Peter Miller, Optinose CEO

Nasal spray mak­er clinch­es first PhI­II win it needs to reach a much, much big­ger mar­ket

Four and a half years af­ter first go­ing pub­lic on the promise of build­ing out a fran­chise for its nasal spray, Opti­nose says it has the land­mark Phase III da­ta to fi­nal­ly move for­ward.

The Yard­ley, PA-based biotech is re­port­ing pos­i­tive topline re­sults from Re­Open1, sug­gest­ing that pa­tients with chron­ic si­nusi­tis would im­prove af­ter us­ing XHANCE, an “ex­ha­la­tion” de­vice that de­liv­ers a cor­ti­cos­teroid named flu­ti­ca­s­one.

A brief surge in the stock price as high as 25% quick­ly gave way to more tem­pered growth, and shares $OPTN are up 1.84% to $2.77.

With­out break­ing out the num­bers, Opti­nose said the tri­al, which en­rolled a to­tal of 332 pa­tients, met both co-pri­ma­ry end­points com­pared to place­bo. The first mea­sured symp­toms by a com­pos­ite score of nasal con­ges­tion, fa­cial pain or pres­sure sen­sa­tion and nasal dis­charge at the end of week four; the sec­ond tracked the change from base­line to week 24 in per­cent of eth­moid and max­il­lary si­nus vol­ume oc­cu­pied dis­ease, as mea­sured by CT scan.

While XHANCE is al­ready ap­proved for peo­ple with nasal polyps, Opti­nose has long sug­gest­ed that chron­ic si­nusi­tis — the kind of nasal con­ges­tion and dis­charge that oc­curs with­out nasal polyps — rep­re­sents a much big­ger, and yet un­reached, mar­ket.

Ap­prox­i­mate­ly 1 mil­lion pa­tients in the US are di­ag­nosed with nasal polyps, CEO Pe­ter Miller said last No­vem­ber. By con­trast, Opti­nose es­ti­mates around 30 mil­lion Amer­i­cans live with chron­ic si­nusi­tis — and there are no FDA-ap­proved treat­ments for them.

A la­bel ex­pan­sion for chron­ic si­nusi­tis will ex­pand the tar­get pa­tient pop­u­la­tion for which our spe­cial­ty-fo­cused sales force can pro­mote XHANCE as an ap­pro­pri­ate treat­ment from ap­prox­i­mate­ly 1 mil­lion pa­tients to ap­prox­i­mate­ly 3 mil­lion pa­tients di­ag­nosed and treat­ed by the physi­cians that we tar­get to­day. In ad­di­tion, there is po­ten­tial for a part­ner to lever­age their de­ployed pres­ence in pri­ma­ry care to ex­pand pro­mo­tion of XHANCE to a to­tal of up to 30 mil­lion peo­ple in the U.S. who have chron­ic si­nusi­tis.

Ac­cord­ing to in­ves­ti­ga­tors, the most com­mon side ef­fects were nose bleeds, na­sopharyn­gi­tis, asth­ma and cataract (nu­clear and cor­ti­cal).

A sec­ond piv­otal tri­al is on­go­ing and Opti­nose ex­pects topline re­sults in the sec­ond quar­ter.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.