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.

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.

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.

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.

The drug maker said the program would allow for continued reimbursement when patients change insurance plans. A Sangamo-partnered gene therapy for hemophilia A is also in the works.

The company announced positive topline results from a Phase II study of mezagitamab and expects to have five programs in Phase III development this fiscal year.

Gilead said the FDA requested a partial clinical hold on the studies as well, just over a week after the drug maker said it would not develop the magrolimab further for blood cancers.

The Phase III trial of BT524 produced positive topline results in patients with acquired fibrinogen deficiency, potentially making it the first fibrinogen concentrate to win US FDA approval for that indication.

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.

In a head-to-head study of Rinvoq versus Dupixent in atopic dermatitis, the AbbVie drug showed superiority on a composite endpoint measuring skin clearance and itch resolution.

Incyte will pay $750m to acquire privately held Escient and its first-in-class oral antagonists of Mas-related G protein-coupled receptors X2 and X4.

Bristol Myers Squibb’s head of major markets, Monica Shaw, wants to improve patients’ access to lifesaving therapies. And the group has several new products ready to roll.

The third Paragon spinout, going public in a reverse-merger with ARCA, announced a concurrent $275m financing to help it develop optimized biologics as potential standards of care in psoriasis and psoriatic arthritis.

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.

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.

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.

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.

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

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.

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.

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

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

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.

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.

Looking to compete with Takeda’s Gattex in short bowel syndrome with intestinal failure, Ironwood succeeds in a Phase III trial, but investors may be concerned with misses on secondary endpoints.

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.

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

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.

Emerging Company Profile: Armed with a new tranche of funding, the Danish group is aiming to improve the lot of infertile couples by altering the microbiome of patients with dysbiosis.

UK-based Ochre Bio has signed its first major deal with Boehringer Ingelheim. Scrip talked to its co-founder and CEO, Jack O’Meara, about its human tissue-based drug discovery platform, its resulting RNA platform for liver disease and how the fledgling drug company's early work mirrored that seen in diagnostics.

Cash-limited Organovo has an FXR agonist showing competitive efficacy and tolerability in MASH, but may need a partner to move forward in that disease as it may be prioritizing its IBD program.

Still a clinical-stage company, Viking is studying promising candidates for both obesity and MASH in Phase II, but may need to prioritize one while partnering the other.

Madrigal faces the challenge of opening the metabolic-associated steatohepatitis (MASH, also known as NASH) market, but the firm has been laying groundwork for over a year.

The Swiss major brings a new ruthlessness to the compounds in its pipeline: if they’re not first or best, they’re out.

In this week's podcast edition of Five Must-Know Things: Genmab buys into ADCs; Roivant set to take on AbbVie in uveitis; Ipsen signs firs ADC pact; South Korea looks to build biotech momentum; and the end of the road for Acorda.

LENZ announced successful results from its Phase III pivotal trial of LNZ100 and plans to apply for US FDA approval in the middle of this year, with commercialization in Q2 2025.

Roivant’s brepocitinib bests data Humira previously posted in non-infectious uveitis, a top cause of blindness. The TYK2/JAK1 inhibitor also is in Phase III for dermatomyositis.

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.

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.

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.

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.

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.

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.

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.

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.

Another data drop from Fabhalta’s IgAN trial looks encouraging, despite a missing abstract.

The company hopes for a “Humira-like” blockbuster with povetacicept, the object of the sector’s biggest M&A deal so far this year.

The Boston, MA biotech is about to enter the clinic with its autosomal dominant polycystic kidney disease candidate but microRNA therapy pioneer Regulus is already setting the pace.

So far, Novartis has only reported that its Phase III trial has succeeded and investors face a wait to find out just how good the drug is.

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.

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.

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.

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