De­spite protests, Mile­stone Phar­ma gets clob­bered by a PhI­II crash in the clin­ic

Mile­stone Phar­ma had all the ear­marks of a clas­sic biotech IPO sto­ry when it went pub­lic last year.

The lit­tle 12-year-old biotech had pos­i­tive mid-stage da­ta to tout to in­vestors that un­der­scored the po­ten­tial of a nasal spray treat­ment to halt parox­ys­mal supraven­tric­u­lar tachy­car­dia — or PSVT — events. A big syn­di­cate put up $80 mil­lion to help them lay a com­mer­cial foun­da­tion. And the stock peaked soon af­ter the com­pa­ny de­buted on Nas­daq as they raised an­oth­er $82 mil­lion.

But the Mile­stone team — and the stock $MIST — crashed at an ug­ly twist in the Phase III de­vel­op­ment path. Their drug, etri­pamil, a fast-act­ing cal­ci­um chan­nel block­er, failed the pri­ma­ry. Shares were rout­ed, free-falling 72% ahead of the bell.

The biotech, based in Mon­tre­al, was look­ing to of­fer an at-home so­lu­tion to a rapid heart­beat that usu­al­ly is un­threat­en­ing but sends some peo­ple back to the ER over and over again. At Phase II, re­searchers said they tracked suc­cess rates of 65% to 95%, beat­ing out the 35% place­bo score.

The pri­ma­ry end­point was a com­par­i­son of the nasal ther­a­py with place­bo over 5 hours in turn­ing SVT to si­nus rhythm, and it fell far short of the mark on sta­tis­ti­cal sig­nif­i­cance with a val­ue of 0.12. It al­so fell short on re­duc­ing trips for emer­gency care, with the same p val­ue.

Joseph Olive­to Mile­stone

Mile­stone, though, isn’t go­ing to give up at this point with­out a strug­gle. There was a pos­i­tive hit on pa­tient-re­port­ed treat­ment sat­is­fac­tion, a pos­i­tive “trend” on the ER mea­sure and they drilled down to make the case on the drug’s ear­ly im­pact. And that’s what they’re tak­ing to the FDA now for ex­plorato­ry talks.

“(O)ut­comes af­ter 100 min­utes, which were af­fect­ed by a very small num­ber of place­bo pa­tients re­main­ing in the study at that time, sug­gest that the de­sign and analy­sis plan used in NODE-301 neg­a­tive­ly im­pact­ed the study’s out­come,”  said CEO Joseph Olive­to in a state­ment.

There’s more da­ta to come, but the com­pa­ny is sound­ing a note of cau­tion on how the Covid-19 out­break might af­fect tri­al op­er­a­tions at this stage.

So­cial im­age: Joseph Olive­to, Mile­stone Phar­ma

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Pearl Huang, former Cygnal Therapeutics president and CEO

UP­DAT­ED: Flag­ship builds a new start­up out of pieces from 2 of its biotechs. And a Roche vet leaves to do some­thing new

Flagship has crafted a new startup out of pieces from a pair of fledglings in the VC’s nest. And a prominent Roche veteran who ran one of the biotechs won’t be making the next leg of the journey.

The new company is called Sonata Therapeutics, which is picking up the work that Inzen was doing related to the cellular microenvironment and combining with Flagship’s Cygnal Therapeutics, which came out of stealth more than 3 years ago and put Pearl Huang — the BeiGene founder and former Roche SVP — at the helm.

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Amidst R&D reshuf­fle, Ver­tex ex­pands its pres­ence in Boston, aim­ing to be­come num­ber one

Vertex Pharmaceuticals has been one of the buzzier names in the bustling Boston biotech scene, but now the company is looking to vault to number one status — at least in terms of physical footprint.

At a ribbon cutting on Tuesday for its new Jeffrey Leiden Center for Cell and Genetic Therapies at the Boston Seaport, Vertex announced it would embark on a new project: The company will build a 344,000 square foot facility in the seaport to accommodate the company’s growing R&D needs, especially in its cell and gene therapies program.

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Martin Shkreli (Dennis Van Tine/MediaPunch/IPX)

In­fa­mous biotech ex­ec Mar­tin Shkre­li gets out of prison, hits the street

Martin Shkreli, the infamous biotech CEO who made headlines for his jeering assault on a legion of critics in and out of Congress, is back on the streets after 4 years inside a federal penitentiary.

Shkreli’s attorney put out a statement Wednesday afternoon saying that the “pharma bro” had been transferred to a halfway house in New York with a few more months to go under federal custody, slated to end September 14. Attorney Benjamin Brafman acknowledged the release and vowed that he and Shkreli are keeping quiet.

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De­spite fed­er­al ef­forts to di­ver­si­fy clin­i­cal tri­als, progress re­mains 'stag­nan­t' — re­port

While calls to diversify clinical trials have grown louder in recent years — gaining support from federal agencies such as the FDA and NIH — progress has largely stalled, according to a new report from the National Academies of Sciences, Engineering and Medicine.

Swaths of patients in racial and ethnic minority groups, as well as LGBTQIA+, pregnant and older adult populations continue to be left out of clinical trials. While some advances have been made in the last 30 years — women now account for roughly half of clinical trial participants — growth in other areas remains stagnant, according to the report, which was mandated by Congress and sponsored by the NIH.

Paul Chaplin, Bavarian Nordic president and CEO

Bavar­i­an Nordic se­cures BAR­DA con­tract for small­pox vac­cine

It seems that smallpox vaccination production is weighing on the mind of the US government. And manufacturer Bavarian Nordic is the latest company to benefit.

Just a few days after Emergent, a company that has made government contracts its lifeblood, acquired the exclusive rights to Tembexa from Chimerix, with a $225 million cash payment and an expected BARDA contract, the agency has offered a contract for smallpox vaccine production.

Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.