NVAX Begins Vaccine Trial In S.Africa, PSTX Slapped With Clinical Hold, UTHR To Face FDA In Apr.

(RTTNews) - Today's Daily Dose brings you news about Applied Therapeutics' ACTION-Galactosemia Kids trial being placed on partial hold by the FDA, Novavax initiating its COVID-19 vaccine trial in South Africa, Sanofi acquiring Principia Biopharma for $100 per share in cash, FDA slapping a clinical hold on Poseida's prostate cancer trial after a patient's death, and Unity Biotechnology's disappointing results from its osteoarthritis trial.

Read on…

1. Applied Therapeutics' ACTION-Galactosemia Kids Study on Partial Hold

Applied Therapeutics Inc.'s (APLT) pediatric phase I/II trial of AT-007 in galactosemia, dubbed ACTION-Galactosemia Kids, has been placed on partial hold by the FDA.

The ACTION-Galactosemia Kids trial is comprised of two parts - a placebo-controlled dose range-finding segment evaluating up to seven days of consecutive dosing to determine the optimal dose in children, followed by a placebo-controlled 90-day study evaluating safety and biomarker efficacy.

Galactosemia is a rare, hereditary disorder of carbohydrate metabolism caused by a deficiency of an enzyme galactose-1-phosphate uridylyl transferase. (Source: NORD).

The company has been asked to provide technical information related to the trial and the clinical hold does not relate to concerns regarding the safety profile of AT-007.

The ACTION-Galactosemia adult trial, which is a phase I/II trial of AT-007 in healthy volunteers and adults with Galactosemia, is ongoing and is not affected by the clinical hold.

APLT closed Monday's trading at $25.71, down 12.07%.

2. Novavax Initiates COVID-19 vaccine Trial in South Africa

Novavax Inc. (NVAX) has initiated a phase IIb efficacy trial of its COVID-19 vaccine candidate NVX-CoV2373 in South Africa. This trial is supported in part by a $15 million grant from the Bill & Melinda Gates Foundation.

NVX-CoV2373 is a stable, prefusion protein made using proprietary nanoparticle technology and includes Novavax's proprietary Matrix-M adjuvant.

In the phase I portion of phase I/II clinical trial, conducted in Australia, NVX-CoV2373 was generally well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera. The phase II portion of this trial is expected to begin this month in the U.S. and Australia.

The company is collaborating with the UK government on the phase III trial of the vaccine, which is scheduled to commence in the UK this quarter.

Novavax has secured $2 billion in funding for its global coronavirus vaccine program, including up to $388 million in funding from the Coalition for Epidemic Preparedness Innovations (CEPI).

NVAX closed Monday's trading at $155.53, up 6.16%.

3. Principia Biopharma to be acquired by Sanofi

Shares of Principia Biopharma Inc. (PRNB) touched an all-time high on Monday, following news of the proposed acquisition of the company by Sanofi.

As per the definitive agreement between the two companies, Sanofi will acquire all of the outstanding shares of Principia for $100 per share in cash, which equates to an aggregate equity value of roughly $3.68 billion.

The deal further strengthens Sanofi's core R&D areas of autoimmune and allergic diseases. The transaction is expected to close in the fourth quarter of 2020. We had alerted PRNB to our premium subscribers on July 8, 2019, when it was trading around $37. (Report titled "More than skin deep...").

PRNB closed Monday's trading at $99.25, up 9.38%.

4. Poseida's Prostate Cancer Trial Placed on Hold after Patient's death

Poseida Therapeutics Inc. (PSTX) plunged more than 34 percent in after-hours trading on Monday, following the FDA's clinical hold on the company's phase I clinical trial for P-PSMA-101 in metastatic castrate-resistant prostate cancer.

The regulatory agency has placed the trial on hold after one of the patients enrolled in the trial died.

According to the company, the patient in question had metastatic castrate-resistant prostate cancer, had failed treatment with multiple anti-cancer agents and was treated with PPSMA-101 in late July. He died of hepatic failure at Day 19 post-treatment.

Although the direct cause of the hepatic failure has not yet been confirmed, the patient developed symptoms consistent with macrophage activation syndrome, often associated with CAR-T therapies, but can have other causes such as infection and autoimmune disease, added the company.

Poseida made its debut on the Nasdaq Global Select Market as recently as July 10, 2020, offering its shares at a price of $16 each.

PSTX closed Monday's trading at $13.00, down 0.76%. In after-hours, the stock fell 34.54% to $8.51.

5. United Therapeutics To Face FDA In April

United Therapeutics Corp.'s (UTHR) supplemental New Drug Application seeking approval for Tyvaso Inhalation Solution for the treatment of pulmonary hypertension associated with interstitial lung disease has been accepted for review by the FDA, with a decision anticipated in April 2021.

Tyvaso is already approved for the treatment of pulmonary arterial hypertension to improve exercise ability.

Interstitial lung disease (ILD) is a group of lung diseases that are characterized by significant scarring or fibrosis of the bronchioles and alveolar sacs within the lungs. Pulmonary hypertension associated with interstitial lung disease, or PH-ILD, is a condition that afflicts roughly 30,000 people in the United States.

Tyvaso, if approved for the expanded indication would be the first and only therapeutic approved for the treatment of PH-ILD.

In the recent second quarter, Tyvaso raked in sales of $119.2 million compared to $109.6 million in the year-ago quarter.

UTHR closed Monday's trading at $110.75, up 1.17%.

6. Unity Biotech Plunges as Osteoarthritis Trial Flops

Unity Biotechnology Inc. (UBX) has reported disappointing 12-week results from its phase II study of UBX0101 in patients with moderate-to-severe painful osteoarthritis of the knee.

In the clinical trial, UBX0101 was tested in dosages of 0.5 mg, 2.0 mg or 4.0 mg and the trial results have revealed that there was no statistically significant difference between any arm of UBX0101 and placebo at the 12-week endpoint for change from baseline in WOMAC-A, an established measurement of pain in osteoarthritis.

Based on the results, Unity Biotech has decided not to advance UBX0101 into pivotal studies but rather focus on its ongoing ophthalmologic and neurologic disease programs.

UBX closed Monday's trading at $4.15, down 66.61%.

7. Stocks That Moved On No News

TFF Pharmaceuticals Inc. (TFFP) closed Monday's trading at $14.06, up 21.73%.

Checkpoint Therapeutics Inc. (CKPT) closed Monday's trading at $2.49, up 21.12%.

Forma Therapeutics Holdings Inc. (FMTX) closed Monday's trading at $39.79, up 20.76%.

ThermoGenesis Holdings Inc. (THMO) closed Monday's trading at $3.66, down 22.62%.

Biofrontera AG (BFRA) closed Monday's trading at $13.86, down 13.86%.