Gene si­lenc­ing com­pa­ny NeuBase Ther­a­peu­tics to re­verse merge its way on­to Nas­daq via trou­bled mi­cro­cap Ohr

Di­et­rich Stephan

Di­et­rich Stephan, one of the pi­o­neers in the field of pre­ci­sion med­i­cine, was in process of rais­ing ven­ture funds for his gene-si­lenc­ing com­pa­ny NeuBase Ther­a­peu­tics when he was in­tro­duced to the team at the strug­gling eye drug de­vel­op­er Ohr Phar­ma­ceu­ti­cal $OHRP. Ini­tial con­ver­sa­tions com­menced less than a month ago, and on Thurs­day, the two sides came to an agree­ment — NeuBase would cat­a­pult on­to the Nas­daq via a re­verse merg­er with Ohr, and pro­pel the de­vel­op­ment of its rare ge­net­ic dis­ease drugs.

NeuBase’s mod­u­lar an­ti­sense pep­tide nu­cle­ic acid (PNA) plat­form — dubbed PA­TrOL — is de­signed to im­prove gene si­lenc­ing ther­a­pies by fus­ing the ad­van­tages of syn­thet­ic ap­proach­es with the pre­ci­sion of an­ti­sense tech­nolo­gies, which in­hib­it the pro­duc­tion of dis­ease-caus­ing pro­teins.

NeuBase’s tech­nol­o­gy has sev­er­al ad­van­tages over tra­di­tion­al an­ti­sense oligonu­cleotides, ac­cord­ing to Stephan, who cur­rent­ly serves as a pro­fes­sor of hu­man ge­net­ics at the Uni­ver­si­ty of Pitts­burgh and has found­ed and co-found­ed at least 13 com­pa­nies, in­clud­ing NeuBase.

Ja­son Slak­ter

NeuBase’s tech­nol­o­gy al­lows for rapid drug de­sign due to the mod­u­lar na­ture of the PA­TrOL plat­form, se­lec­tiv­i­ty for spe­cif­ic RNA/DNA se­quences, the abil­i­ty to pen­e­trate the blood brain bar­ri­er, broad sys­temic dis­tri­b­u­tion for mul­ti-tis­sue dis­ease and high cell per­me­abil­i­ty. Ear­ly in vi­vo da­ta have al­so in­di­cat­ed no im­mune re­sponse and a low cost of goods, which could be es­sen­tial for scal­a­bil­i­ty in po­ten­tial­ly ad­dress­ing a wide range of ge­net­ic dis­eases, in­clud­ing can­cer.

But at this point, NeuBase is still in the process of val­i­dat­ing its ap­proach and has much work ahead — it is still con­duct­ing pre­clin­i­cal stud­ies, and ex­pects to en­ter the clin­ic with­in the next few years.

“Io­n­is has gen­er­at­ed aware­ness around the po­ten­tial for an­ti­sense tech­nolo­gies, but we are con­fi­dent that our PA­TrOL plat­form has sig­nif­i­cant ad­van­tages that will al­low us to ex­pand be­yond the space that Io­n­is $IONS has cre­at­ed,” Stephan told End­points News.

The com­pa­ny plans to file an IND for a drug for Hunt­ing­ton’s dis­ease in 2020 and one for my­oton­ic dy­s­tro­phy in 2021.

If the deal with Ohr is con­sum­mat­ed, NeuBase has pro­posed trad­ing un­der the sym­bol $NB­SE and will re­tain the ma­jor­i­ty 80% stake of the com­bined com­pa­ny, while Ohr will get a 20% stake. Stephan will lead the com­bined com­pa­ny.

“Fol­low­ing a com­pre­hen­sive re­view of strate­gic al­ter­na­tives, Ohr’s Board of Di­rec­tors con­clud­ed that the pro­posed trans­ac­tion with NeuBase is in the best in­ter­est of our stock­hold­ers…we in­tend to hold a spe­cial meet­ing of Ohr share­hold­ers in the first half of 2019 to vote on this merg­er,” Ohr CEO Ja­son Slak­ter said in a state­ment on Thurs­day.

Ohr’s shares $OHRP sky­rock­et­ed near­ly 82% be­fore the bell on the news.

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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Pascal Soriot, AstraZeneca CEO (AP Images)

Pas­cal So­ri­ot cash­es in As­traZeneca’s chips on Mod­er­na for $1.2B cash in­jec­tion

While still working to prove its own Covid-19 vaccine, AstraZeneca has reportedly capitalized on the success of another.

The company has sold off its 7.7% stake in Moderna and turned it into $1.2 billion in cash, according to the Times, beefing up the reserves just as Pascal Soriot is wrapping up his $39 billion acquisition of Alexion and its rare disease pipeline.

AstraZeneca’s stock sale follows a similar move by Merck in December. But like its pharma brethren, the British giant is keeping its R&D collaborations with Moderna.

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Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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Fi­bro­Gen shares skid low­er as a sur­prise ad­comm rais­es risks on roxa OK

FibroGen will likely have to delay its US rollout for roxadustat once again.

In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed.

Just a few weeks ago, SVB Leerink analyst Geoffrey Porges predicted that the roxa approval could come ahead of the PDUFA date on March 20 — effusive despite already being let down once by the FDA’s extension of its review back in December. AstraZeneca, which is partnered with FibroGen on the chronic kidney disease-related anemia drug, disclosed regulators had requested further clarifying analyses of clinical data.

In­tro­duc­ing End­pointsF­DA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

UP­DAT­ED: Feds clear the road for J&J to start de­liv­er­ing mil­lions of dos­es of their Covid-19 vac­cine — but frets linger about run­ner-up sta­tus

All the pieces needed to trigger a third wave of Covid-19 vaccine supply to start washing over the US fell neatly into place over the weekend.

After providing for a brief mime of regulatory judiciousness, the FDA stamped their emergency approval on J&J’s Covid-19 vaccine Saturday, adding to the Biden administration’s plan aimed at ending the pandemic in the near term — at least in the US. The CDC came through on Sunday with its stamp of approval and J&J is reportedly expected to start delivering vaccine sometime in the next few days.

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