Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Biogen pushes anti-tau drug into Phase 3 after endpoint miss
Biogen's tau-targeting investigational antisense oligonucleotide therapy diranersen failed its primary Phase 2 endpoint of demonstrating a dose-dependent reduction in brain tau, dropping the company's shares by around eight percent. However, the drug still showed enough promise to propel it into late-stage testing. Data from the CELIA trial presented at the Alzheimer's Association International Conference in London showed that all three doses outperformed placebo after 18 months, although the lowest 60mg dose produced the strongest results. That arm slowed decline on the Clinical Dementia Rating Sum of Boxes (CDR-SB) scale by 26 percent and delivered consistent reductions in brain tau, marking the first Phase 2 evidence that a drug can remove toxic tau tangles in people with Alzheimer's disease. Despite the failed primary endpoint and questions over the dose-response findings, the company plans to advance diranersen into Phase 3, with the cognitive signal and tau reductions offering a potential new approach alongside approved amyloid-targeting therapies. – Bree Foster
Lilly acquires AtaiBeckley in a $2.8 billion psychedelics bet on treatment-resistant depression
Eli Lilly announced this week it will acquire AtaiBeckley for $6.75 per share in cash plus up to $2.50 per share in contingent value rights tied to development and regulatory milestones, representing an upfront equity value of approximately $2.8 billion with up to $1 billion more on the table. The deal centers on BPL-003 (mebufotenin benzoate), a synthetic intranasal form of 5-MeO-DMT that has received FDA Breakthrough Therapy designation and recently initiated Phase 3 activities for treatment-resistant depression. In a Phase 2b study, a single roughly two-hour in-clinic session produced rapid and durable reductions in depressive symptoms lasting months. A second program, VLS-01, a buccal film formulation of DMT, is currently in Phase 2b for treatment-resistant depression, with an additional (R)-MDMA program in Phase 2 for social anxiety disorder. AtaiBeckley's approach is rooted in neuroplasticity — the idea that treatment-resistant depression involves a loss of synaptic connectivity rather than simply a neurotransmitter imbalance, and that psychedelic-class compounds may restore it. The acquisition signals that Lilly, fresh off reshaping metabolic disease with GLP-1s, is placing a serious bet that psychiatry is next — and that psychedelic-assisted therapy, long on the scientific fringe, has enough clinical evidence to justify a multibillion-dollar commitment. – Andrea Corona
FDA approves first oral cholesterol pill
On Thursday, Merck’s novel macrocyclic peptide, Lipfendra, became the world’s first oral pill approved to treat high cholesterol. The drug works by binding to the PCSK9 (proprotein convertase subtilisin/kexin type 9) protein and stopping it from causing damage to low-density lipoprotein (LDL) receptors that keep cholesterol low. Injectable versions of PCSK9 inhibitors have already been on the market since 2015, however the pill version is much more palatable for consumers than shots — and was shown to be just as effective in Phase 3 trials, lowering LDL cholesterol by nearly 60 percent. This new option could dominate the market, which is currently valued around $22.6 billion. “This is a pivotal moment as we bring the first US FDA-approved oral PCSK9 inhibitor to adults with high LDL-C, offering patients an important new option. We’re proud of our work with regulators on this rigorous and efficient review process,” said Dean Li, President of Merck Research Laboratories in the press release. – Allison Whitten
Drug trial aims to prevent Bundibugyo virus disease after exposure
Researchers have begun enrolling patients in the Democratic Republic of the Congo and Uganda into a clinical trial testing whether a drug can prevent Bundibugyo virus disease, an illness with no approved point-of-care diagnostic tests, therapeutics, or vaccines. The EBO-PEP trial will assess whether Gilead Sciences' antiviral obeldesivir can prevent disease in people who have had high-risk contact with confirmed cases. The trial, which has been in preparation since 2024, aims to enroll nearly 1,000 adults and children over 12 who have been exposed to the virus but do not yet have symptoms. Recruitment began on July 14 in Ituri province, the outbreak's epicenter, with researchers monitoring participants daily for 21 days. A separate compassionate-use protocol will provide Gilead’s remdesivir to exposed children under 12 and pregnant or breastfeeding women, for whom there are currently insufficient data to support the use of obeldesivir. The Bundibugyo virus has caused 2,032 confirmed cases and 756 deaths as of July 13. – Bree Foster
An oral minoxidil pill shows strong Phase 2 results for female pattern hair loss, a first in nearly 30 years
Veradermics announced this week positive topline results from its open-label Phase 2 trial of VDPHL01, a proprietary extended-release oral minoxidil formulation, in women with mild-to-moderate pattern hair loss. Among participants who received either a once-daily or twice-daily 4.5mg dose for six months, roughly 89 and 90 percent respectively reported improved or much improved hair coverage, with improvements observed as early as month two. Mean non-vellus hair count increased by approximately 22.7 and 23.3 hairs per square centimeter in the once-daily and twice-daily arms. The drug showed no treatment-related serious adverse events and no cardiac adverse events of special interest, a notable safety point given that cardiovascular effects have historically complicated oral minoxidil use. Unlike immediate-release formulations, VDPHL01 uses a gel matrix designed to avoid peak concentration spikes while extending time above the threshold needed to stimulate hair growth. If approved, it would be the first FDA-approved oral treatment for female pattern hair loss, a condition affecting an estimated 30 million women in the US with no current prescription oral options. A Phase 2/3 registration-directed trial is underway, with topline data expected in the first half of 2027. – Andrea Corona
Insilico and Bora collaborate in $2.5B deal for AI drug discovery
Insilico Medicine announced plans to partner with Taiwan-based contract drugmaker Bora Pharmaceuticals in a deal that could exceed $2.5 billion. The collaboration will aim to merge Insilico’s proprietary Pharma.AI platform with Bora’s global development, manufacturing, quality, and commercialization. “Together, we aim to build a scalable and repeatable model that can advance promising drug candidates more efficiently, positioning Bora at the forefront of AI-enabled pharmaceutical development and manufacturing, and establish a long-term growth platform through the development and commercialization of high-value proprietary assets,” said Bobby Sheng, CEO of Bora Pharmaceuticals, in the press release. For Insilico, the deal follows several other high-profile partnerships earlier this year between Lilly and Takeda. – Allison Whitten
