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.

Tal Zaks, Moderna CMO (Moderna via YouTube)

UP­DAT­ED: NI­AID and Mod­er­na spell out a 'ro­bust' im­mune re­sponse in PhI coro­n­avirus vac­cine test — but big ques­tions re­main to be an­swered

The NIAID and Moderna have spelled out positive Phase I safety and efficacy data for their Covid-19 vaccine mRNA-1273 — highlighting the first full, clear sketch of evidence that back-to-back jabs at the dose selected for Phase III routinely produced a swarm of antibodies to the virus that exceeded levels seen in convalescent patients — typically in multiples indicating a protective response.

Moderna execs say plainly that this first stage of research produced exactly the kind of efficacy they hoped to see in humans, with a manageable safety profile.

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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

Tillman Gerngross, Adagio Therapeutics CEO

An­ti­body leg­end Till­man Gern­gross is el­bow­ing his way in­to the Covid-19 R&D cru­sade: 'I don’t see this end­ing any­time soon'

One of the most influential — and outspoken — scientists at work in the field of antibody discovery is jumping into the frenzied race to create new therapeutics to treat and prevent Covid-19. And he’s operating with the conviction that the current outbreak now once again spreading like wildfire will create plenty of demand for what he has in mind.

Dartmouth professor and Adimab CEO Tillman Gerngross tells me he’s raised $50 million from a group of close VCs to spin out a new company — Adagio Therapeutics — with a full C-suite team assembled to hire up a staff and keep rolling toward the clinic.

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Who are the women blaz­ing trails in bio­phar­ma R&D and lead­ing the fight against Covid-19? Nom­i­nate them for End­points' spe­cial re­port

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.

Mer­ck KGaA takes its I/O op­tion on F-star Ther­a­peu­tics; Nephron spends $215M, eye­ing spot in Covid-19 vac­cine chain

→Merck KGaA has taken an early option on an immuno-oncology program developed at F-star Therapeutics. This is their second option in the collaboration. And they added a pair of preclinical discovery programs to the alliance as well.

Any biotech going public these days wouldn’t feel right if they didn’t upsize the offering. And that’s just what Phase I biotech Pandion Therapeutics did. The autoimmune company is now selling 7 million shares, a 1.5 million share bump, for $16 to $18 a share.

Full Bril­in­ta study re­sults show the blood thin­ner re­duces rate of sec­ondary stroke

AstraZeneca once projected its Brilinta drug to peak at $3.5 billion in sales, and though the blood thinner never reached that lofty goal, it received the latest positive signs in a string of recent good news.

The pharma released full details from its THALES study Thursday morning, which measured the effects of Brilinta and aspirin against aspirin alone in treating patients who had an acute ischemic stroke or transient ischemic attack. When taken twice daily with once-a-day aspirin for 30 days, the Brilinta combo reduced the risk of stroke and death by 17 percent, meeting the primary endpoint of the study.

GSK’s Shin­grix leader Guil­laume Pfe­fer has jumped on board Flag­ship to helm a biotech hy­brid as Afeyan’s lat­est CEO-part­ner

After spending 4 years in a senior post with GlaxoSmithKline’s star team positioning Shingrix for a blockbuster approval, Guillaume Pfefer is headed back to the biotech world — in style.

Pfefer has signed on to join Noubar Afeyan’s busy group of partners at Flagship, and he’s taking the helm of an upstart — which today is being merged with another Flagship startup — with some grand plans of its own. The announcement this morning notes that Pfefer will run Kintai Therapeutics, one of the grads of the Flagship labs.

Donald Trump and Anthony Fauci (AP Images)

Covid-19 roundup: Rus­sia hack­ers tar­get US, UK vac­cine and drug re­searchers; Fau­ci fires back at White House cam­paign to un­der­mine him

Russia has tried to steal a Covid-19 vaccine and therapeutics researcher from pharmaceutical and academic institutions in the US, UK and Canada, Britain’s National Cyber Security Centre said Thursday.

The NCSC said that hacking attempts came from a group known as APT129, also known as “Cozy Bear,” that “almost certainly operate as part of Russian intelligence services.” The Canadian Communication Security Establishment, US Department for Homeland Security, the Cybersecurity Infrastructure Security Agency, and the National Security Agency shared the assessment, the NCSC said.

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John Furey, Imvax CEO

A neu­ro­sur­geon spent the past 30 years de­vel­op­ing a neoanti­gen tu­mor vac­cine. Now he has $112M for a piv­otal test

As a neurosurgeon, David Andrews knew there wasn’t much he could do for his glioma patients after resecting — rarely fully — their tumor. Even with the best treatment and care available, median overall survival is just somewhere between 14 and 16 months.

Then in the 1990s, his mentor at Thomas Jefferson University introduced him to Renato Baserga, a pathologist who had been studying the effect of using antisense oligonucleotide to knock out the insulin-like growth factor type 1 receptor in cancers. As IGF-R1 drives tumor growth and metastasis, the preclinical reasoning went, implanting a molecule targeting the receptor together with the tumor material near lymph nodes can slow down the spread of the cancer.

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