost rose 6.8%, leading to $248 million more in spending. And third is Pfizer\u2019s breast cancer drug Ibrance, whose wholesale price rose 6.9% and resulted in $151 in additional spending.<\/p>\n

\u201cWe continue to see list price increases above inflation for many of the most costly drugs,\u201d ICER chief medical officer David Rind said, adding that \u201cmany had no substantial new evidence to support such price increases.\u201d<\/p>\n

Read more.<\/a><\/p>\n

More reads<\/h2>\n