Corcept starts tender for 7.5M shares

 Corcept Therapeutics Incorporated (Nasdaq: CORT) (“Corcept”), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrine, oncologic, metabolic and neurological disorders by modulating the effects of the hormone cortisol, today announced that it has commenced a modified Dutch Auction tender offer for the purchase of up to 7,500,000 shares of Corcept’s common stock, par value $0.001 per share (each, a “Share,” and collectively, “Shares”) or such lesser number of Shares as are properly tendered and not properly withdrawn, at a price not greater than $22.00 per Share nor less than $19.25 per Share, to be paid to the seller in cash less any applicable withholding taxes. The tender offer is made in accordance with the terms and subject to the conditions described in the offer to purchase, the related letter of transmittal and other related materials, as each may be amended or supplemented from time to time.

The closing price of the Shares on The Nasdaq Stock Market on March 3, 2023, the last full trading day before the start of the tender offer, was $19.20 per Share. The tender offer is scheduled to expire one minute after 11:59 P.M., New York City Time, March 31, 2023, unless the offer is extended or terminated.

Corcept believes that the repurchase of Shares pursuant to the tender offer is consistent with its long-term goal of maximizing stockholder value and that the tender offer is an efficient way to give stockholders the opportunity to receive a return of their investment by tendering some or all of their Shares.

The tender offer is contingent upon at least 3,000,000 Shares being tendered. The tender offer is also subject to terms and conditions, which are described in detail in the offer to purchase. Specific instructions and a complete explanation of the terms and conditions of the tender offer are contained in the offer to purchase, the related letter of transmittal and other related materials, which will be mailed to stockholders of record promptly.

https://finance.yahoo.com/news/corcept-therapeutics-announces-commencement-tender-130000055.html

90% Survival Improvement in High-Risk Pulmonary Embolism with Inari Medical FlowTriever

 Inari Medical, Inc. (NASDAQ: NARI) (“Inari”) a medical device company with a mission to treat and transform the lives of patients suffering from venous and other diseases, announced positive results from the FLAME study in high-risk/massive pulmonary embolism (“PE”). The data was presented during the Late-Breaking Clinical and Investigative Horizons session at the 2023 ACC (American College of Cardiology) conference on March 5th by national Principal Investigator (PI) Mitchell Silver, DO, FACC, FVSM, RPVI, an interventional cardiologist and vascular medicine specialist at OhioHealth Riverside Methodist Hospital.

FLAME is the largest prospective study of interventional treatment in high-risk PE, a patient population with a historical mortality rate of 25-50%. The study collected data on patients treated with FlowTriever and on those treated with other therapies in a context arm. The study was stopped early due to overwhelmingly positive outcomes in the FlowTriever patients.

The primary endpoint measured a composite of meaningful in-hospital clinical outcomes, including mortality, major bleeding, clinical deterioration, and escalation to an alternate therapy. The endpoint was met in the FlowTriever arm, driven predominantly by a low mortality rate of 1.9%. This represented a 90% reduction compared to the 29.5% mortality rate seen in patients treated with other therapies in the context arm.

https://finance.yahoo.com/news/flame-study-demonstrates-90-survival-130000492.html

BioVie: More Positive Findings from Phase 2 Parkinson’s Trial

 BioVie Inc., (NASDAQ: BIVI) (“BioVie” or the “Company”) a clinical-stage company developing innovative drug therapies for the treatment of neurological and neurodegenerative disorders and advanced liver disease, today announced additional preliminary findings from its Parkinson’s Disease (PD) Phase 2 trial. The preliminary findings show that significantly more patients treated with the Company’s drug, NE3107, were assessed as being in the “ON” state in the morning after withholding their usual standard of care (SOC) for at least 8 hours before taking their usual morning Parkinson’s medications, compared to patients on SOC alone plus placebo (p<0.02).

Many PD patients complain of having rigid muscles and difficulties getting out of bed in the morning, which is referred to as the muscles being in an “off state.” Conversely, patients are considered to be in the “on state” if they retain sufficient muscle control. The “on state” is clinically meaningful for PD patients because it is the time when their motor symptoms are most optimally controlled, allowing for better movement and stability.

https://finance.yahoo.com/news/biovie-announces-additional-findings-phase-130000852.html

Ikena urothelial cancer candidate fast tracked

 Ikena Oncology, Inc. (Nasdaq: IKNA, “Ikena”), a targeted oncology company forging new territory in patient-directed cancer treatment, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for IK-175, the Company’s novel aryl hydrocarbon receptor (AHR) antagonist, in combination with immune checkpoint inhibitors in patients with advanced urothelial carcinoma who have progressed on or within three months of receiving the last dose of checkpoint inhibitors.

https://finance.yahoo.com/news/ikena-oncology-receives-fda-fast-130000639.html

With 2nd chance at FDA approval, BioMarin preps for hemophilia gene therapy launch

 With the recent FDA approval for Sanofi and Sobi’s Altuviiio, the hemophilia A field is heating up. But a subsequent approval for BioMarin's gene therapy Roctavian could bring even more change.

If approved by the FDA's decision deadline of March 31, BioMarin's Roctavian would be the first hemophilia A gene therapy in the U.S. The drug would provide a one-time treatment option for patients who otherwise have to take routine prophylactic treatments and often require transfusions.

Roctavian works by delivering a gene that helps the body produce a protein called factor VIII, which helps the blood clot. For patients with hemophilia, blood doesn't clot properly because of a lack of the protein, raising the risk of serious complications.

During BioMarin’s recent fourth-quarter earnings conference call, company executives delved into their Roctavian outlook. Even ahead of the potential approval, the company is seeing interest from patients in the U.S. 

So far, around 300 U.S. patients have “engaged with BioMarin directly” to learn more about the treatment, CEO Jean-Jacques Bienaimé said on the call. The company will follow up with those patients directly upon the launch, Executive Vice President and Chief Commercial Officer Jeff Ajer added.

The treatment won an approval in Europe last August. So far, BioMarin has secured a major payer agreement in Germany, the first European country where Roctavian will launch, at a price of 1.5 million euros per treatment. A federal agreement on reimbursement is being negotiated.

In the U.S., some uncertainty remains about the exact timing of the launch.

While the drug is currently slated for an FDA decision on March 31, there's a chance the agency might delay its review, BioMarin's president of R&D, Hank Fuchs, M.D., explained on the call.

That's because BioMarin recently submitted three-year phase 3 data on the drug as requested by the FDA. If the agency sees that as a "major amendment" to the application, it could trigger a three-month delay, Fuchs said.

That wouldn't be the first regulatory delay for the medicine. After rejecting the gene therapy in 2020, the FDA requested more details and analyses last June, pushing BioMarin’s resubmission back to September 2022.

Meanwhile, as part of the latest review, the FDA in December conduced an inspection of the company’s manufacturing facility in California, coming back with comments and observations about the production setup. BioMarin believes all of the FDA's findings are "addressable," Fuchs said. 

Before March 31, the FDA will also inspect some of the clinical study sites.

On the subject of reimbursement in the U.S., the company is currently fine-tuning a warranty contract to offer to payers. Under such an agreement, the company would stand behind the drug's efficacy and repay some of the costs if the drug doesn't work as expected.

Gene therapy prices are quite high, and Roctavian will be no different. However, the Institute for Clinical and Economic Review (ICER) has said BioMarin's gene therapy would be cost effective at a price of $2.5 million. That’s because regular prophylaxis adds up over a patient’s lifetime. For instance, ICER estimated the annual cost of managing the disease with Roche’s Hemlibra is $640,000 per year.

Elsewhere in the hemophilia field, CSL and uniQure's hemophilia B gene therapy last November won an FDA approval. That drug sports a $3.5 million list price.

Once payer talks are sorted and the FDA approves Roctavian, the drug will be ready for launch. BioMarin has homed in on a “relatively small number” of the largest and most capable hemophilia treatment centers to dole out the therapy, Ajer said. The company has manufactured enough supply to cover both the U.S. and European markets.

BioMarin projects 2023 revenues for the therapy to come in between $100 million and $200 million based on the "current uncertainty" of U.S. approval timing.

https://www.fiercepharma.com/pharma/biomarin-prepares-hemophilia-gene-therapy-roctivians-second-chance-fda-approval

Voyager: $25 M Payment for License of Next-Gen AAV Capsids for Multiple Neurologic Disease

 Options exercised on two targets; Voyager eligible to receive associated development, regulatory, and commercial milestones, plus tiered royalties

With option exercise, potential to expand agreement to include capsids for up to two additional targets extends for next 18 months

https://finance.yahoo.com/news/voyager-therapeutics-receive-25-million-120000507.html

Vir upped to Overweight from Neutral by JPMorgan

Target to $34 from $35

https://finviz.com/quote.ashx?t=VIR&ty=c&ta=1&p=d