Axial — New Medicines #31. Get these analyses to your inbox —…
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New medicines #31 — December 5, 2020 — December 11, 2020

A weekly overview of the development of new medicines.

- Bellicum Pharmaceuticals announced that a clinical hold has been placed on patient enrollment and dosing in its phase 1/2 trial evaluating BPX-601 in patients with previously treated metastatic pancreatic or prostate cancer.

- BioCryst Pharmaceuticals announced that the FDA approved oral, once-daily Orladeyo (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

- BioNTech with Pfizer earned Emergency Use Authorization (EUA) approval by the FDA for the COVID-19 vaccine for use in individuals 16 and older.

- Curis announced promising preliminary data from its ongoing open-label, single arm phase 1 trial of CA-4948 in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS):

Marrow blast reductions were observed in all evaluable patients (6 patients)

Two patients experienced a marrow complete response

- Fate Therapeutics announced interim data from its phase 1 dose escalation trial of FT516 in combination with rituximab for patients with relapsed / refractory B-cell lymphoma:

Three of four patients achieved an objective response, including two complete responses (CR)

- Gamida Cell announced updated data from its phase 1 trial of GDA-201, a natural killer (NK) cell-based cancer immunotherapy for the treatment of patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM):

Of the 19 patients with NHL, 13 complete responses and one partial response were observed, with an overall response rate of 74% and a complete response rate of 68%

- Immutep released overall survival (OS) data from its phase 2b AIPAC trial of eftilagimod alpha in combination with paclitaxel chemotherapy (efti group) in comparison to a combination of placebo and paclitaxel chemotherapy in patients with HER2-negative/HR positive metastatic breast cancer (HR+ MBC):

In the total patient population, first OS data (based on approx. 60% of events) show that patients in the efti group had an improving OS trend with a median OS of 20.2 months compared to 17.5 months for patients in the comparator group, indicating a survival benefit of +2.7 months (HR = 0.83; p = 0.14). The OS analysis is based on a 2-sided false positive probability of 0.05

- Lipocine announced tentative FDA approval of Tlando (testosterone undecanoate), an oral testosterone replacement therapy (TRT).

- Lyra Therapeutics announced that its phase 2 LANTERN trial of LYR-210 for chronic rhinosinusitis (CRS) did not meet the primary endpoint.

- Oncternal Therapeutics announced updated interim clinical data from the ongoing phase 1/2 CIRLL (Cirmtuzumab and Ibrutinib targeting ROR1 for Leukemia and Lymphoma) clinical trial, in patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL):

The company noted a best objective response rate of 87% reported for 15 patients with relapsed/refractory mantle cell lymphoma, with a median follow-up of 12.1 months

- Pluristem Therapeutics announced that the company will be discontinuing its phase 3 trial for the treatment of critical limb ischemia (CLI) following interim analysis from an independent Data Monitoring Committee (DMC) that the trial is unlikely to meet the primary endpoint by the time of the final analysis.

- Rocket Pharmaceuticals released promising preliminary data from its open-label, Phase 1 trial of RP-A501 for the treatment of Danon Disease:

Cardiac LAMP2B protein expression by immunohistochemical staining was greater than 50% of normal LAMP2B in two patients with follow-up data of up to one year; these levels far exceeded the threshold estimated in females who largely do not develop heart failure until several decades later than males

In addition, RP-A501 demonstrated reduction of myocardial cell disarray and accumulation of autophagic vacuoles, a hallmark of Danon Disease

The company also mentioned that upon dose escalation one of the two treated patients experienced a non-persistent, immune-related event that was classified as a drug product related serious adverse event

- Savara Pharmaceuticals announced its phase 3 trial of AeroVanc (vancomycin hydrochloride inhalation powder) in people living with cystic fibrosis (CF) who have Methicillin-resistant Staphylococcus aureus (MRSA) lung infection did not meet the primary endpoint.

- Sutro Biopharma announced interim data from its phase 1 trial of STRO-002 in patients with ovarian cancer:

Of the 31 evaluable patients, 1 patient achieved a complete response (CR) and 9 patients achieved a partial response (PR)

Twenty-three patients (74%) achieved disease control at 12 weeks, while 18 patients (58%) achieved disease control at 16 weeks

- TG Therapeutics announced that the company met the primary endpoints in both of its two phase 3 trials (ULTIMATE 1 & 2), evaluating ublituximab, compared to teriflunomide in patients with relapsing forms of multiple sclerosis (RMS):

Ublituximab treatment resulted in an annualized relapse rate (ARR) of <0.10 in each of ULTIMATE 1 & 2, with a relative reduction in ARR of approximately 60% and 50%, respectively, over teriflunomide

- Tonix Pharmaceuticals announced that its phase 3 trial of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.01) in participants with fibromyalgia.

Source: BPC (reformatted), SY, Bioworld