FDA Grants Orphan Drug Designation to Coherus Biosciences’ Toripalimab for Small Cell Lung Cancer

The FDA granted orphan drug status to Coherus Biosciences’ Toripalimab (TuoYi) based on data from the phase 3 JUPITER-08 study in patients with extensive-stage Small-cell Lung Cancer (ES-SCLC). Patients in the current randomized, double-blind trial are getting toripalimab, a PD-1 inhibitor, plus a platinum-based chemotherapeutic such as cisplatin or carboplatin, and etoposide as first-line therapy for ES-SCLC, as opposed to placebo plus the same chemotherapy backbone. The trial is estimated to enroll 420 patients who will get a toripalimab or placebo injection paired with chemotherapy at 240 mg/6mL per vial every 3 weeks for up to 2 years of treatment.

The primary endpoints are progression-free survival (PFS) as determined by the investigator and overall survival. PFS by RECIST 1.1 criteria as judged by a blinded independent review board, objective response rate, duration of response, and disease control rate are the major secondary end goals.

Eligibility criteria included being 18 years or older, having histologically confirmed ES-SCLC, and have not previously received treatment or immune checkpoint inhibitors for their condition. In addition, patients had to remain treatment-free for at least 6 months after their last round of chemotherapy or radiotherapy. Asymptomatic brain metastases that had been previously treated were permitted. Patients were ruled out of the trial if they had previously had systemic treatment for ES-SCLC, had previously received any CD137 agonist or immune checkpoint inhibitor, or had current or untreated central nervous system metastases.

The FDA has also granted priority review for toripalimab’s biologics license application in recurrent or metastatic Nasopharyngeal Cancer. The prescription drug user fee act is scheduled to go into effect in April 2022.

Furthermore, toripalimab was the first PD-L1 monoclonal antibody approved in China in four indications: unresectable or metastatic melanoma after the failure of standard systemic therapy; recurrent or metastatic nasopharyngeal carcinoma after failure of at least two lines of prior therapy; locally advanced urothelial carcinoma after failure of platinum-containing chemotherapy or progression within 12 months of neoadjuvant platinum-containing therapy; and in combination with cisplatin plus gemcitabine as a first-line treatment for patients with locally recurrent or metastatic nasopharyngeal carcinoma.

FDA Grants Fast Track Designation to Keymed’s CMG901

The FDA has granted Keymed’s CMG901 fast track designation for the unresectable or metastatic Gastric and Gastroesophageal Junction Cancer treatment in patients who have relapsed or are resistant to approved therapies.

CMG901 is a Claudin 18.2 inhibitor and it functions by attaching to cells that express Claudin 18.2. Tumor cells subsequently transport CMG901 to the lysosome, where the cytotoxic payload is released. This procedure stops the tumor cells’ cell cycles and apoptosis. CMG901 has also been demonstrated to promote cellular and soluble immune effectors, limiting the activation of antibody-dependent cellular cytotoxicity and complement-dependent cytotoxicity and therefore killing Claudin 18.2-positive cells.

In preclinical trials, the Claudin 18.2-targeted treatment was more effective at killing gastric cancer cells than the zolbetuximab analog or CMG901 unconjugated antibody. In addition, the drug was well-tolerated in the stomach cancer model and had a favorable safety profile.

An open-label, phase 1 dose-escalation, and dose-expansion research is now underway to evaluate CMG901’s safety, tolerability, and preliminary efficacy in patients with advanced, unresectable, or metastatic solid tumors. In Part A, 162 patients will be enrolled in two portions of the trial to look at the key endpoints of the number of patients with dose-limiting toxicity and the incidence, severity, and outcome of treatment-emergent adverse events (TEAEs) and serious AEs. The primary endpoint in Part B will be the objective response rate as measured by RECIST v1.1 and calculating the appropriate phase 2 dose of CMG901. So far, CMG90 is the only drug in its class to have received fast-track status.

Biogen Pulls EU Application for Alzheimer’s Drug Aduhelm

In one of the latest regulatory updates, Biogen has withdrawn its European application for its Alzheimer’s drug Aduhelm. Biogen stated that it reached the decision following interactions with the European Medicines Agency which suggested that the data provided so far would not be enough to support the approval and marketing authorization of Aduhelm (aducanumab) in the US.

In 2020, Biogen filled the application with the EMA to recommend the drug application for approval in Europe. However, earlier in December 2021, the EMA had raised concerns about the safety of the drug and rejected its application. The Committee for Medicinal Products for Human Use (CHMP) stated that the drug is able to reduce levels of beta-amyloid in the brain, however, it is unable to support the link between amyloid reduction and patient improvement, along with the brain swelling and bleeding risks associated with the drug. 

Aducanumab, also known as Aduhelm is surrounded by controversy since its accelerated approval in the US. The US Centres for Medicare and Medicaid Services (CMS) through a draft decision has recommended a restrictive coverage plan for the drug which had limited the use of Aduhelm to patients enrolled in clinical trials.

Vicore Initiates Digital Therapeutics Study for Anxiety in IPF Patients

Curebase has announced the initiation of the pilot phase of COMPANION, the first clinical investigation of a new digital therapeutics (DTx) for patients with Idiopathic Pulmonary Fibrosis (IPF). Vicore Pharma is a clinical-stage pharmaceutical company that owned the DTx and is a prominent player in the respiratory disease therapeutics segment. Last year, Vicore entered into collaboration with Alex to develop a DTx for patients with IPF. Curebase’s DCT platform and services will enable Vicore to recruit patients for the COMPANION clinical study offering the potential to expand patient recruitment to more diverse and non-mobile patient populations. 

The COMPANION study is a randomized, controlled, parallel-group clinical investigation assessing the impact of Vicore’s digital cognitive behavioral therapy on psychological symptom burden, specifically anxiety and depressive symptoms, in adults diagnosed with idiopathic pulmonary fibrosis (dCBT IPF). 

IPF is a rare, chronic, and progressive fibrosing interstitial pneumonia. People from middle-aged and older adults are found to be more affected by IPF. It impacts the lung tissue (alveoli in particular) by either thickening, stiffening, or persistent and progressive scarring (fibrosis) which grows irreversibly over time. The COMPANION study will take place in the US and is designed to minimize the burden on IPF patients, conducted using Curebase’s decentralized and virtual clinical research solutions. As per DelveInsight, in 2021, the total diagnosed prevalent cases of Idiopathic Pulmonary Fibrosis in the United States was found to be approximately 73,041, which is expected to increase by 2032.

FDA Approves New Acne Treatment Winlevi for the First Time in 40 Years

It’s been decades since a novel treatment of acne hit the scene, but that is set to change with Cassiopea’s approval for Winlevi.  

The FDA on Thursday approved the new prescription of acne medication, set for use in people of age 12 years and older.

Approximately 50 million people are affected by acne in the United States. Still, there hasn’t been a new mechanism of action in the field in nearly 40 years, Cassiopea CEO Diana Harbort said ahead of the approval. Winlevi is a “first-in-class topical treatment” to treat acne at the “hormonal level for both males and females,” she added. 

Winlevi is a topical cream that targets hormonal acne by blocking androgen receptors. Androgens are a family of hormones that include testosterone and are responsible for signaling to your skin to produce sebum by binding to androgen receptors. By blocking these receptor sites, Winlevi reduces sebum production and helps to reduce acne caused by hormone imbalances.

The company expects dermatologists will use Winlevi in combination with existing treatments to address multiple facets of the condition, such as clogged hair follicles and inflammation. Doctors have indicated they’re enthusiastic about the new option, Harbort said, and the company expects “widespread acceptance and use.” 

They’ll still have to wait a while; Cassiopea isn’t expecting to launch its new medication until next March. In the meantime, the company is meeting with payers and incorporating lessons from other companies that have been established during the pandemic to prep for its rollout. 

When Cassiopea launches Winlevi, the new drug will face off against branded options from Galderma, Bausch & Lomb, and Menlo. 

Samsung Biologics Completes Acquisition of Samsung Bioepis

Samsung Biologics announced today that it had completed the acquisition of Biogen’s stake for USD 2.3 billion. With the accomplishment of the first payment of USD 1.0 billion under the terms of the acquisition agreement, Samsung Biologics has fully captured Samsung Bioepis as a wholly-owned subsidiary. Over two years, the remaining USD 1.3 billion will be made in installments.

“Today’s announcement marks a remarkable milestone for Samsung Biologics in our continued venture into the biosimilar business and escalating the biosimilar growth,” said John Rim, CEO and President of Samsung Biologics. “

The acquisition was funded by a portion of a paid-in capital increase of KRW 3 trillion raised by issuing new shares, which will be used to support the company’s strategic growth plans. The proposal deal gives Samsung Bioepis improved autonomy and agility in business operations, biosimilar development capabilities, accelerating sales growth, operating margin improvements, and future performance in the development of novel therapies.

Samsung Biologics will continue to serve on its commitment to clients & shareholders by providing quality-driven CDMO biomanufacturing services.

Roche’s Breast Cancer Drug Fails in Phase 2 Trial

In another blow to the developing oral Selective Estrogen Receptor Degrader (SERD) class, Swiss-based Roche announced in the release of its first-quarter results, that Giredestrant failed the phase 2 acelERA study in Advanced Breast Cancer. As a second or third-line treatment, 303 patients with estrogen receptor-positive, human epidermal growth factor receptor 2-negative locally advanced or metastatic Breast Cancer was randomly assigned to receive Giredestrant or the physician’s choice of AstraZeneca’s Faslodex or an aromatase inhibitor. In each 28-day cycle, participants in the Giredestrant cohort took the oral SERD once a day. Giredestrant failed to exceed the physician’s choice of endocrine monotherapy in terms of progression-free survival, causing the trial to miss its primary aim.

In the battle to bring an oral SERD to market, Roche is trailing Radius’ Elacestrant, but while the front runner hit the mark in a key trial in October, a closer look into the data in December led its stock to tank. Sanofi’s Amcenestrant is also in competition, although it failed a critical study last month. Sanofi, like Roche, pitted its oral SERD against a variety of drugs, including Faslodex, as a second-line or later treatment of ER+/HER2- advanced or metastatic breast cancer. In the aftermath of data that devastated Radius Health’s shares and Sanofi’s prospects, the failure of Roche’s Giredestrant is generating concerns about the drug ahead of more data releases.

The Giredestrant failure comes only weeks after a long shot on anti-TIGIT drug Tiragolumab failed, continuing Roche’s pipeline’s poor start in 2022. Going into the year, the company listed the Giredestrant study on a shortlist of key anticipated investigational drug data drops, along with four studies of Tiragolumab and a study of Gantenerumab in Alzheimer’s disease. After going 0 for 2, Roche is now counting on three trials of Tiragolumab and a study of Gantenerumab to save its late-phase pipeline. 

Ashai Kasei Medical Acquires Bionova Scientific

In a strategic complementary addition, Binova Scientific, a US biologics CDMO (contract development and manufacturing organization), will be acquired by the US branch of Asahi Kasei Medical, which is owned by Tokyo-based Asahi Kasei. The deal was signed on April 14, 2022 (JST) through a subsidiary in the United States. Bionova Scientific, LLC is a biopharmaceutical company that provides contract process development and GMP-compliant contract manufacturing services to biopharmaceutical companies, particularly those developing next-generation antibody-based drugs.

Asahi Kasei Medical, on the other hand, manufactures and sells Planova filters and bioprocess equipment, and has been expanding rapidly its biosafety contract testing business, with the acquisition of Austrian viral safety testing services provider Virusure Forschung and Entwicklung GmbH in 2019 and US-based mycoplasma testing services provider Bionique Testing Laboratories LLC in 2021.

With the acquisition of Bionova Scientific, Asahi Kasei Medical will be able to add a biopharmaceutical CDMO to its bioprocess business. Since process development and GMP manufacturing operations are critical to the overall success of biopharmaceutical customers’ businesses and the launch of new drugs, Asahi Kasei Medical will be able to grow its bioprocess business rapidly by better serving the industry and reaching a broader range of customers, including those at the cutting edge of next-generation biopharmaceuticals.