New Medicines #33
Tags: Venture Capital, Biotechnology, Investing, Healthcare, Medicine

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New medicines #33 — December 19, 2020 — December 24, 2020

A weekly overview of the development of new medicines.

- Alnylam Pharmaceuticals with Novartis (NYSE: NVS) announced the FDA issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for inclisiran, a potential treatment for hyperlipidemia in adults who have elevated low-density lipoprotein cholesterol (LDL-C):

The cited unresolved facility inspection-related conditions for the CRL

The conditions will be conveyed to the European manufacturing facility within 10 business days

- Altimmune announced the FDA issued a clinical hold on its Investigational New Drug (IND) application for AdCOVID, a single-dose intranasal COVID-19 vaccine candidate:

Protocol modifications and the submission of additional Chemistry, Manufacturing and Controls (CMC) data were requested by the FDA

The company noted that it does not anticipate a significant impact on the overall clinical development timeline

- Amgen with AstraZeneca announced their phase 3 SOURCE trial did not meet the primary endpoint of a statistically significant reduction in the daily oral corticosteroid (OCS) dose, without loss of asthma control, with tezepelumab compared to placebo.

- ChemoCentryx released data from its phase 2 trial of avacopan in C3 Glomerulopathy:

While the trial saw a significant improvement in renal function, the primary endpoint (change from baseline in the C3G Histologic Index for Disease Activity) was not met

- Karyopharm Therapeutics announced that the FDA approved Xpovio (selinexor) in combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.

- Myovant Sciences received approval from the FDA for its Relugolix monotherapy tablet for the treatment of adult patients with advanced prostate cancer.

- Soligeni announced that top-line data from its phase 3 trial evaluating SGX942 (dusquetide) in the treatment of severe oral mucositis (SOM) in patients with head and neck cancer (HNC) did not meet the primary endpoint.

- Spectrum Pharmaceuticals announced that data from Cohort 3 of its phase 2 trial evaluating poziotinib with non-small cell lung cancer (NSCLC) with HER2 exon 20 insertion mutations, did not meet the primary endpoint:

The company still intends to file a New Drug Application in 2021 based on data from Cohort 2 of the trial which met the primary endpoint earlier this year

The company also announced late last year that data from Cohort 1 did not meet the primary endpoint

- Supernus Pharmaceuticals announced that its phase 3 trial of SPN-812 in adults (P306) for the treatment of attention deficit hyperactivity disorder (ADHD) met the primary endpoint (p=0.0040) compared to placebo in improving the symptoms of ADHD.

- uniQure announced that their hemophilia B gene therapy program, including its phase 3 HOPE-B trial, has been placed on clinical hold by the FDA:

Patient dosing is complete in each of uniQure’s three hemophilia B gene therapy studies, and there is no plan to enroll or treat additional patients

- Urovant Sciences announced the FDA approved the New Drug Application (NDA) for once-daily 75 mg Gemtesa (vibegron) for the treatment of overactive bladder (OAB) with symptoms of urge urinary incontinence (UUI), urgency, and urinary frequency in adults.

- Voyager Therapeutics announced that the FDA placed a clinical hold on the phase 2 RESTORE-1 clinical trial of NBIb-1817 (VY-AADC).

Source: BPC (reformatted), SY, Bioworld