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22 May 2020 22:31

Orphan

Open Orphan raises €13.4m to ramp up Covid-19 study

This was a 3.8% premium to their value on the trading day before Open Orphan announced that its hVIVO operation and diagnostics firm Quotient had struck a major deal to support Covid-19 antibody testing in the UK. "As a result of Covid-19, we are seeing unprecedented growth opportunities as pharma, biotech and governments around the world focus funding on finding solutions to Covid-19 and other respiratory diseases," he said. "As such, we're developing several new revenue streams including by speeding up the development of both a seasonal coronavirus and a Covid-19 virus challenge study model to capitalise upon the Company's inbound demand from Covid-19 vaccine developers globally. The FDA has granted an orphan drug designation to fam-trastuzumab deruxtecan-nxki (Enhertu) for the treatment of patients with gastric cancer, including gastroesophageal junction (GEJ) cancerTrastuzumab deruxtecan, a HER2-directed antibody-drug conjugate, previously demonstrated a statistically significant and clinically meaningful improvement in objective response rate (ORR) and overall survival (OS) versus physician's choice of chemotherapy in patients with HER2-positive metastatic gastric or gastroesophageal cancer in the phase 2 DESTINY-Gastric01 trial.Full findings of the phase 2 study will be presented at the 2020 ASCO Virtual Scientific Program.Based on these data, as well as results from a phase 1 study, the FDA granted the agent a breakthrough therapy designation in May 2020 for the treatment of patients with HER2-positive unresectable or metastatic gastric or GEJ adenocarcinoma who have received 2 or more prior regimens, including trastuzumab (Herceptin).In the open-label, multicenter phase 2 DESTINY-Gastric01 trial, investigtors enrolled 189 Japanese and South Korean patients with HER2-expressing (IHC3+ or IHC2+/ISH+) advanced gastric/GEJ adenocarcinoma with progression on at least 2 prior regimens, including trastuzumab and 5-fluouracil and platinum-based chemotherapy. Patients were randomized 2:1 to receive either trastuzumab deruxtecan at 6.4 mg/kg once every 3 weeks or physician's choice of paclitaxel or irinotecan monotherapy on the same schedule.Beyond the primary end point of ORR, secondary end points included OS, progression-free survival (PFS), duration of response, disease control rate, time to treatment failure, and safety/pharmacokinetics.Moreover, Daiichi Sankyo, which jointly develops trastuzumab deruxtecan with AstraZeneca, reported that safety data showed that the tolerability of trastuzumab deruxtecan was consistent with reports from a phase 1 study.The most common adverse events (AEs; ≥30%) included hematologic and gastrointestinal, including neutrophil count decrease, anemia, nausea and decreased appetite.

No ILD-related deaths occurred in patients with gastric cancer in the phase 1 trial nor in the DESTINY-Gastric01 trial.In the phase 1 trial, which is part of the basis for the breakthrough therapy designation, results showed that trastuzumab deruxtecan induced a confirmed ORR of 43.2% (n = 19) and a disease control rate (DCR) of 79.5% among 44 evaluable patients with HER2-positive gastric/GEJ cancer.3 Among 24 patients who received prior treatment with irinotecan, the ORR and DCR were 41.7% (n = 10) and 79.2%, respectively.Patients received trastuzumab deruxtecan at a dose of 5.4 mg/kg or 6.4 mg/kg. Grade 4 hematologic TEAEs included 2 cases each of grade 4 decreased platelet count, decreased WBC count, and decreased neutrophil count.Drug-related TEAEs led to treatment discontinuation in 5 patients: pneumonitis (n = 3), decreased appetite (n = 1), and decreased platelet count (n = 1).Trastuzumab deruxtecan is currently approved by the FDA for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received 2 or more prior anti–HER2-based regimens in the metastatic setting.Orphan drug designation is designated for agents intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in United States. In the press release, Daiichi Sankyo noted that an estimated 27,600 new cases of gastric cancer will be diagnosed in 2020, and the disease could also lead to more than 11,000 deaths this year.1. Trastuzumab deruxtecan (DS-8201a) in patients with advanced HER2-positive gastric cancer: a dose-expansion, phase 1 study. That was a premium of 3.8% to Open Orphan's closing price of 10.6p on Friday, May 7, being the date immediately before news its subsidiary hVIVO had signed a coronavirus (COVID-19) antibody testing partnership with NASDAQ-listed medical devices firm.

Open Orphan said the cash injection will allow it to ramp up it's antiviral testing to 3,000 a day; expand its laboratory services to meet demand from vaccine developers, and strengthen the balance sheet. In a statement confirming completion of the fundraising, Cathal Friel, executive chairman of Open Orphan, said: "We are delighted by the heavily oversubscribed fundraise which has brought many new leading blue-chip institutional shareholders to our share register. As such, we're developing several new revenue streams including by speeding up the development of both a seasonal coronavirus and a COVID-19 virus challenge study model to capitalise upon the Company's inbound demand from COVID-19 vaccine developers globally." He added: "We have also opened up our laboratory services offering to third party pharma and biotech companies, as in the case of the recently announced Nearmedic contract, and from our state-of-the-art viral laboratory have launched a transformational COVID-19 antibody testing service which, unlike the home and online testing kits, offers 100% accuracy and the potential to complete up to 3,000 tests per day on a single machine. Earlier this week, Open Orphan confirmed that its COVID-19 Antibody Microarray machine is on-site at hVIVO's laboratory in east London and is undergoing testing. Dublin-listed pharma services company Open Orphan has raised up to £12 million (€13.4 million) following a conditional offer for shares by technology platform PrimaryBid. Open Orphan said these challenge study models have the ability to speed up the development of a vaccine by two or three years.

The company said on Friday it was "pleased to announce a conditional offer for subscription via PrimaryBid of new ordinary shares" at an issue price of 11 pence per share. Open Orphan is the result of executive chairman Cathal Friel reversing his pharma services business of the same name into Dublin-listed drug clinical trials manager Venn Life Sciences last year. The FDA has granted an Orphan Drug designation to fam-trastuzumab deruxtecan-nxki (Enhertu) for the treatment of patients with gastric or gastroesophageal junction (GEJ) cancer.1 This decision was based on findings from the phase II DESTINY-Gastric01 clinical trial, in which the HER2-directed antibody-drug conjugate demonstrated statistically significant and clinically meaningful improvements in both the objective response rate (ORR) and overall survival (OS) with the study drug compared with physician's choice of chemotherapy, either irinotecan or paclitaxel. The multicenter, open-label study enrolled patients with advanced gastric/GEJ adenocarcinoma who expressed HER2 (IHC3+ or IHC2+/ISH+) and had progressed on at least 2 prior lines of therapy, including trastuzumab (Herceptin), 5-flourouracil, and platinum-based chemotherapy. Overall, 188 patients from Japan and South Korea were randomized 2:1 to receive either trastuzumab deruxtecan at a dose of 6.4 mg/kg every 3 weeks or the physician's choice of chemotherapy.

The secondary end points included OS, progression-free survival (PFS), duration of response, disease control rate, time to treatment failure, and safety/pharmacokinetics. In the phase I study, which was published in Lancet Oncology, investigators saw preliminary activity and a manageable safety profile with treatment with trastuzumab deruxtecan in heavily pretreated patients with HER2-positive gastric or GEJ cancer. At least 1 grade 3 or greater TEAE was observed in 28 patients (64%), and 48% were determined to be related to the study drug. Trastuzumab deruxtecan also received a Breakthrough Therapy Designation for the treatment of patients with HER2-positive unresectable or metastatic gastric or GEJ adenocarcinoma who received at least 2 prior lines of therapy that included trastuzumab. In Japan, trastuzumab deruxtecan received a Sakigake designation from the Ministry of Health, Labour, and Welfare and a supplemental new drug application was submitted for the potential use of the agent as a treatment for patients with HER2-positive gastric cancer.