Ascendis: FDA finds deficiencies in hypoparathyroidism application

 Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has notified the Company that, as part of their ongoing review, the FDA has identified deficiencies in the Company’s New Drug Application (NDA) for TransCon PTH (palopegteriparatide) in hypoparathyroidism that at this time precludes them from holding further discussions about labeling and post-marketing requirements/commitments. The deficiencies were not disclosed in the letter. The FDA also stated that this does not reflect their final regulatory decision on the Company’s application.

https://finance.yahoo.com/news/ascendis-pharma-provides-regulatory-reviews-110000613.html

Ocuphire corporate update

 Financial Profile Improved Markedly with Global License Agreement to Develop and Commercialize Nyxol for All Three Indications with Cash Runway into 2025

PDUFA Date of September 28, 2023 Set for Nyxol® in its First Indication, Reversal of Pharmacologically-induced Mydriasis; $10 Million Milestone Linked to Approval

Oral APX3330 Achieved Statistical Significance on a Potential Registration Endpoint for Diabetic Retinopathy in ZETA-1 Trial; End-of-Phase 2 FDA Meeting Planned

https://finance.yahoo.com/news/ocuphire-pharma-announces-financial-results-120000345.html

Why Orchestra BioMed has been jumping

 Shares of Orchestra BioMed Holdings (OBIO 26.42%) have been in an uptrend, according to data provided by S&P Global Market Intelligence. The healthcare stock closed at $12.18 last Friday and rose to as high as $17.43 on Tuesday. Orchestra BioMed has a 52-week low of $7.49 and a 52-week high of $19.18.

The medical device technology company went public via a special purpose acquisition company merger (SPAC) on Jan. 26 and is up more than 54% since it began trading. The stock rose a day after the global market intelligence company Tracxn named Orchestra BioMed to its list of top cardiac and vascular disorders start-ups.

The medical device company focuses on partnerships with medical device companies, helping them to develop new products. Its revenue will come from royalties payments, as well as double-digit revenue sharing from its products. It has a strategic partnership with Medtronic to help develop BackBeat Cardiac Neuromodulation Therapy, a potential hypertension treatment for patients with cardiac pacemakers.

The company's other flagship product is its Virtue Sirolimus AngioInfusion Balloon (SAB), which it is partnering with Terumo on. The device is designed to deliver an extended-release formulation of the antirejection drug sirolimus during a balloon angioplasty.

The reason for the excitement regarding Orchestra is both of its main pipeline candidates address large potential patient populations. The company estimates that the annual market for the BackBeat Cardiac Neuromodulation Therapy is more than $2 billion for pacemaker patients with hypertension, and possibly as much as $8 billion if approved for the larger population with serious hypertension. The market for the Virtue SAB is thought to be more than $3 billion.

The company had, as of Dec. 31, $67.8 million in cash, and last year it lost $2.7 million. If its devices win regulatory approval, the stock will likely jump much higher because it will begin to earn royalties from its partners.

https://www.fool.com/investing/2023/03/24/why-shares-of-orchestra-biomed-jumped-this-week/

OncoSec misses endpoint in combo trial

 OncoSec Medical Incorporated (NASDAQ: ONCS) (the Company or OncoSec), a clinical-stage biotechnology company developing intratumoral immunotherapies to stimulate the patient's immune system to target cancer cells and eradicate disease, today announced primary endpoint data from the Phase 2 KEYNOTE-695 clinical trial. This global, open-label single-arm trial is evaluating TAVO™-EP, OncoSec's proprietary interleukin 12 (IL–12) encoding plasmid delivered by intratumoral electroporation, in combination with KEYTRUDA® (pembrolizumab), Merck's anti-PD-1 therapy, in patients with unresectable or metastatic (Stage III/IV) melanoma who had confirmed disease progression after at least 12 weeks exposure to immediate prior anti-PD-1 antibody therapy (pembrolizumab or nivolumab). The last patient started treatment in December 2020; clinical database lock occurred in October 2022. The primary endpoint of overall response rate (ORR) per RECIST v1.1 assessed by blinded independent central review (BICR) was not met.

https://finance.yahoo.com/news/oncosec-announces-clinical-data-keynote-110000460.html

Akebia: Positive Top-Line Results from Vadadustat Alternative Dosing Study

 

• Data demonstrated that vadadustat met the primary and secondary efficacy endpoints and was non-inferior to an ESA as a treatment for anemia due to chronic kidney disease when administered three times a week at the time of dialysis

• Vadadustat demonstrated a similar safety profile to long-acting ESA when used three times a week

https://finance.yahoo.com/news/akebia-therapeutics-announces-positive-top-123000699.html

Alzamend Starts Phase I/IIA Trial for Alzheimer's Dementia Immunotherapy Vaccine

 Alzamend Neuro, Inc. (Nasdaq: ALZN) ("Alzamend"), an early clinical-stage biopharmaceutical company focused on developing novel products for the treatment of Alzheimer’s disease ("Alzheimer’s"), bipolar disorder, major depressive disorder ("MDD") and post-traumatic stress disorder ("PTSD"), today announced the initiation of a phase I/IIA clinical trial for its immunotherapy vaccine (ALZN002) to treat mild to moderate dementia of the Alzheimer’s type. The purpose of this trial is to assess the safety, tolerability, and efficacy of multiple ascending doses of ALZN002 compared with that of placebo in 20-30 subjects with mild to moderate morbidity. The primary goal of this clinical trial is to determine an appropriate dose of ALZN002 for treatment of patients with Alzheimer’s in a larger Phase IIB efficacy and safety clinical trial, which Alzamend expects to initiate within three months of receiving data from the initial trial.

https://finance.yahoo.com/news/alzamend-neuro-announces-initiation-phase-125000747.html

Ensysce: Positive Results from Oral Human Abuse Potential Trial

 Reduced Drug Liking of PF614 versus Oxycodone ~

Ensysce Biosciences, Inc. ("Ensysce" or the "Company") (NASDAQ:ENSC), today announced positive results from a key study on the oral human abuse potential of PF614, a potential next generation pain medicine.

The study met all key endpoints and showed PF614 had significantly lower scores of "Drug Liking" than the oxycodone comparator at both the low and mid doses studied. These results should allow Ensysce to start the next phase of development, by entering into end of phase 2 discussions with the FDA for commencement of phase 3 trials.

On the primary endpoint of "Drug Liking at this moment" PF614 produced statistically lower effects than oxycodone at the lowest dose (p<0.0001), and statistically significant "Overall Drug Liking" at both the low and mid doses (p<0.0001 and p=0.0025, respectively). PF614 also had a significantly longer median time to reach peak levels for "Drug Liking" than oxycodone at all three dose levels, which is important for reducing abuse potential of opioid agents.

The secondary endpoint "Take Drug Again" was met at both the low and mid dose of PF614 (p<0.001 and p=0.0038 respectively) and was numerically lower than comparator even at the high dose, demonstrating that recreational drug users are less motivated to abuse PF614 compared to immediate-release oxycodone.

https://finance.yahoo.com/news/ensysce-biosciences-announces-positive-results-120000287.html