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Other Therapy Areas

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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

Anthos Changing Course With Factor XIa Inhibitor, Targeting Atrial Fibrillation Patients First

With a Phase II study stopped for efficacy in bleeding reduction, Anthos hopes to bring abelacimab, a dual Factor XI/XIa inhibitor to market to prevent strokes in a-fib patients and to treat cancer patients with thrombosis.

Business Strategies Clinical Trials

Johnson & Johnson Gets Its Aprocitentan Money Back

The US company has returned rights to the hypertension product to Idorsia, which was already in the grip of a cash crunch and will now face greater difficulties getting the drug to market. 

Deals Blood & Coagulation Disorders

BMS Broadens Market, De-Risks Long-Term Revenue Potential For Reblozyl

As a first-line treatment for anemia in patients with myelodysplastic syndromes, Reblozyl is approved for indications that will account for 75%-80% of the $4bn-plus in sales Bristol has forecast by 2029.

Approvals Launches

US FDA AdComm For Vertex’s Sickle Cell Gene Therapy ‘Not A Concern’

The US biotech revealed the US Food and Drug Administration would be holding an advisory committee meeting for exa-cel during its second quarter earnings, but analysts are unfazed by the news amid robust revenues and raised guidance.

Sales & Earnings Companies

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

All Systems Go At Almirall To Enter Tough Atopic Dermatitis Market With Ebglyss

Following a positive opinion from the CHMP, the Spanish company is hoping to launch its challenger to Dupixent in the EU before the end of the year.

Immune Disorders Business Strategies

Quick Listen: Scrip's Five Must-Know Things

In this week's podcast edition of Five Must-Know Things: Biomarin’s next chapter; Amgen’s obesity plans; Daiichi’s new ADC data; Acelyrin hit by clinical failure; and Skyrizi beats Stelara in Crohn’s.

Commercial Clinical Trials

Acelyrin Goes From Float To Sink

A catastrophic late-stage clinical failure wipes 60% off the group’s value – and boosts a rival. 

Clinical Trials Research & Development

Paradigm Plots Revival Of Butterfly Skin Drug After Amicus Deal

Chiesi-owned Amryt and Krystal Biotech have approved treatments for epidermolysis bullosa but they only treat one subtype - dystrophic - and a single wound. Paradigm CEO Robert Ryan tells Scrip that SD-101, a topical cream he helped develop over 20 years ago, has the potential to be offer a far more extensive and efficacious solution for many more sufferers.   

Deals Rare Diseases
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Ear

Set Alert for Ear

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

Otonomy’s Otividex Fails Again In Phase III Meniere’s Study

Otividex did not significantly reduce vertigo three months after treatment, so Otonomy appears to be moving on from Meniere’s disease and focusing on earlier-stage tinnitus and hearing loss candidates.

Clinical Trials Business Strategies

Fennec Nears Market For Chemo-Induced Hearing Loss in Children

Cisplatin is very effective but two thirds of children treated with the drug for cancer are left with some hearing loss. If approved this year, Fennec's Pedmark could go some way to preventing that distressing side effect.

Ear Drug Review
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Ear Nose Throat

Set Alert for 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

AZ’s Fasenra Joins GSK’s Nucala In Nasal Polyp Pursuit Of Sanofi’s Dupixent

Further competition to Sanofi/Regeneron’s Dupixent in nasal polyps is on the horizon but the newcomers will have some catching up to do.

Companies Clinical Trials

Gastrointestinal

Set Alert for Gastrointestinal

A Year And A Half After Giving Up On TAK-721, Takeda Seeks Approval In EoE

The US FDA rejected TAK-721 for eosinophilic esophagitis (EoE) in 2021, requiring another study, and Takeda ended development in 2022, but in a newly accepted NDA it is seeking a short-term treatment indication.

FDA Inflammation

Quick Listen: Scrip's Five Must-Know Things

In this week's podcast edition of Five Must-Know Things: Biomarin’s next chapter; Amgen’s obesity plans; Daiichi’s new ADC data; Acelyrin hit by clinical failure; and Skyrizi beats Stelara in Crohn’s.

Commercial Clinical Trials

First Wave Rises With Sanofi GI Deal

First Wave is licensing Sanofi’s 5-HT4 receptor partial agonist capeserod and is having to pay a mere $500,000 in upfront fees for the privilege.

Deals Gastrointestinal

AbbVie’s Skyrizi Bests Stelara Head-To-Head In Crohn’s

Data showing superiority on endoscopic remission and non-inferiority on clinical remission could position AbbVie’s IL-23 inhibitor to move ahead of Stelara in treatment paradigm.

Clinical Trials Business Strategies
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Gynecology Urology

Set Alert for Gynecology Urology

Organon Widens Business Development Lens To Maintain Women’s Health Focus

Organon has not strayed from its women’s health path, but is entering into earlier deals and considering technologies beyond therapeutics, while advocating increased investment in the field.

Deals Business Strategies

GSKs Gepotidacin Could Become First Novel Antibiotic For uUTIs In 20 Years

The UK major’s first-in-class asset has matched standard-of-care in two Phase III urinary tract infection trials, setting it up to become the first novel antibiotic for the condition in 20 years with a clear niche in the second-line setting.

Clinical Trials Infectious Diseases

Drug Makers Denounce Court Ruling Overturning Abortion Drug Approval

Industry executives, trade groups and medical organizations said the ruling undermines the US FDA, flies in the face of science and would harm innovation by drug makers if allowed to stand.

Legal Issues FDA

Scrip Asks…What Does 2023 Hold For Biopharma? Part 8: Therapeutic Area Advances

Neuroscience and oncology fuel high hopes for therapeutic R&D progress among 90-plus surveyed biopharma industry leaders.

Scrip Asks Research & Development

Liver Hepatic

Set Alert for Liver Hepatic

The Game Looks Up For Galecto

The Boston-based biotech is weighing up its options as the fall-out from its failed IPF candidate casts a shadow over Galecto’s future.

Restructuring Business Strategies

Biopharma’s Must-Know Q4 Catalysts

Cytokinetics has a lot riding on the upcoming Phase III readout for aficamten, and crunch time is coming for plenty of other groups, too. 

Clinical Trials Companies

Infographic: A Snapshot Of Non-Alcoholic Steatohepatitis R&D

After a multi-year lull, NASH R&D has picked up this past year with key data readouts, several programs advancing toward Phase III and Madrigal's resmetirom under FDA review with a March 2024 action. This graphic provides a look at the overall status of NASH drug R&D.

Business Strategies Drug Approval Standards

A Look Into The Future Of NASH

The liver disease is one of the hottest areas of drug development. Here are some upcoming catalysts to look out for.

Business Strategies Clinical Trials

Ophthalmic

Set Alert for Ophthalmic

Otsuka Expands Ocular Gene Therapy Interests Through ShapeTX Deal

Otsuka steps into the ocular gene therapy field through a technology licensing deal worth up to $1.5bn with ShapeTX, which in turn further expands the US firm's global reach following a $3bn collaboration with Roche. 

Gene Therapy Deals

Outlook Aims To Reverse US FDA’s Rejection Of Bevacizumab For AMD

Outlook Therapeutics received a complete response letter citing manufacturing issues and seeking more data for Lytenava (ONS-5010), its bevacizumab injection for wet age-related macular degeneration.

Complete Response Letters Ophthalmic

Oculis Eyes Success In Wide Range Of Ophthalmic Conditions

Oculis has enjoyed a strong first six months of 2023 after demonstrating the potential of OCS-01 to treat a variety of eye conditions and establishing a presence in the US.

Ophthalmic Business Strategies

Apellis Bounces Back After Needle Discontinuation Amid Syfovre Safety Concerns

Real-world cases of retinal vasculitis linked with Syfovre for geographic atrophy in age-related macular degeneration may have resulted from a material used in a specific filter needle, the US firm revealed, as analysts remained optimistic on the product.

Drug Safety Companies
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Orthopedics

Set Alert for Orthopedics

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

AstraZeneca’s Soliris Wins EU Recommendation In Pediatric gMG As Competition Looms

The UK major’s older C5 inhibitor product could soon receive EU approval for children and adolescents with the rare disease, but emerging competition from biosimilar developers and rival firms are starting to stack up.

Approvals Companies

Argenx Gets Under Skin Of Rivals With Vyvgart Hytrulo Approval

The US approval of a more convenient formulation of its myasthenia gravis therapy, Vyvgart, should cement its market position as well as change the definition of what being "well-controlled means for patients", Argenx hopes.

United States Companies

Inozyme Targets Underserved Mineralization Disorders Market With First-In-Class Enzyme Therapy

Emerging Company Profile: Fresh from a $73m follow-on offering last year, the US biotech is readying its lead asset for a pivotal trial in ultra-rare but debilitating mineralization disorders that affect infants and children.

Emerging Company Profile 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

Jardiance Improves Double-Digit Growth Potential With US Approval In CKD

Boehringer expects Jardiance’s new chronic kidney disease indication to keep its sales growing by double-digit percentages over the coming years, aided by the drug’s ability to reduce hospitalizations.

Approvals Launches

Travere Hopes For FDA Full Approval Despite Filspari Trial Endpoint Miss

The company said the “totality of data” from the confirmatory PROTECT trial in IgA nephropathy showed the drug slowed decline in kidney function.

Clinical Trials FDA

Mironid Gets Roche Backing To Tackle Hereditary Kidney Disease

These are interesting times for Mironid, a Glasgow-based biotech developing small molecule therapeutics for the treatment of autosomal dominant polycystic kidney disease, a life-threatening hereditary condition.

Renal Financing

Calliditas Confident Despite Cutting Tarpeyo Sales Guidance

The Swedish biotech’s immunoglobulin A nephropathy therapy Tarpeyo is still selling well but “market access friction” is slowing growth. As US regulators grant a priority review to its data package for full approval, Calliditas’s prospects look promising.

Sales & Earnings Renal
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Wound Healing Tissue Repair 

Set Alert for Wound Healing Tissue Repair

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

Promore’s Enserpetide Has Phase II Setback In Scar Prevention, Focus Shifts To Ropocamptide

The Swedish firm’s lead program has unexpectedly disappointed in a Phase II scar prevention trial, prompting it to turn its efforts to its only other clinical asset for the treatment of chronic wounds.

Clinical Trials Wound Healing & Tissue Repair
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