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ALRN To Report Data In Q4, IMUC Hits New High, VYGR Pulls Out Of ABBV Deal, VNDA To Face FDA In Dec.

biotech aug04 lt

Today's Daily Dose brings you news about the progress in Aileron's phase Ib clinical study of ALRN-6924, CymaBay's positive results from ENHANCE study, FDA approval of GW Pharma's Epidiolex for a new indication, Immunic's multiple sclerosis trial results, Rhythm Pharma's anticipated milestones, and the progress in the trial conducted by COVID R&D Alliance.

Read on…

1. Aileron to Report Phase Ib Data of ALRN-6924 in Q4

Aileron Therapeutics (ALRN) has completed enrollment in the dose optimization expansion cohort of its ongoing open-label phase Ib clinical study of ALRN-6924.

In the phase Ib study, ALRN-6924 is being tested as a therapeutic agent administered ahead of chemotherapy to prevent chemotherapy-induced bone marrow toxicities, such as severe anemia, thrombocytopenia, and neutropenia, in patients with p53-mutated small cell lung cancer (SCLC) who are being treated with Topotecan.

The interim data from the dose optimization part of the phase Ib study were reported in June of this year. The results demonstrated that treatment with ALRN-6924 resulted in protective effects against severe chemotherapy-induced anemia and thrombocytopenia in patients.

The final phase 1b dose optimization data, including data from the dose optimization expansion cohort as well as pharmacodynamic biomarker and tumor efficacy data, are expected to be reported in the fourth quarter of this year.

ALRN closed Monday's trading at $0.90, up 3.02%.

2. Seladelpar Trial Data Enhances CymaBay

CymaBay Therapeutics Inc.'s (CBAY) phase III study evaluating Seladelpar for the treatment of primary biliary cholangitis, dubbed ENHANCE, has yielded positive results.

Primary biliary cholangitis (PBC) is a serious and potentially life-threatening autoimmune disease of the liver characterized by impaired bile flow (cholestasis) and accumulation of toxic bile acids. It is associated with fibrosis, cirrhosis, liver failure, and other clinical symptoms such as fatigue and pruritus.

According to the trial results, Seladelpar achieved the primary composite outcome with high statistical significance in 78.2% of patients in the 10 mg group and 57.1% in the 5 mg group compared to 12.5% on placebo after 3 months. There was also a statistically significant improvement in pruritus for patients with moderate-to-severe itch treated with Seladelpar 10 mg compared to placebo.

The encouraging results strongly support the re-initiation of phase III study to confirm the potential of Seladelpar to be a best-in-class treatment for PBC, noted the company.

CBAY closed Monday's trading at $4.88, up 37.46%.

3. GW Pharma's Epidiolex Scores another FDA Nod

GW Pharmaceuticals plc's (GWPH) EPIDIOLEX has won FDA approval for the expanded indication as a treatment for seizures associated with Tuberous Sclerosis Complex.

Tuberous sclerosis complex (TSC) is a rare genetic condition that causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys, and lungs and is a leading cause of genetic epilepsy.

EPIDIOLEX, an oral pharmaceutical formulation of purified cannabidiol, is already approved in the U.S. for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome, two rare and difficult to treat conditions of childhood-onset epilepsy. In Europe, the drug is approved under brand name EPIDYOLEX for the treatment of seizures associated with LGS or Dravet syndrome.

The total net product sales of Epidiolex in the first quarter of 2020 were $116.1 million compared to $33.5 million in the year-ago quarter.

GWPH closed Monday's trading at $132.87, up 4.11%.

4. Immunic Touches New High on EMPhASIS Data

Shares of Immunic Inc. (IMUX) jumped more than 27 percent on Monday, following positive top-line data from its phase II trial of lead asset, IMU-838, in patients with relapsing-remitting multiple sclerosis, dubbed EMPhASIS.

The study achieved all primary and key secondary endpoints, indicating activity in the patients, noted the company.

According to the trial data, at week 24, there was a statistically significant reduction of 62% in combined unique active magnetic resonance imaging lesions in patients receiving 45mg of IMU-838 once daily and 70% reduction for the 30mg once-daily dose compared to placebo.

In other news, the company announced that it intends to offer and sell 5 million shares of its common stock in an underwritten public offering, with the underwriters having an option for 30 days to purchase up to an additional 750,000 shares of its common stock.

Immunic's cash and cash equivalents, as of June 30, 2020, were $48.6 million.

IMUX closed Monday's trading at $22.00, up 27.91%. In after-hours, the stock was down 8.41% at $20.15.

5. Rhythm Pharma on Track

Rhythm Pharmaceuticals Inc. (RYTM) has a couple of catalysts to watch out for in the coming months.

The company's lead asset Setmelanotide, proposed for the treatment of pro-opiomelanocortin (POMC) deficiency obesity and Leptin receptor deficiency obesity, is under priority review by the FDA, with a decision expected on November 27, 2020.

A phase III study evaluating Setmelanotide in Bardet-Biedl syndrome and Alström syndrome is underway, with topline data from the combined pivotal trial anticipated in the fourth quarter of 2020 or early in the first quarter of 2021.

As of June 30, 2020, the company had cash, cash equivalents, and short-term investments of $228.6 million.

RYTM closed Monday's trading at $20.20, up 5.10%.

6. I-SPY COVID Trial Moves Ahead

A clinical trial that is designed to evaluate the efficacy of Cenicriviroc, Otezla and Firazyr in severely ill, hospitalized COVID-19 patients who require high-flow oxygen, dubbed I-SPY COVID trial, has dosed the first patient.

The I-SPY COVID trial is being conducted by members of the COVID R&D Alliance namely AbbVie Inc. (ABBV), Amgen Inc. (AMGN), and Takeda Pharmaceutical Co. Ltd. (TAK).

While Cenicriviroc is an investigational drug being studied as a treatment for NASH, Otezla is an approved drug for the treatment of psoriatic arthritis, plaque psoriasis, and oral ulcers associated with Behçet's Disease, and Firazyr is also an approved drug indicated for the treatment of acute attacks of hereditary angioedema.

The I-SPY COVID Trial is based on Quantum Leap Healthcare Collaborative's adaptive platform trial design, which is intended to increase trial efficiency by minimizing the number of participants and time required to evaluate potential treatments.

TAK closed Monday's trading at $18.52, up 1.76%.

7. Voyager Cuts Short Journey with Abbvie

Voyager Therapeutics Inc. (VYGR) has terminated its tau and alpha-synuclein vectorized antibody collaborations with AbbVie (ABBV) that were formed in 2018 and 2019, respectively.

The collaborations were formed to develop potential new treatments for Alzheimer's disease and other tau-related neurodegenerative diseases as well as treatments for Parkinson's disease and other synucleinopathies.

Voyager had received upfront payments to perform research and preclinical development of vectorized antibodies directed against tau and alpha-synuclein as part of the agreements. Now that the deals have been terminated, Voyager has regained full clinical development and commercialization rights to certain product candidates developed within the context of the collaboration for the tau program.

VYGR closed Monday's trading at $11.33, up 2.35%. In after-hours, the stock was down 11.74% at $10.00.

8. Vanda Pharma to Face FDA on Dec.1

Vanda Pharmaceuticals Inc. (VNDA) has sought FDA approval for its marketed non-24-hour sleep-wake disorder drug Hetlioz in the indication of Smith-Magenis Syndrome.

Smith-Magenis Syndrome (SMS) is a developmental disorder and patients present with a number of physical, mental, and behavioral problems. The most common symptom of SMS is a severe sleep disorder associated with significant disruption in the lives of patients and their families.

The FDA decision on Hetlioz for the expanded indication is expected on December 1, 2020.

VNDA closed Monday's trading at $10.57, up 4.86%.

9. Stocks That Moved On No News

Cocrystal Pharma Inc. (COCP) closed Monday's trading at $2.36, up 39.64%.

Liminal BioSciences Inc. (LMNL) closed Monday's trading at $21.29, up 30.53%.

Trevena Inc. (TRVN) closed Monday's trading at $2.94, up 26.72%.

Achieve Life Sciences Inc. (ACHV) closed Monday's trading at $11.72, down 14.01%.

Sonnet BioTherapeutics Holdings, Inc. (SONN) closed Monday's trading at $3.54, down 13.87%.

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