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.

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.

Phillip Gomez, SIGA CEO

UP­DAT­ED: On the back of SIGA Tech­nolo­gies' win with the FDA, the mon­key­pox virus sees the com­pa­ny spring­ing to fur­ther ac­tion

As the cases of monkeypox now sit at well over 100 worldwide and have spread to multiple continents, the orders for any type of vaccine against monkeypox are seeing nations and medical bodies looking to get their hands on anything and everything. And now SIGA Technologies seems to be getting in on the action.

According to Euronews, SIGA Technologies, a pharmaceutical company that is focused on providing medical countermeasures to biological and chemical attacks, is now in talks with several European authorities looking to stockpile its antiviral that can counter monkeypox. The drug known as tecovirimat or Tpoxx was approved by the FDA in 2018 as a vaccine for smallpox but was approved by the European Medicines Agency to also act against monkeypox, cowpox and complications from immunization with vaccinia.

Lutz Hegemann, Novartis president of global health

No­var­tis li­cens­es out leukemia drug as part of new glob­al coali­tion to in­crease ac­cess to can­cer treat­ments

The Union for International Cancer Control (UICC) has gathered a slate of Big Pharmas for its new collaboration in hopes of increasing access to cancer medicines in lower income countries, UICC announced yesterday.

Dubbed ATOM, or Access to Oncology Medicines, the coalition includes AstraZeneca, BeiGene, Novartis, Bristol Myers Squibb, Roche, Gilead, and Sanofi, among other organizations. The goal of the partnership is to increase generic and biosimilar development of cancer drugs as well as license out essential medicines to these countries. The third part of the partnership includes building up the infrastructure to diagnose cancers and properly handle cancer medicines.

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Bris­tol My­ers dusts off an old Cel­gene chemother­a­py for use in chil­dren with a rare blood can­cer

Bristol Myers Squibb is bringing new life to a chemotherapy drug from the old Celgene pipeline as it touts another approval in a rare form of blood cancer affecting young children.

The FDA on Friday approved Vidaza (chemically known as azacitidine) for pediatric patients 1 month and older with newly diagnosed juvenile myelomonocytic leukemia (JMML). The new indication marks the first approval for Vidaza in more than a decade, though it’s commonly used to treat acute myeloid leukemia and myelodysplastic syndromes (MDS).