Know What's Happening in Pharma
Know What's Happening in Pharma
FDA approves KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA has granted approval to KRESLADI, a gene‑editing therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiency‑I in children without a matching bone‑marrow donor. This marks the first FDA‑approved product directly funded by California’s Institute for Regenerative Medicine (CIRM), highlighting the agency’s impact on translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma from GHO Capital
The FDA reauthorized its rare pediatric disease priority review voucher program, bolstering a wave of regulatory incentives that include new guidance on plausible mechanisms and a Rare Disease Evidence Principles framework. Yet ultra‑rare indications still struggle to attract traditional venture capital because of tiny patient populations and limited revenue upside. Non‑profit OTXL is tackling the funding gap by leveraging tax‑exempt status, a partner network, and an AI‑driven platform to de‑risk shelved assets and package them for investors. Simultaneously, royalty‑financing and shared‑risk payer models are emerging as creative financing alternatives.
President Trump negotiated lower prices for some drugs on TrumpRx, but discounts only apply to cash paying patients not using their insurance. Many drugs already had big discounts through manufacturers or coupons, and some have generic equivalents that are already...
President Trump has called for his deals with drug companies to be enacted into legislation. It's hard to know what that means since the deals are not public.
IQVIA reported fourth‑quarter 2025 revenue of $4.34 billion, a 10.3% year‑over‑year increase, driven by strong organic growth and roughly two percentage points from recent acquisitions. Adjusted EBITDA rose 5% to $1.046 billion and adjusted diluted EPS climbed 9.6% to $3.42. The company...
United Therapeutics reported record Q4 2025 revenue of $800 million, a 7% year‑over‑year increase driven by double‑digit growth of its Tyvaso DPI inhaler and Orenitram. The company unveiled new 80‑112 µg Tyvaso DPI cartridges, expanding dosing flexibility, and highlighted unprecedented results from...
Geron Corp reported full‑year 2025 Rytelo net revenue of $184 million, with Q4 sales of $48 million, and projected 2026 revenue between $220 million and $240 million. Operating expenses are expected to dip to $230‑$240 million, a $20 million reduction year‑over‑year, while cash balances sit at...
Sarepta Therapeutics reported a blockbuster fourth quarter, with ELEVIDYS gene‑therapy sales of $384 million, surpassing guidance by $60 million, and total net product revenue of $1.79 billion for 2024, up 56% year‑over‑year. The company achieved GAAP profitability and cash‑flow positivity, confirming the financial...
Schrödinger reported Q2 FY2025 revenue of $54.8 million, up 16% year‑over‑year, driven by 15% software growth to $40.5 million and 19% drug‑discovery growth to $14.2 million. Gross margin slipped to 68% as the mix shifted toward lower‑margin services and new predictive‑toxicology investments. Net...
ACADIA Pharmaceuticals reported record adjusted 2025 revenue of $1.08 billion, up 14% year‑over‑year, driven by strong performance of NUPLAZID and DAYBUE. The company forecast 2026 total revenue between $1.22 billion and $1.28 billion, with NUPLAZID sales expected to reach $760‑$790 million and DAYBUE $460‑$490 million....
Revolution Medicines reported that its lead KRAS inhibitor doraxonrasib secured breakthrough therapy, orphan drug, and a FDA National Priority Voucher, underscoring its potential in pancreatic cancer. Phase I data revealed median overall survival of 13.1‑15.6 months and a 47% objective...
Soleno Therapeutics reported $190.4 million in net revenue for fiscal 2025, representing less than nine months of commercial sales of its VICAT XR therapy. The company turned profitable, posting $20.9 million net income and generating $48.7 million of operating cash in Q4, ending the...
Mirum Pharmaceuticals reported $521 million in 2025 net product sales, a 55% year‑over‑year increase driven by Livmarli and bile‑acid medicines, surpassing guidance. Operating expenses rose to $543 million, yet the company generated positive cash flow and ended the year with $391 million in...
A multi‑omics investigation identified molecular signatures that link alcohol craving to acamprosate treatment response in patients with alcohol use disorder (AUD). The study highlighted IL17RB genetic variants and elevated plasma TNFSF10 as robust predictors of therapeutic success, while proteomic and...
University of Toronto researchers unveiled LUMI‑lab, an AI‑driven self‑driving lab that combined a 28‑million‑molecule foundation model with active‑learning robotics to synthesize and test over 1,700 lipid nanoparticles. The system independently identified brominated lipid tails as a potent new class for...
Researchers at Northwestern University have created HYDRACs, hybrid degrading copolymers that bind and eliminate traditionally "undruggable" oncogenic proteins such as MYC and KRAS. The polymers display target‑recognizing peptides on one side and degron motifs on the other, directing the proteins...
The FDA has granted approval for Dupixent (dupilumab) to treat allergic fungal rhinosinusitis (AFRS) in adults and children six years and older, marking the first indication for this rare sinus disease. The approval follows a 52‑week trial that demonstrated significant...
Accord BioPharma’s Filkri (filgrastim‑laha) received FDA approval as the fifth biosimilar to Amgen’s Neupogen. The indication set mirrors Neupogen’s, covering chemotherapy‑induced neutropenia, AML, bone‑marrow transplant, severe chronic neutropenia, and radiation‑induced syndrome. Approval was based on two randomized trials in healthy...
New data presented at CROI 2026 show that people living with HIV who have maintained viral suppression for 25‑30 years on antiretroviral therapy regain robust HIV‑specific CD8⁺ T‑cell function. Using flow cytometry and single‑cell RNA sequencing, researchers found these cells display...
The FDA has granted traditional approvals to ten oncology drugs that originally received accelerated approval after post‑marketing studies verified clinical benefit. The list includes agents such as Braftovi, Epkinly, and Padcev, with conversion dates ranging from 2022 to 2026. These...
There is one major barrier blocking AI from truly reshaping biology and no one is talking about it. Biology is not static. Most AI breakthroughs so far have treated proteins, cells, and systems as frozen objects. That abstraction worked to get...
Lot's of requests to discuss the Wedbush report on $ABVX today. It's a doozy. Obviously I'm a biased bull so sure, take my thoughts with a grain of salt, but this is truly, awful work from my perspective....
University of Waterloo researchers have engineered the anaerobic bacterium Clostridium sporogenes to eat solid tumors from the inside out. By inserting an oxygen‑tolerant gene and coupling it to a quorum‑sensing circuit, the bacteria survive at the tumor periphery only after...
Th FDA is absolutely correct, and confirms what I’ve said all along. This $IBRX @DrPatrick “lymphopenia” push means nothing until or unless they come up with real data showing patient benefit. The “number” doesn’t matter.
$HELP adds former Pfizer CMO Dr. Freda Lewis-Hall to its board. Big pharma credibility bump. Now the pipeline has to earn it. https://t.co/CK7PsBE9B8
FDA Commissioner Marty Makary argues that any drug deemed safe, non‑addictive and not prone to misuse should be sold over the counter. He cites anti‑nausea medicines, hormonal contraception and common analgesics as candidates, while urging regulators to abandon a paternalistic...
If FDA is serious about moving with urgency in rare disease, the key question is evidentiary standards. Speed matters. But clarity on what constitutes “adequate and well-controlled” in ultra-small populations matters more. Predictability > rhetoric https://t.co/XVXg71NmGq
Given 1mg/week of Zepound showed 0.7% reduction in A1c in trials, and so is actually an efficacious dose, this microdose would just be $30/month. (Would imply using for longer than recommended after opening).
Pharmaceutical and biotech firms are mobilizing against two Trump administration proposals that would tie Medicare drug prices to those paid in other affluent nations. The initiatives – the GLOBE model for Part B physician‑administered drugs and the GUARD model for Part D...
The evolution to hepta-refractory myeloma involves sequential loss of CD38, BCMA and GPRC5D [Feb 17, 2026] Riedhammer et al. @LeukemiaJnl https://t.co/EyGBz7sEQz #mmsm #PrecisionMedicine #caxtx
A wave of unapproved anti‑aging peptide injections is sweeping social media, driven by influencers and celebrities. Most products are sold on the gray market, often from China, and lack FDA approval or robust clinical data. Experts warn of safety risks,...
Novo Nordisk announced steep price cuts for its GLP‑1 diabetes and obesity drugs, aiming to broaden patient access amid intensifying pricing scrutiny. AbbVie is committing $380 million to expand API manufacturing capacity, reinforcing supply‑chain resilience. Bora Pharmaceuticals and GSK sealed a...
Vir climbs on Astellas deal, study results for ‘masked’ T cell engager https://t.co/336c9FJdjt by @Lilah_Alvarado $VIR + 27% $JANX $ALPMY
Novo to cut GLP-1 drug prices; Palvella soars on study data https://t.co/UAs4IETgPX $NVO $PVLA+ 32% $GOSS $VNDA $PFE #biotech
Researchers at Cornell uncovered a previously unknown thermogenic pathway in white adipocytes, where free fatty acids induce proton leakage through the mitochondrial ADP/ATP carrier (AAC). This AAC‑mediated uncoupling mirrors brown‑fat heat production without involving UCP1. In mouse models, enhancing intracellular...
The FDA’s Generic Drugs Program reported 217 approvals for FY‑2026, including 23 first‑time generics and 45 first‑cycle approvals. Tentative approvals reached 89, while complete responses rose to 402, reflecting a busy review pipeline. Prior Approval Supplements (PAS) saw 532 approvals,...
The FDA has published an updated list of 189 pediatric exclusivity determinations made under Section 505A of the Federal Food, Drug, and Cosmetic Act since September 2007. Each entry includes the drug name, sponsor, determination date, and links to the original Written...
Failing to find a buyer, BioMarin is voluntarily withdrawing Roctavian from the market. A fitting end for what was once one of the highest-profile gene therapies in development — and a reminder of the gap between promising science and a viable...
The Center for Drug Evaluation and Research (CDER) released its February 2026 Guidance Agenda, outlining the draft and revised guidances it plans to develop this calendar year. The agenda features the first issuance of a Level 1 draft guidance alongside updates to...
Novo, searching for a spark, spotlights new data for three-pronged obesity drug https://t.co/pPo9Oc1wlF $NVO $LLY #obesity
FDA Issues Plausible Mechanism Pathway Draft Guidance to Spur Innovation for Individualized Therapies https://t.co/OZW6Q27zMa via @Inside_PM
The FDA issued a warning letter to MedisourceRx after an inspection found the facility’s compounded sterile drugs failed to meet 503B outsourcing‑facility requirements. Specific violations include marketing unapproved new drugs, misbranding due to inadequate labeling, and inadequate adverse‑event reporting. The...
Immedica wins accelerated approval for once-rejected rare disease drug, plans to sell PRV - https://t.co/wU3AfqQzjw
Pfizer’s post‑COVID strategy pivots from vaccine and antiviral sales to rebuilding growth through oncology and metabolic assets. The 2026 guidance excludes COVID revenue, projects modest 4% operational growth, and anticipates a $1.5 billion patent‑cliff hit. To offset these pressures, Pfizer completed...
Stäubli Robotics unveiled the next‑generation Sterimove at INTERPHEX 2026, positioning it as the world’s first mobile robot certified for Grade A, B, C and D cleanroom environments. The platform combines sealed, cleanroom‑compatible construction with advanced collision‑avoidance sensing, dual emergency stops...
Our perspectives piece on Agentic AI for biomedical research has been published in Nature Biotechnology https://t.co/PpFeGcvv0x #agenticAI #artificialintelligence #agents https://t.co/xoADUc7T7K
Slate starts up with $130M and a headache drug from China https://t.co/6DEltQAI8s by @gwendolynawu #biotech #startups $HLUBF
Sciwind Biosciences granted Pfizer China exclusive rights to commercialize ecnoglutide injection in Mainland China. Sciwind will keep the marketing authorization and handle R&D, registration, manufacturing, and supply, receiving up to $495 million in milestone payments. Ecnoglutide, a cAMP‑biased GLP‑1 receptor agonist,...
Cross trial and China data caveats, but 19.7% at 24 weeks for $NVO triple G looks quite good. Probably a few % points better than $LLY triple G at 24-week timepoint. https://t.co/ylgf4vTEOq
Researchers at Sultan Qaboos University have isolated three antimicrobial peptides from dromedary camels, with CdPG-3 and CdCATH showing potent activity against methicillin‑resistant Staphylococcus aureus and multidrug‑resistant Escherichia coli. The peptides kill bacteria by disrupting their membranes while exhibiting low toxicity...
Immuto’s CEO Faraz Choudhury explains that conventional target discovery, which focuses on gene mutations and protein abundance, overlooks disease‑specific protein conformations. The company’s platform maps surface protein conformations (SPCs) in living, patient‑derived models using high‑resolution mass spectrometry and AI‑driven analysis,...
