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Other Therapy Areas

Set Alert for Other Therapy Areas

Aesthetics

Set Alert for Aesthetics

Revance Wins First Daxxify Therapeutic Indication, Plans Limited Initial Rollout

Similar to its post-approval strategy in aesthetics less than a year ago, Revance will initially target a small number of doctors as it Daxxify to market in the US for cervical dystonia with a broader launch in 2024.

Approvals Launches

BiologicsMD Uses Unique Fusion Proteins To Treat Alopecia Safely

Emerging Company Profile: The US biotech is developing a series of recombinant fusion proteins to tackle alopecia and other diseases without supressing the immune system.

Emerging Company Profile Companies

Finance Watch: Brent Saunders Plots Return To Deal-Making With A SPAC IPO

Public Company Edition: The former Allergan CEO is taking a special purpose acquisition corporation public to generate $460m for deals in familiar biopharma niches. Also, IPOs are expected to keep up a robust pace in the fall and Albireo raises $160m on the success of its drug for pediatric liver diseases.

Financing Business Strategies

ITC Takes Medytox’s Hand In US Botulinum Case, Bans Daewoong’s Imports

USITC’s initial decision in the Medytox and Daewoong botulinum dispute is favorable to Medytox and poised to take a toll on the Korean firm and partner Evolus as they face a 10-year ban on imports of their product to the US.

Aesthetics Legal Issues
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Blood and Coagulation Disorders

Set Alert for Blood and Coagulation Disorders

Agios Shows Pyrukynd Can Reduce Transfusions In Thalassemia

Having already posted Phase III success in non-transfusion dependent patients, Agios’s Pyrukynd now has shown the ability to reduce transfusions in alpha and beta thalassemia patients.

Clinical Trials Business Strategies

EHA Preview: ASCO Overlap Gives Smaller Companies Chance To Shine

Cargo Therapeutics has got an early boost from its abstract to be presented the European meeting, while Shattuck has suffered on safety doubts for its CD47 inhibitor.

Clinical Trials Commercial

EHA Preview: Agios Takes Spotlight In Thalassemia

The company expects to have data from transfusion-dependent and non-transfusion dependent patients this year, with a potential expanded US approval in 2025 for Pyrukynd.

Companies Clinical Trials

Novo Takes On Roche In Hemophilia

Mim8 looks good in hemophilia A but Hemlibra’s lead is all but unassailable.

Clinical Trials Commercial

Dental Oral

Set Alert for Dental Oral

Start-up AFYX Applies Ultra-Sticky Patch Technology To Underserved Oral Mucosal Disease

Emerging Company Profile: Patients with oral lichen planus suffer from painful lesions in the mouth that are hard to treat with topical steroids, leaving room for development of a new delivery method for the established clobetasol.

StartUps and SMEs Dental Oral
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Dermatology

Set Alert for Dermatology

InflaRx Undaunted By Dermatology Rivals

The group says INF904 can make billions in two highly competitive skin diseases, but this looks optimistic.

Clinical Trials Commercial

Soterios Topical Could Take On JAK Class In Alopecia Areata

The UK biotech unveils Phase II data showing an ability to reduce severity of mild-to-moderate AA, with full hair restoration in some patients. If approved, STS-01 will compete with Olumiant and Litfulo.

Clinical Trials Drug Approval Standards

J&J Continues Atopic Dermatitis Effort With Numab Deal

Johnson & Johnson, after several prior failed R&D attempts in AD, will pay Numab $1.25bn to acquire a subsidiary and its Phase II-ready bispecific antibody for key unmet needs in the disease.

M & A Business Strategies

J&J Gets Into The Anti-TSLP Game With Proteologix Buyout

The privately held biotech will bring J&J a pair of bispecific antibodies expected to target multiple pathologic pathways in atopic dermatitis and asthma.

M & A Business Strategies
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Ear

Set Alert for Ear

Regeneron Gene Therapy Reaches New Heights In Restoring Hearing

The US biotech’s DB-OTO has made headlines worldwide after a baby with profound genetic deafness experienced improved hearing to normal levels within 24 weeks after a single intracochlear injection.

Ear Gene Therapy

Fennec Finds Ex-US Commercial Partner For Pedmark In UK’s Norgine

Norgine will market Pedmark, indicated to reduce risk of chemotherapy-related hearing loss in pediatric cancer, in the EU and UK. It will seek additional approvals in Australia and New Zealand.

Deals Business Strategies

Acousia To Refresh Stagnant Hearing Loss Space With New Potassium Channel Approach

The German biotech plans to prevent chemotherapy-induced hearing loss and treat chronic hearing loss by targeting a potassium channel in the ear’s sensory hair cells as it tackles a widespread but side-lined area.

Emerging Company Profile Companies

Lilly Builds On Gene Therapy Focus With Akouos Takeout

With the planned $487m acquisition of sensorineural-focused Akouos, Lilly makes its second gene therapy M&A play in two years, on top of partnering/financing activity in the space.

M & A Gene Therapy
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Ear Nose Throat

Set Alert for Ear Nose Throat

Regeneron Strikes A CHORD With Deafness Gene Therapy

A very early success with a gene therapy for a rare form of deafness puts Regeneron ahead of Lilly and Sensorion.

Gene Therapy Ear, Nose & Throat

Fennec Prepared For Market As Pedmark Finally Gets FDA Nod

After successive CRLs in 2020 and 2021, the drug gained a broad label for hearing loss in children treated with cisplatin for localized, non-metastatic solid tumors.

Approvals Cancer

Optinose Looking For A Partner To Optimize Big Opportunity In Chronic Sinusitis

With positive Phase III data for its Xhance (fluticasone propionate) nasal spray in chronic sinusitis, Optinose believes it is poised to expand the target market for the drug 10-fold.

Ear, Nose & Throat Clinical Trials

Budesonide CRL Another Setback For Takeda’s ‘Wave 1’ Pipeline

Setback for company's near-term launch goals and US patients with rare esophageal inflammatory condition as regulator asks for additional clinical data. 

United States Complete Response Letters

Gastrointestinal

Set Alert for Gastrointestinal

Tremfya Shows Head-To-Head Edge Versus Stelara In Crohn’s

Johnson & Johnson tested Tremfya against its established Stelara as the “benchmark” in IBD. Tremfya, already up for approval in ulcerative colitis, is planned for filing in Crohn’s later in 2024.

Clinical Trials Business Strategies

MRM Health Pulls A Pouchitis Hit Out Of The Bag

The mid-stage success is encouraging, but taking on Takeda won’t be easy.

Clinical Trials Companies

BMS’s Plans For Zeposia In Crohn’s Dubious Following Phase III Miss

The oral S1P modulator, approved to treat ulcerative colitis and MS, missed its primary endpoint in the first of two Phase III induction studies in moderate-to-severe Crohn’s disease.

Clinical Trials Drug Approval Standards

Mirador Lands $400m To Pursue I&I Precision Medicine Approach

Ex-Prometheus team will use its big data platform to validate targets, select indications and optimize trial enrollment in the development of precision therapies for inflammatory and immune disorders.

Financing Business Strategies
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Gynecology Urology

Set Alert for Gynecology Urology

Bayer’s Menopause Drug Has An Edge But Still Much To Prove

Phase III results suggest Bayer’s elinzanetant has a safety and efficacy advantage over its Astellas rival in reducing hot flashes, but investors are still not convinced the drug will be a blockbuster.

Companies Clinical Trials

Odd Coupling: J&J, RallyBio Collaborate As Only Firms Focused On FNAIT

While not co-developing each other’s drugs, J&J and financially troubled RallyBio are working on complementary approaches to the rare disease fetal and neonatal alloimmune thrombocytopenia.

Rare Diseases Business Strategies

Genmab Adds To Sector ADC Buying Spree With $1.8bn ProfoundBio Acquisition

The buyout reflects the Danish biotech’s plans to expand in oncology drug development and commercialization.

Cancer M & A

Scrip Asks…What Does 2024 Hold For Biopharma? Part 6: Therapeutic Area Advances

More than 100 biopharma executives and experts told Scrip their predictions for therapeutic area advances in the coming year. The recent commercial success of GLP-1s in diabetes and obesity and their potential in further disease areas fuelled excitement around the metabolic space. Expectations were also positive in neurology following the launch of Leqembi for Alzheimer’s disease in 2023, while the multiple opportunities to improve cancer treatment kept oncology top of the pile overall.

Scrip Asks Advanced Therapies

Liver Hepatic

Set Alert for Liver Hepatic

Akero Shows More Strong Fibrosis Data, But Analysts Cite Safety Worry

Akero is developing weekly injectable efruxifermin for MASH with solid 96-week efficacy data and three Phase III studies underway. But will it find a treatment niche in a competitive space?

Business Strategies Clinical Trials

Ipsen Gets Ahead Of The Qirv in Cholangitis

Iqirvo gains accelerated approval, but Gilead’s seladelpar is probably only two months behind.

Approvals Commercial

EASL: Sagimet Pill Is Overlooked But Could Make A Splash In MASH

While injectables have had all the limelight, some analysts see a bright future for Sagimet’s oral denifanstat, potentially as a backbone therapy in combination with other treatments for MASH.

Clinical Trials Liver & Hepatic

Viking Bolsters MASH Case With 52-Week Histology Data

Viking will look to plan a Phase III program with the FDA after showing 52-week success on MASH resolution and fibrosis reduction with VK2809. The firm still hopes to move forward with a partner.

Clinical Trials Business Strategies

Ophthalmic

Set Alert for Ophthalmic

Skye Bioscience Looks To Build Appetite For CB1 Obesity Drug

The company will take on Novo Nordisk in reviving the CB1 inhibitor mechanism to develop a more tolerable long-term weight loss drug, after exiting ophthalmology.

Companies Clinical Trials

Oculis Looks To Move Dry Eye Candidate To Phase III Despite Mixed Dataset

Oculis’s licaminlimab showed ability to treat a sign of dry eye disease in a genetic subpopulation, but data in the fuller Phase IIb trial enrollment were less impressive.

Clinical Trials Business Strategies

Merck & Co. To Enter Ophthalmology With EyeBio Buyout

Veteran developers stay on to accelerate development of potential first-in-class drug to challenge Bayer/Regeneron’s Eylea and Roche’s Vabysmo in a deal worth up to $3bn.

Clinical Trials Commercial

Rezolute Sees Big Opportunity For Oral Eye Disease Drug

The company reported positive topline data from a Phase II study testing RZ402 in patients with diabetic macular edema.

Clinical Trials Ophthalmic
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Orthopedics

Set Alert for Orthopedics

Ultragenyx Data De-Risk Phase III Trial Of Setrusumab

The Mereo-partnered drug is in pivotal development for osteogenesis imperfecta. Ultragenyx also recently reported positive Phase III data for a rare disease gene therapy.

Clinical Trials Drug Approval Standards

Bridgebio Ready To Challenge BioMarin In Achondroplasia

The company sees a $5bn opportunity to treat children with achondroplasia and the less severe growth disorder variant, hypochondroplasia.

Clinical Trials Companies

Dyne Hopes DM1 And DMD Mid-Stage Data Can Lead To Accelerated Approval

Phase I/II data for its ADC candidates in myotonic dystrophy type 1 and Duchenne muscular dystrophy indicate potential for biomarker-based accelerated approval in both diseases, Dyne asserts.

Business Strategies Clinical Trials

Ipsen’s Sohonos Clinches First US Approval For Ultra-Rare Bone Disorder

Just a month after an EU rejection, the French drugmaker’s oral retinoic acid receptor gamma agonist Sohonos has won a US thumbs up for the treatment of fibrodysplasia ossificans progressiva, an ultra-rare disorder that turns soft tissue into bone.

Approvals Rare Diseases
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Poisoning

Set Alert for Poisoning

SERB Pays $800m For Boston Scientific Specialty Pharma Business

The sale of BTG Specialty Pharmaceuticals to two affiliates of European specialty firm SERB leaves Boston Scientific with BTG’s interventional oncology and vascular products.

Deals M & A

£80m To Tackle Snakebite Is A Wellcome Boost

Two major initiatives have been announced which hope to revolutionize the treatment of snakebites, which claims the lives of up to 138,000 people a year.

Poisoning Research & Development

Indivior Pact Buys Addex Time For Progressing Dipraglurant And Pipeline

Addex Therapeutics says its licensing deal with Indivior to develop and commercialize its investigative addiction therapy ADX71441 'buys time' to find resources for progressing its own pipeline.

Commercial Companies

US Capitol Capsule: Move over Ebola! Fake drugs fight needs global attention, too

Much of the attention at the 2015 World Health Assembly (WHA) in Geneva, Switzerland later this month is expected to be focused on reforming the World Health Organization (WHO) and the failures by the international public health agency in responding to the ongoing Ebola epidemic in West Africa.

Infectious Diseases Cancer
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Renal

Set Alert for Renal

Quick Listen: Scrip's Five Must-Know Things

In this week's podcast edition of Five Must-Know Things: ASCO preview; Asahi Kasei’s US acquisition; Novartis aims for renal disease dominance; paying for gene therapies; and US BIOSECURE Act diluted.

Clinical Trials M & A

Asahi Kasei Furthers Its US, Health Care Growth With $1.1bn Calliditas Buy

Asahi Kasei established a US presence and expertise in renal disease when it bought Veloxis in 2020 and will expand in both areas with Calliditas’s Tarpeyo for immunoglobulin A nephropathy (IgAN).

M & A Renal

Novartis Aims To Dominate Rare Renal Disorders

Fabhalta and atrasentan are odds-on for approval in C3 glomerulopathy and IgA nephropathy, respectively, but commercial success might be harder to come by.

Clinical Trials Renal

First-To-Market Calliditas Picking Up Speed In IgA Nephropathy

A few hiccups aside, Tarpeyo is on track in the US, but major competition from Novartis is just round the corner.

Sales & Earnings Companies
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Wound Healing Tissue Repair 

Set Alert for Wound Healing Tissue Repair

Healios Acquires Bankrupt Athersys, Expands Cell Therapy R&D Programs

Tokyo-based Healios will lead the global clinical studies for Athersys’ cell therapy MultiStem for ARDS and start US Phase II trials for trauma following their long-term collaboration.

Deals M & A

Swiss R&D Innovation: A Tale Of Two Firms Developing Cell Therapies For Wounds

While perhaps best known for historical achievements in immuno-oncology and CNS disorders, the Swiss biotech sector is slowly cultivating a new area of expertise in dermatology. Scrip spoke with two firms applying cell therapy technologies to the challenging area of chronic and acute wounds.

Wound Healing & Tissue Repair Companies

Vertex’s Type 1 Diabetes Cell Therapy Gets A Boost – And Some Competition

Vertex has invested heavily in advancing its stem-cell based candidates for type 1 diabetes, but Sernova has inched ahead in the race to bring a functional cure for the condition to patients.

Metabolic Disorders Wound Healing & Tissue Repair

Krystal Clears Path To Market For Topical Gene Therapy Vyjuvek

The company estimates there are about 9,000 reimbursable patients with dystrophic epidermolysis bullosa worldwide and anticipates a third-quarter launch following FDA approval.

Approvals Gene Therapy
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