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ARDS Turning Heads, AXSM's Momentum Continues, SVRA Abuzz, Jiankui Jailed

pharma 061917 30dec19 lt

Today's Daily Dose brings you news about Aridis Pharma's collaboration with privately-held Mapp Biopharmaceutical; FDA approval for AZN/MRK's Lynparza for yet another indication; Axsome Therapeutics' continuing momentum; Sol-Gel's acne trial results; update on X4 Pharmaceuticals' Mavorixafor, and jail term for the Chinese scientist He Jiankui.

Read on…

Aridis Pharmaceuticals Inc. (ARDS), which has developed a novel monoclonal antibody discovery, antibody production and yield maximizing technology called APEX, has entered into a collaboration with privately-held Mapp Biopharmaceutical, Inc.

What is APEX?

APEX is a technology suite comprising of a monoclonal antibody (mAb) discovery platform technology that enables screening and single-cell cloning of antibody-producing B-cells using microarrays ('APEX NanoArrays'), a proprietary mAb cell line technology called BREATH CHO that enables gene editing-assisted gene swapping of the candidate antibody genes with endogenous versions to generate a high mAb productivity cell line, and a gene editing-based technology to activate endogenous genes in antibody production cell lines to increase mAb production.

Under this collaboration, Aridis will apply its APEX technology to develop mAb productivity enhanced versions of an undisclosed number of CHO cell lines that were originally developed by MappBio.

H.C. Wainwright analyst Vernon Bernardino has initiated coverage on Aridis Pharma with a "Buy" rating and $7 price target.

ARDS closed Monday's trading at $5.28, up 29.41%.

The FDA has approved AstraZeneca (AZN) and Merck & Co., Inc's (MRK) Lynparza as a 1st-line maintenance treatment of germline BRCA-mutated metastatic pancreatic cancer. The approval follows the recommendation from the US FDA Oncologic Drugs Advisory Committee on December 17th for Lynparza in the above indication.

In clinical trials, Lynparza reduced the risk of disease progression or death by 47% in patients whose disease had not progressed on at least 16 weeks of a 1st-line platinum-based chemotherapy regimen. The drug is now the only approved targeted medicine in biomarker-selected patients with advanced pancreatic cancer.

Lynparza is already approved by the FDA for the treatment of ovarian cancer, fallopian tube cancer, peritoneal cancer, and breast cancer. The drug generated sales of $847 million for AstraZeneca in the first nine months of 2019, representing growth of 93% over the year-ago period.

AZN closed Monday's trading at $50.01, down 0.85%.

Axsome Therapeutics Inc.'s (AXSM) phase III trial of AXS-07 in migraine, dubbed MOMENTUM, has met two regulatory co-primary endpoints of pain freedom and freedom from most bothersome symptom at 2 hours, compared to placebo. The study also met the key secondary endpoint, demonstrating statistically significant superiority to the active comparator rizatriptan on sustained freedom from migraine pain.

The NDA filing of AXS-07 in the acute treatment of migraine is anticipated in the second half of 2020.

Related Reading

Axsome Therapeutics' Awesome Journey From $3 To $100, What Next?

AXSM touched a new all-time high of $109.94 in intraday trading on Monday, before closing at $103.83, up 1.81%.

Savara Inc.'s (SVRA) Molgradex, an investigational inhaled formulation of recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP), has been granted Breakthrough Therapy designation by the FDA.

In a phase III trial, dubbed IMPALA, the results of which were announced in June of the year, Molgradex failed to demonstrate the primary endpoint of the alveolar-arterial oxygen gradient (A-aDO2). However, the study showed statistically significant improvement in the St. George's Respiratory Questionnaire (SGRQ), a key secondary endpoint.

The Company expects to announce data from an open-label follow-up period of IMPALA (weeks 24-48) in Q1 2020.

Molgradex is also under a phase IIa clinical study for the treatment of NTM in non-cystic fibrosis (CF) patients, dubbed OPTIMA. Data from this study is also expected in Q1 2020.

Another drug candidate in the pipeline is AeroVanc, which is under a pivotal phase III clinical study for the treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in cystic fibrosis. Top-line results from the study are anticipated in late 2020 or early 2021.

SVRA closed Monday's trading at $4.91, up 183.82%.

Shares of Sol-Gel Technologies Ltd. (SLGL) soared as much as 125% in intraday trading on Monday, following positive top-line results from two phase III trials of Twyneo for the treatment of acne vulgaris.

The primary endpoints for both trials included the proportion of patients who succeeded in achieving at least a two-grade reduction from baseline and Clear (grade 0) or Almost Clear (grade 1) at Week 12. Both the trials achieved the primary endpoints. Twyneo was also found to be well-tolerated.

Twyneo is an investigational, combination of microencapsulated tretinoin 0.1% and microencapsulated benzoyl peroxide 3% cream.

The Company is planning to submit the NDA for Twyneo in the second half of 2020. If approved, Twyneo has the potential to be the first acne vulgaris treatment to bring together benzoyl peroxide and a potent retinoid, tretinoin, in a once-daily cream.

SLGL closed Monday's trading at $14.60, up 58.18%.

Shares of Wave Life Sciences Ltd. (WVE) plunged nearly 50% on Monday, following topline data from an ongoing Phase 1b/2a PRECISION-HD2 trial evaluating investigational therapy WVE-120102 for Huntington's disease.

In the study, WVE-120102 demonstrated a statistically significant reduction in disease-causing mutant HTT protein in cerebrospinal fluid compared to placebo. However, there was no difference in total HTT protein or neurofilament light chain in treated patients vs. placebo suggesting WVE-120102 may have a potentially differential effect on huntingtin as measured by the mHTT and tHTT assays.

In October 2019, Wave initiated an open-label extension (OLE) study open to patients outside of the U.S. who participated in the Phase 1b/2a PRECISION-HD2 trial and dosing of patients in the OLE is ongoing. Another Phase 1b/2a trial assessing WVE-120101 in early manifest HD patients who carry SNP rs362307 (SNP1), dubbed PRECISION-HD1, is underway, with top-line results expected in the second half of 2020.

WVE closed Monday's trading at $7.99, down 49.46%.

X4 Pharmaceuticals Inc. (XFOR) has initiated a phase Ib clinical trial of Mavorixafor in combination with Imbruvica for the treatment of Waldenstrm's macroglobulinemia, a rare form of non-Hodgkin's lymphoma.

The clinical trial is expected to enroll approximately 12-18 patients, with initial results expected in the second half of 2020.

XFOR closed Monday's trading at $10.79, down 0.64%.

He Jiankui, the Chinese scientist who in November 2018 helped create gene-edited twins by using the gene-editing tool CRISPR-Cas9 to give them HIV immunity, has been sentenced to three years in jail and has been fined 3 million yuan ($430,000) by the Shenzhen Nanshan District People's Court, according to reports.

The twins are said to carry edited CCR5. The gene CCR5 is considered a 'doorway' for HIV to enter and infect immune system cells. The scientist claimed to have disabled the CCR5 gene in the embryos before they were implanted into the mother.

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