Eloxx Program Updates

 Investigational New Drug (IND) application submitted to U.S. Food and Drug Administration (FDA) for ELX-02 for the treatment of Alport syndrome with nonsense mutations

Rebound in average UPCR 3-months post treatment provides further evidence that proteinuria remission in one out of three patients was drug related

All 3 Alport patients (100% response rate) treated with ELX-02 had biopsy confirmed disease regression, suggesting clinical benefit likely with longer treatment duration

Significant strategic interest in ZKN-013 following FDA approval to start Phase 1 single ascending dose (SAD) trial

https://www.biospace.com/article/releases/eloxx-pharmaceuticals-provides-program-updates-on-elx-02-and-zkn-013/

Mereo Bio Corporate Update

 “The first half of 2023 was highlighted by significant updates on each of our lead rare disease programs, as we announced, alongside our partner Ultragenyx, positive data from the dose-selection Phase 2 portion of the Phase 2/3 Orbit study of setrusumab in osteogenesis imperfecta (OI), and we received regulatory guidance around the pivotal study design for alvelestat in Alpha-1 Antitrypsin Deficiency-Associated Lung Disease (AATD-LD),” said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo. “With the two Phase 3 setrusumab studies underway, which triggered a $9 million milestone payment from Ultragenyx, and with continued progress being made on the alvelestat program, we believe we are well positioned with several important potentially value creating inflection points expected in the coming quarters. With cash and short-term deposits of $53.1 million (£42.1 million) as of June 30, 2023, and with the subsequent milestone payment from our partner Ultragenyx, plus the sale of common stock under our at-the-market offering program, we continue to expect that we have sufficient runway to fund operations into 2026.”

https://www.biospace.com/article/releases/mereo-biopharma-reports-interim-financial-results-for-the-six-months-ended-june-30-2023-and-provides-corporate-update/

Nurix, Seagen Targeted Protein Degradation, Antibody-Drug Conjugation to Advance New Cancer Therapeutics

 Nurix and Seagen to develop a portfolio of Degrader-Antibody Conjugates (DACs): antibodies that deliver a targeted protein degrader payload to selectively kill cancer cells

Nurix to receive a $60 million upfront payment, in addition to the potential for approximately $3.4 billion in milestone payments plus future royalties

Nurix retains an option for U.S. profit sharing and co-promotion on two products arising from the collaboration

Nurix to host a conference call today, September 7 at 8:30 a.m. ET

At 8:30 a.m., ET, September 7, 2023, Nurix will host a conference call and webcast to discuss this update. The live webcast, with an accompanying presentation, will be accessible under the Events and Presentations page in the Investors section of the company’s website here. To participate in the live conference call, please follow this link. A replay of the webcast and call will be archived on the Nurix website for approximately 30 days after the event.

https://finance.yahoo.com/news/nurix-announces-strategic-collaboration-seagen-110000783.html

Elicio: Foundation Grant to Fund Research for Two Therapeutic Cancer Vaccines

 

• Funds will advance research into ELI-007, a mutant BRAF-peptide vaccine, and ELI-008, a p53 hotspot mutation-peptide vaccine, with the aim of developing multivalent cancer vaccines targeting several mutations

• This $2.6 million grant is an addition to the $2.8 million that GIRF awarded Elicio in September 2022

https://finance.yahoo.com/news/elicio-therapeutics-receives-2-6-120000387.html

Roche Sees Return on $310M Investment with Alnylam’s Phase II Hypertension Win

 Alnylam Pharmaceuticals is already making progress on its July 2023 deal with Roche to co-develop and commercialize zilebesiran, the former’s investigational RNAi therapeutic. The Cambridge biotech reported positive topline Phase II results Thursday for its partnered gene-silencing therapy to treat hypertension. 

In the KARDIA-1 trial, Alnylam’s zilebesiran met the primary endpoint of significantly reducing 24-hour mean systolic blood pressure (SBP) at three months for patients at high cardiovascular risk with hypertension. Zilebesiran helped lower patients’ blood pressure by more than 15 mmHg over placebo at the two highest doses tested. Key secondary endpoints for the trial were also met, according to Alnylam. 

Most blood pressure drugs work by getting rid of excess water in the body to relax the blood vessels and make the heart pump with less force. Conversely, zilebesiran is a gene-silencing therapy that inhibits liver-expressed angiotensinogen (AGT), which plays a key role in the pathogenesis of hypertension. The RNAi therapeutic was associated with durable knockdown of serum AGT levels through month six. 

The safety and tolerability profile were “encouraging,” according to Alnylam, with one death due to cardiopulmonary arrest that was considered unrelated to the drug. The adults enrolled in the study had untreated hypertension or who were on stable therapy with one or more medications. 

A second Phase II study of the drug, KARDIA-2, is underway evaluating zilebesiran in combination with one of three standard antihypertensive agents. Topline results are anticipated in early 2024. 

Cardiovascular disease remains the leading cause of death globally, with hypertension its most prevalent modifiable risk factor for premature cardiovascular death.   

“Despite the availability of several classes of oral anti-hypertensive treatments, up to 80% of individuals globally remain uncontrolled, leaving them at an increased risk of cardiovascular, cerebrovascular and renal disease,” George L. Bakris, director of the American Heart Association Comprehensive Hypertension Center, said in a statement. 

Roche inked a co-development and commercialization agreement with Alnylam for zilebesiran less than two months ago. Under the deal, Alnylam snagged $310 million upfront in cash with a potential $2.8 billion on the line in milestone payments. The two will share U.S. profits equally if the drug gets approved, while Roche holds the rights outside the country. 

In January 2023, AstraZeneca scooped up cardiorenal disease biotech CinCor Pharma for its investigational hypertension treatment. A Phase II study of baxdrostat showed the drug was able to reduce SBP in patients with treatment-resistant hypertension by 11 mmHg over placebo. AstraZeneca is looking to combine the treatment with its blockbuster diabetes drug Farxiga.  

https://www.biospace.com/article/roche-sees-return-on-310m-investment-with-alnylam-s-phase-ii-hypertension-win-/

Baxter takeover speculation

 Baxter (BAX) pared most of an earlier decline amid some vague takeover speculation.

https://seekingalpha.com/news/4009960-baxter-pares-decline-amid-vague-takeover-speculation

Insulet shares slump on concerns of sales hit from new diabetes drugs

 Insulin pumps maker Insulet Corp said on Thursday a new generation of diabetes drugs, known as GLP-1s, could delay the time that diabetes patients become dependent on insulin, sending its shares down 10%.

CEO James Hollingshead said at the Wells Fargo Healthcare Conference the company does not expect its long-term market for insulin pumps to be impacted.

"What we might see is GLP-1s impacting the time to progression on the insulin therapy," said Hollingshead.

Insulet manufactures and sells insulin delivery devices, under 'Omnipod' brand, which eliminate the need for multiple daily injections for people with insulin-dependent diabetes.

https://news.yahoo.com/insulet-shares-slump-concerns-sales-145629493.html