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.

As South Korea’s drug ministry decides to rescind approval of Medytox’s botulinum toxin product following manipulation of manufacturing and quality control documents, competition among existing rivals and newcomers is expected to further intensify.

BioMarin maintains its lead in the race to win approval for a hemophilia A gene therapy in the US by resubmitting Roctavian two years after the FDA issued a CRL in response to the original BLA.

The first gene-edited therapy will be submitted for sickle cell disease and transfusion-dependent beta thalassemia; it will compete with bluebird in both indications, but analysts anticipate blockbuster sales in excess of $2bn.

Danicopan seen as potential add-on therapy for paroxysmal nocturnal hemoglobinuria patients getting suboptimal results from Soliris or Ultomiris therapy. AstraZeneca will investigate filing for approval on 12-week data.

The company presented interim data for NTLA-2002 in hereditary angioedema, along with interim data for Regeneron-partnered NTLA-2001 in transthyretin amyloidosis with cardiomyopathy. The company plans a rapid expansion of its programs.

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 this week's podcast edition of Five Must-Know Things: a big US approval for BMS; Roche touts its diverse R&D pipeline; Lumakras’s mixed Phase III results in lung cancer; Japan controversy over a COVID-19 antiviral; and GSK's vaccine strategy in India.

The Swiss major’s Cosentyx has succeeded in two trials for the debilitating skin condition but there are concerns that the data may not be sufficient to shift AbbVie’s Humira off the top spot for treatment.

Chief commercialization officer Chris Boerner said BMS plans to replace Otezla as the oral standard of care for moderate-to-severe psoriasis with Sotyktu (deucravacitinib); its list price is about 40% higher than Amgen’s product.

Arcutis's CEO believes that with "a modest investment," the company can generate proof-of-concept data "against a de-risked target in a high-value indication,” Ducentis's preclinical atopic dermatits drug DS-234.

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.

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.

Astellas adds a large-market drug candidate to its regenerative medicine portfolio, while Frequency Therapeutics almost doubles its fundraising to date. It traded ex-US rights to hearing loss candidate FX-322 for $80m up front and up to $545m in milestone fees plus royalties.

German biotech Acousia Therapeutics closes a series B round with corporate and institutional VC firms, and aims to advance its drugs into clinical trials for hearing loss associated with cancer chemotherapy. 

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. 

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

The company also hopes that positive safety data for pediatric patients could help with discussions with the FDA to make an OTC version of the drug.

Billions of dollars have been wiped off the value of big pharma stocks as the market finally woke up to widescale litigation concerning allegations of increased cancer risk associated with the use of ranitidine. Two companies, GSK and Sanofi, are confident that there is no link. 

A weak yen and strong mainstays helped Takeda in its fiscal first quarter, enabling Japan's largest pharma company to overcome the impact of a big divestment last year, while several new products got off on the right foot in the US.

CEO Pierre Belichard tells Scrip that the French biotech has enough cash and assets to prosper alone but it is also weighing up a reverse merger that would give Enterome a listing on the NASDAQ.

Having surrendered the rights to linzagolix, its GnRH antagonist for bleeding associated with uterine fibroids that the FDA blocked, ObsEva is showing the door to most of its staff in a bid to cut costs and keep in business.

Myovant and partner Pfizer have expected a slow ramp for Myfembree in uterine fibroids and new indication endometriosis as they build awareness and grow the market for the GnRH antagonist class.

The Swiss drug maker will initiate a "mass dismissal" and return rights to its lead drug candidate after the US FDA alerted the company to NDA deficiencies.

A year after Organon spun out of Merck, the company has delivered on its financial guidance, generated double-digit biosimilars sales growth and executed six deals to grow in women’s health.

Obeticholic acid failed to separate from placebo in a Phase III study of non-alcoholic steatohepatitis patients with cirrhosis, but Intercept said it still plans to refile an NDA for pre-cirrhotic NASH by year’s end.

Axcella’s AXA1125, a therapeutic combination of amino acids, shows liver stiffness, liver enzyme and fat reduction benefits in Phase IIb interim look, but the firm lacks the cash to complete the trial.

Focused on rare liver diseases such as acute-on-chronic liver failure following its 2020 disappointment in NASH, Genfit looks to become sector leader in ACLF and other indications such as hepatic encephalopathy.

With Akero’s FGF21 analog showing ability to reduce fibrosis and resolve non-alcoholic steatohepatitis, some analysts think classmate BIO89-100 could yield a better overall profile. NASH data are starting to pour in.

The French firm will use the funds to establish proof-of-concept for two gene therapy programs in a rare eye condition as it strives to become a world leader in ophthalmic genomic medicine, its CEO tells Scrip.

The US firm’s high-dose reformulation of Eylea reduced dosing burden for patients with DME and wet AMD in two pivotal studies and could match Roche’s rival drug.

The company plans to file the complement C5 inhibitor with the US FDA, but Apellis already has a drug pending for the indication.

The company is seeking FDA approval for a first-time ophthalmic formulation of bevacizumab and also refiled its BLA.

Emerging Company Profile: US firm Peptilogics is advancing through the clinic with its lead orthopedic anti-infective drug candidate based on an AI platform that utilizes algorithms to improve peptide design.

Faced with a scarily short cash runway and an ongoing expensive mid-stage study of an allogeneic cell therapy for the treatment of delayed-union bone fractures, Bone Therapeutics has found a saviour in France's Medsenic.

Major Korean firm Yuhan is seeing progress across multiple novel drug candidates licensed out to global partners, with two now entering pivotal trials. An innovative therapy for degenerative disc disease licensed to Spine BioPharma has received an FDA nod for a Phase III program and may change the standard of care for chronic low back pain patients.

The company said the study of JTA-004 failed to meet its primary and key secondary endpoints, though a post-hoc analysis indicated activity in a patient subset.

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.

Terlivaz is the first product approved to improve kidney function in adults with hepatorenal syndrome (HRS). The approval is a milestone in a long regulatory road for the drug.

Phase II data position cemdisiran well for Phase III, but several agents are already well ahead. However, Alnylam foresees a polypharmacy approach.

Amgen is acquiring a pipeline-in-a-pill with ChemoCentryx’s Tavneos, currently approved for ANCA-associated vasculitis, at a time when the company’s existing portfolio is struggling to maintain sales growth.

Roche is not disclosing Phase II results yet, but must exceed late-stage rival drug’s 50% reduction in protein build up to compete in immunoglobulin A nephropathy.

Centaur-CytoTools introduces novel topical treatment for diabetic foot ulcers in India, for which they see limited direct competition and hope to change the way the difficult-to-treat diabetes complication is managed. Phase III studies are ongoing in Europe.

Ghent, Belgium-based start-up, AgomAb Therapeutics NV secures funds and license to develop HGF-mimetic agonistic monoclonal antibodies for regeneration of damaged tissues.

Focused on autologous fibroblast cell therapies for rare diseases, the biotech appears cash-poor despite one candidate moving into Phase II and another ready to enter the clinic.

CEO Crowley points to the need to focus on a pair of Fabry candidates, including the pending NDA for migalastat, and notes that other companies have reached Phase III in EB.